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Displaying 1 - 200 of 213
Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study is for 1 to 21-year-olds whose hematologic cancer has relapsed to take medication called Tagraxofusp. Relapse means that cancer has come back after treatment. Tagraxofusp is a drug approved by the FDA (Food and Drug Administration) but is not approved to treat the cancers of people in this study. This study is being done to find out if…
Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study will enroll with children, adolescents and young adults that are less than 22 years old that have returned or 'relapsed' acute myeloid leukemia (AML) diagnoses. Within this group, the study is also targeting those are also unable to receive additional chemotherapy treatments containing anthracycline, which is a type of antibiotic that…
Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This is a phase 1 study for participants with relapsed/refractory acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), or juvenile myelomonocytic leukemia (JMML) diagnosis. This study will observe the safety, side effects, and best dose of Imetelstat as an investigational drug, or drug that is not approved by the Food and Drug Administration (FDA…
Condition: Pediatrics
Investigator: Robin Goland, MD
Status: Currently Recruiting
A future without Type 1 Diabetes (T1D) starts with you. Get screened! Thanks in large part to T1D family members, we understand T1D as a disease that progresses in three distinct stages. This new definition in conjunction with TrialNet's Pathway to Prevention screening, allows for earlier detection and intervention.
Condition: Pediatrics
Investigator: Ashley Blanchard, MD
Status: Currently Recruiting
The purpose of the study is to study how to make the home safer for youth at risk of suicide. Our goal is to learn how to better help families whose children are seen in the emergency room for self-injury or suicidal thoughts or actions.
Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Currently Recruiting
TReatment for ImmUne Mediated PathopHysiology (TRIUMPH) is a multi-center, three-arm clinical trial of immunosuppressive therapy for children with acute liver failure. This is a double-blind study, so the study doctors and participants will not know to which arm the participants are assigned. The study will determine if corticosteroids or equine anti-…
Condition: Pediatrics / Cancer
Investigator: James Hinkley Garvin Jr, MD
Status: Currently Recruiting
Children and young adults with diffuse midline glioma will be treated with Focused Ultrasound (FUS) with DEFINITY microbubbles and neuro-navigator-controlled sonication. The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known cure exists, and determine…
Condition: Pediatrics
Investigator: Maria Kwok, MD, MPH
Status: Currently Recruiting
What's fast-moving, all-encompassing, and plays no favorites? Sepsis, an indiscriminate systemic condition that escalates so rapidly, national guidelines recommend treatment begin within 20 minutes of arrival to the emergency department. Crystalloid resuscitation with normal saline or balanced fluids (including lactated Ringers and Plasma-Lyte) is the…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Currently Recruiting
You are being asked to participate in this study because you have indolent SM (ISM) or because you have Monoclonal mast cell activation syndrome (mMCAS). Currently, there are no available targeted therapies for patients with ISM or mMCAS. The study will test the drug BLU-263. BLU-263 is a new study drug that is being developed for treating people like you,…
Condition: Pediatrics
Investigator: Meyer Kattan, MD
Status: Currently Recruiting
We are interested in learning more about these diseases and how to treat them. Our research program is conducting several studies including: a study to learn if treatment with a drug named dupilumab can help reduce asthma attacks in children, a study to observe a specific type of irritation in the lungs and how it relates to asthma symptoms, and more! If…
Condition: Cancer / Leukemia
Investigator: Andrew Lipsky, MD
Status: Currently Recruiting
This research is being done because Richter's transformation, which is a life-threatening complication of chronic lymphocytic lymphoma (CLL), and is associated with poor overall survival rate. The study drug administration is a combination of the investigational drug, Polatuzumab vedotin, in combination with Rituximab, Etoposide, Prednisone,…
Condition: Pediatrics
Investigator: Jennifer Bain, MD
Status: Currently Recruiting
This is a Phase IIA Multicenter, open-label, 12-week study to investigate the pharmacokinetics and safety and to provide proof of mechanism of the drug, Alogabat (RO7017773) in children and adolescents aged 5-17 years old with Angelman Syndrome with the deletion genotype.
Condition: Pediatrics
Investigator: Mishaela Rubin, MD
Status: Currently Recruiting
We are looking for children between the ages of 8-14 with Type 1 diabetes and without Type 1 diabetes who are interested to participate on a research study to find out how bones are affected in children and adolescents with Type 1 diabetes as compared to people without Type 1 diabetes.
Condition: Pediatrics
Investigator: Ashwini Rao, EdD, OTR
Status: Currently Recruiting
Child to Adult Neurodevelopment in Gene Expanded Huntingtons Disease (ChANGE HD) HD has long been considered an adult-onset disease. However, research has shown that brain changes are evident before motor symptoms begin. In fact, the gene responsible for HD plays a role in brain development and is present throughout the lifespan. With gene knockdown therapy…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Currently Recruiting
You are being asked to participate in this study because you have Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) and we are testing the study drugs, SEA-CD70, to find out what its side effects are and to see if the study drug is safe for people with these types of cancer are testing to see if SEA-CD70 is safe for people, and its side effects…
Condition: Pediatrics
Investigator: Usha Krishnan, MD
Status: Currently Recruiting
This study is being done to determine whether early combination therapy with two study drugs called sildenafil (REVATIO) and bosentan (TRACLEER) can improve pulmonary hypertension when compared to using sildenafil alone. Pediatric pulmonary arterial hypertension (PAH) is a severe medical problem that often worsens with time. It is caused by problems with…
Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study is called a screening study and the purpose of this study to find better ways to diagnose and treat leukemia that has come back after treatment or difficult to treat. Bone marrow, blood, and medical information about cancer and treatment will be collected. The results from this screening study may give other information about leukemia that is…
Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study is called a screening study and the purpose of this study to find better ways to diagnose and treat leukemia that has come back after treatment or difficult to treat. Bone marrow, blood, and medical information about cancer and treatment will be collected. The results from this screening study may give other information about leukemia that is…
Condition: Pediatrics
Investigator: Yufeng Shen, PhD
Status: Currently Recruiting
Each year more than 1,000 babies in the United States are born with a Congenital Diaphragmatic Hernia (CDH). For the majority of these babies, the cause of the CDH is unknown. The DHREAMS (Diaphragmatic Hernia Research & Exploration; Advancing Molecular Science) study was created to improve the understanding of the molecular genetic basis of CDH. This…
Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Currently Recruiting
In this research, we want to learn more about how to improve care for children who are listed for or who have received a liver transplant. The goal of this research is to look at how different hospitals treat these children and to look at their outcomes to help improve care, decrease complications, shorten length of hospital stay, reduce hospital re-…
Condition: Pediatrics
Investigator: Liat Itzhaky, PhD
Status: Currently Recruiting
Your child is invited to participate in a research study. Participation in the study includes one meeting at Washington Heights. Results from this study will assist in modifying an existing behavioral intervention to fit children of this age. For more details, please contact Dr. Liat Itzhaky at 646-774-7634 or e-mail: li2185@cumc.columbia.edu. Participants…
Condition: Skin Cancer / Melanoma
Investigator: Alexander Wei, MD
Status: Currently Recruiting
The purpose of this study is to identify the safest and most tolerable dose of the study drug, DYP688 that can be given to patients. Also to evaluate how well DYP688 might work in treating certain types of cancer including metastatic uveal melanoma and other melanomas with GNAQ/11 mutations (or genetic changes to the gene GNAQ/11). Metastatic means the…
Condition: Skin Cancer / Melanoma
Investigator: Benjamin Izar, MD
Status: Currently Recruiting
The purpose of this study is to compare V940 plus pembro given after surgery to a placebo plus pembro given after surgery and to study how effective the study drug is. A placebo looks like a study drug, but it has no active ingredients. V940 has not yet been approved by the Food and Drug Administration (FDA). Pembrolizumab is FDA approved but it has not…
Condition: Pediatrics
Investigator: Rachel Marsh, PhD
Status: Currently Recruiting
To learn more, click the blue box "Do you qualify" and our study team will contact you. This research study will examine how an iPad-based cognitive training game combined with cognitive behavioral therapy (CBT) helps children with excessive worries and related repetitive behaviors (obsessive compulsive disorder). After completing the training,…
Condition: Pediatrics
Investigator: Mercedes Martinez, MD
Status: Currently Recruiting
The purpose of this study is to better understand how common and how severe pruritis (itching) is in children and adults under 21 years of age with Primary Sclerosing Cholangitis (PSC). The study involves the completion of two short online surveys. If you have any questions regarding this study, please contact the Transplant Clinical Research Center ( TCRC).
Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
This study will utilize an investigational drug combination (also known as the study drugs) called relatlimab and nivolumab as a possible treatment for recurrent/relapsed (returned) or refractory (treatment-resistant) cHL and NHL. An investigational drug combination is one that has not been approved by regulatory agencies, such as the United States (US)…
Condition: Pediatrics
Investigator: Randy Auerbach, PhD
Status: Currently Recruiting
We are recruiting depressed adolescents ages 13-18 years. As a participant in our study, we will: (1) interview your child about current and past thoughts, feelings, and behaviors, (2) teach your child mindfulness strategies, (3) collect brain activity data with MRI (non-invasive) while your child practices mindfulness, and (4) send short surveys to your…
Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Currently Recruiting
In this study, we will collect information from the medical record of pediatric liver transplant recipients who received live vaccines as part of their routine post-transplant care. This data will be combined with data from other centers across the United States and Canada to help give us a better understanding of the safety and efficacy of live vaccines in…
Condition: Pediatrics / Child and Adolescent Health
Investigator: Lisa Eisler, MD
Status: Currently Recruiting
We are studying the effects of iron supplementation in patients undergoing surgery for scoliosis. If you are scheduled or are planning to schedule a scoliosis surgery in greater than 3 months, please contact us to determine eligibility.
A Study for Patients with Advanced Melanoma, Solid Tumors or Lymphoma Using Study Drug Pembrolizumab
Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Currently Recruiting
The purpose of this study is to determine if a study drug called pembrolizumab is safe and effective in the treatment of children and adolescents who have advanced melanoma, or advanced, relapsed, or refractory PD-L1 positive malignant solid tumor or other lymphoma, relapsed or refractory classical Hodgkin lymphoma (rrcHL), advanced, relapsed or refractory…
Condition: Pediatrics
Investigator: Lawrence Lustig, MD
Status: Currently Recruiting
The CHORD clinical trial, sponsored by Decibel Therapeutics, is looking at the use of the gene therapy DB-OTO for children born with hearing loss related to changes in the OTOF gene. The goal of this trial is to assess different doses of DB-OTO to find out if it is safe, well-tolerated, and its effect on improving hearing. DB-OTO is a gene therapy that has…
Condition: Pediatrics
Investigator: Yufeng Shen, PhD
Status: Currently Recruiting
Study examining the genetic causes of birth defects.
Condition: Pediatrics / Child and Adolescent Health
Investigator: Ilene Fennoy, MD
Status: Currently Recruiting
We know that puberty is a major time of change! Adolescents develop new decision-making skills and independence. It is possible that some of these changes are due to the hormonal changes during puberty. One of the ways we treat transgender and gender non-conforming teens is to suppress or "block" puberty. We know that this is a safe thing to do…
Condition: Pediatrics
Investigator: Vidhu Thaker, MD
Status: Currently Recruiting
We will enroll children with severe early onset obesity (BMI 99th percentile) diagnosed prior to 6years of age or other endocrine disorders. We will ask questions about their health, eating behavior and perform a brief physical examination. We will collect saliva or blood to perform genetic testing. We will also invite family members to participate in the…
Condition: Cancer / Leukemia
Investigator: Ran Reshef, MD
Status: Currently Recruiting
The study involves research of two study drugs (TSC 100 and TSC 101), given in combination with the standard of care (SOC). The purpose of the study is to see if TSC 100 and TSC 101 are safe and effective in treating your blood cancer. The study also is measuring the effect of these study drugs on your immune system (immunogenicity of study drugs). TSC 100…
Condition: Pediatrics
Investigator: Ilene Fennoy, MD
Status: Currently Recruiting
We are doing this research study to find out what happens to children who are overweight and how the things we recommend as a part of routine clinical care affect your child. The purpose of this study is to understand what happens to children as a result of being overweight, to link these outcomes to specific clinical measures, and to determine how…
Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to find the best dose of Asciminib that can be given safely in children with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP) having previously been treated with one or more tyrosine kinase inhibitors (TKIs). Tyrosine kinase inhibitors are a group of medications that block chemical…
Condition: Pediatrics
Investigator: Martha Welch, MD
Status: Closed
The purpose of this study is to compare neurodevelopment and activity in infants born very preterm (26 to 33 6/7 weeks gestational age (GA)) receiving Standard Care (SC) or Family Nurture Intervention (FNI) in the neonatal intensive care unit (NICU). This study investigator hypothesizes that FNI will improve: i) neonatal electroencephalographic activity ii…
Condition: Cancer / Leukemia
Investigator: Markus Mapara, MD
Status: Closed
The purpose of the study is to learn if a transplant using bone marrow recovered from deceased organ and tissue donors is safe and effective in patients undergoing bone marrow transplant (BMT). This study treatment does not include any investigational drugs. The medicines and procedures in this study are standard of care for transplant. Bone marrow cells…
Condition: Cancer / Skin Cancer
Investigator: Benjamin Izar, MD
Status: Closed
The purpose of this study is to see if IMC-F106C, in combination with nivolumab, works better than the standard therapy (nivolumab alone or in combination with relatlimab) at making tumors stop growing or shrink. IMC-F106C has not yet been approved by the Food and Drug Administration (FDA). Nivolumab and the combination of nivolumab + relatlimab is FDA…
Condition: Skin Cancer / Melanoma
Investigator: Benjamin Izar, MD
Status: Closed
The purpose of this study is to see if the combination of fianlimab (REGN3767) and cemiplimab (REGN2810) is an effective treatment compared to pembrolizumab in participants that have had melanoma removal surgery but are still at high risk for the return of the disease. This study involves 2 study drugs: fianlimab (REGN3767) and cemiplimab (REGN2810). The…
Condition: Pediatrics / Cancer
Investigator: Prakash Satwani, MD
Status: Closed
The main purpose of this research study is to determine if the experimental product, KTE-C19, when administered after you receive 3 days of chemotherapy, is safe and effective in treating your leukemia or non-Hodgkin lymphoma.
Condition: Pediatrics
Investigator: Prakash Satwani, MD
Status: Closed
The main purpose of this research study is to determine if the experimental product, KTE-C19, when administered after you receive 3 days of chemotherapy, is safe and effective in treating your leukemia or non-Hodgkin lymphoma.
Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Closed
The purpose of the study is to collect scientific data on pediatric liver transplantation to learn more about how children do after liver transplant. Collecting these data is expected to help researchers learn more about liver transplant in children. This information may improve medical care of your child and other children who need and who have received…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
You are being asked to participate in this study because you have acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) or leukemia of mixed lineage (the leukemia cells cannot be classified as AML or ALL or where both types of AML and ALL cells are present). Because you have already received standard therapy that is no longer helping, or there is…
Condition: Pediatrics
Investigator: Brooke Aggarwal, EdD, MS, FAHA
Status: Closed
High school students ages 14-18 and their parent(s) may be eligible to participate in a 12-week (consisting of a 6-week pre-pilot and 6-week pilot) sleep health education program.
Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 study of a drug called uproleselan. We are testing new experimental drugs such as uproleselan in the hopes of finding a treatment that may be effective against acute myeloid leukemia, myelodysplastic syndrome or mixed phenotype acute leukemia that has come back or that has not responded to standard therapy. This study looks at how well…
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
This study will be performed to determine if Panzyga (the study drug) can help with preventing infections in patients who have chronic lymphocytic leukemia (CLL). Panzyga is a study drug administered via injection into the vein, which contains human antibodies also called immunoglobulins. It is manufactured by Octapharma. Your participation in this clinical…
Condition: Pediatrics
Investigator: Olatundun Williams, MD
Status: Closed
This study is being done to identify the best outcomes for hematopoietic cell transplantation (HCT) or other treatment (enzyme replacement or gene therapy), which is life-saving therapy for children with SCID (severe combined immunodeficiency), leaky SCID, Omenn syndrome and reticular dysgenesis.
Condition: Pediatrics
Investigator: Daniel Tsze, MD
Status: Closed
The purpose of this research study is to create a decision-making tool that will help doctors decide which children with headaches require a CT ("CAT scan," "computed tomography") or MRI scan of their head as part of their emergency department care.
Condition: Pediatrics / Down Syndrome
Investigator: Michael DiLorenzo, MD
Status: Closed
Down syndrome (DS) occurs in roughly 1 out of 700 births in the U.S. Half of all children with Down syndrome have congenital heart disease (CHD), often requiring corrective heart surgery. Even though CHD in children without DS is known to be associated with developmental problems, few studies have explored how CHD can affect the development and behavior of…
Condition: Pediatrics
Investigator: Olatundun Williams, MD
Status: Closed
The purpose of this study is to understand the life course of PIRD patients and their family members that have the same gene change but do not have the same symptoms. PIRD is mostly due to inherited changes in the genes that normally have immune cells fight infections and regulate the immune system. There are many types of problems that patients with PIRD…
Condition: Pediatrics
Investigator: Dana Goldner, MD
Status: Closed
This study evaluates whether odevixibat is safe and effective compared to placebo in children with biliary atresia who have recently undergone a Kasai procedure. This is a double-blind study, so the study doctors and the child/parent will not know whether the child is receiving odevixibat or placebo. Participation will involve several research visits over…
Condition: Cancer / Skin Cancer
Investigator: Shaheer Khan, DO
Status: Closed
This trial is being done to look at the effects of RP1 in kidney, liver, heart, and lung transplant recipients with skin cancer. These effects could be good (for example, shrinking of tumors) or bad (called side effects). Researchers will study the effects and how long they last. This study is being done to find out how effective RP1 is in treating the…
Condition: Cancer / Leukemia
Investigator: Markus Mapara, MD
Status: Closed
You are being asked to take part in this study because you have acute myeloid leukemia (AML). Vor Biopharma, is a company that is testing a possible new treatment for AML. This possible new treatment, is VOR33. In this study, your blood-forming stem cells will be genetically modified to become VOR33. VOR33 has not been approved by the Food and Drug…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to evaluate the effects of etavopivat, good and bad, in individuals with myelodysplastic syndrome (MDS).
Condition: Cancer / Childhood Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the best dose of Quizartinib that can be given safely with chemotherapy in children and young adults with acute myeloid leukemia (AML) which has come back or has not responded to the standard therapy. Quizartinib will be given by mouth once a day from Day 6 through Day 28. It will be given in combination with…
Condition: Cancer / Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the best dose of Quizartinib that can be given safely with chemotherapy in children and young adults with acute myeloid leukemia (AML) which has come back or has not responded to the standard therapy. Quizartinib will be given by mouth once a day from Day 6 through Day 28. It will be given in combination with…
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
The purpose of this study is to test the safety and effectiveness of an investigational treatment called pirtobrutinib (LOXO-305) plus venetoclax and rituximab (PVR) versus venetoclax and rituximab (VR). Pirtobrutinib is an investigational (or experimental) drug that may treat your Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma (CLL/SLL).
Condition: Pediatrics
Investigator: Daniel Tsze, MD
Status: Closed
The purpose of this research study is to find out what is the best dose of intranasal midazolam for children to help them stay calm and hold still while they are having a cut in their skin fixed.
Condition: Pediatrics
Investigator: Stephanie Levasseur, MD
Status: Closed
Although supraventricular tachycardia (SVT), including atrial flutter (AF), are the most common causes of intended in-utero fetal therapy, none of the medication used to date has been evaluated for their effects on the mother and her baby in a randomized controlled clinical trial (RCT). In the absence of such evidence, there is no consensus for the optimal…
Condition: Cancer / Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to find out if the drugs called Nivolumab and 5-Azacytidine can be given safely in children and young adults with Acute Myeloid Leukemia (AML) which has come back after treatment or has not responded to standard therapy. These drugs are thought to work by turning on genes that limit the growth of cancer cells. The study drugs…
Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to find out if the drugs called Nivolumab and 5-Azacytidine can be given safely in children and young adults with Acute Myeloid Leukemia (AML) which has come back after treatment or has not responded to standard therapy. These drugs are thought to work by turning on genes that limit the growth of cancer cells. The study drugs…
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
The purpose of this study is to find out whether people who are currently receiving treatment with venetoclax can stop treatment and remain off-treatment for at least 12 months, if they are free of residual disease (MRD- negative) when they stop treatment.
Condition: Skin Cancer / Melanoma
Investigator: Benjamin Izar, MD
Status: Closed
The purpose of this study is to determine the safety and efficacy of CMP-001 when given in combination with nivolumab for patients with unresectable (not capable of being surgically removed) or metastatic (cancer spreads to a different body part from where it started) melanoma. CMP-001 will be given as an injection into the tumor and nivolumab will be given…
Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Closed
The purpose of this study is to learn more about the treatment of children and teenagers whose leukemia has come back (has relapsed) or has not gone away after other treatments (is refractory). Participants will receive the study drug, called carfilzomib in combination with standard chemotherapy, that are dexamethasone, daunorubicin, vincristine and PEG-…
Condition: Pediatrics
Investigator: Cindy Neunert, MD
Status: Closed
The purpose of this study is to investigate the safety and effectiveness of eltrombopag (investigational drug) in treating children and adolescents with newly-diagnosed immune thrombocytopenia (ITP), which is a rare blood disorder.
Condition: Skin Cancer / Melanoma
Investigator: Benjamin Izar, MD
Status: Closed
The purpose of this study is to test the study drug ALKS 4230 to see how well the drug works on patients with advanced cutaneous melanoma or subjects with advanced mucosal melanoma and have received prior treatment with an anti-programmed death-ligand 1 (anti-PD-L1) therapy, and your cancer has spread. This study will explore how well the drug works on…
Condition: Pediatrics / Cardiology
Investigator: Stephanie Levasseur, MD
Status: Closed
Although supraventricular tachycardia (SVT), including atrial flutter (AF), are the most common causes of intended in-utero fetal therapy, none of the medication used to date has been evaluated for their effects on the mother and her baby in a randomized controlled clinical trial (RCT). In the absence of such evidence, there is no consensus for the optimal…
Condition: Cancer / Leukemia
Investigator: Ran Reshef, MD
Status: Closed
The purpose of this study is to find the highest dose of STK-009 that is safe in combination with a dose of SYNCAR-001 and to learn what side effects are experienced in people with certain lymphomas or leukemias. This study will also evaluate how much SYNCAR-001 and STK-009 is in your blood at various times, whether your immune system becomes activated,…
Condition: Cancer / Leukemia
Investigator: Ran Reshef, MD
Status: Closed
The purpose of this study is to evaluate the safety and efficacy of venetoclax and azacitidine compared to Best Supportive Care in patients with Acute Myeloid Leukemia (AML) after an allogeneic stem cell transplant (SCT). This is the first randomized study comparing venetoclax in combination with azacitidine versus best supportive care in subjects with AML…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
You are being asked to participate in this study because you have a myeloproliferative neoplasm (MPN) that has transformed to accelerated or blast phase. MPN is a group of diseases in which the bone marrow makes too many blood cells. The different types of MPN are myelofibrosis (MF), essential thrombocythemia (ET), or polycythemia vera (PV). Over time,…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
You are being asked to participate in this study because you have been diagnosed with a blood cancer called Acute Myeloid Leukemia (AML) with or suspected to have a specific gene mutation (TP53 mutation) for which you have not received any prior AML treatment. Tumor protein p53 (TP53) is a gene that codes for a tumor suppressor protein, which is normally…
Condition: Cancer / Skin Cancer
Investigator: Shaheer Khan, DO
Status: Closed
The purpose of this study is determine if cemiplimab will prevent Cutaneous Squamous Cell Cancer (CSCC) from returning after surgery and radiation.
Condition: Pediatrics / Child and Adolescent Health
Investigator: Maria Kwok, MD, MPH
Status: Closed
The purpose of this study is to evaluate the effectiveness and adverse reactions of the various types of noninvasive positive pressure supportive use in the management of children with moderate to severe acute asthma exacerbation.
Study of Olanzapine in Children Receiving Chemotherapy for Hematopoietic Stem Cell Transplant (HSCT)
Condition: Pediatrics / Cancer
Investigator: Diane George, MD
Status: Closed
This research study is to find out whether adding olanzapine to standard antiemetics will be helpful in controlling chemotherapy-induced vomiting (CINV) in children receiving high-dose cyclophosphamide for Blood/Bone Marrow Transplant (BMT) conditioning.
Condition: Pediatrics / Cancer
Investigator: James Hinkley Garvin Jr, MD
Status: Closed
The purpose of this study is to find the best dose of a drug called MTX110 and a contrast agent called gadolinium that can be given safely in children with newly diagnosed diffuse midline gliomas, which is a kind of brain tumor. All patients enrolled in the study will receive infusion of MTX110 and Gadolinium delivered with a pump directly into the tumor…
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of the study is to determine an appropriate dose of KRT-232 in combination TL-895 or KRT-232 alone for treatment of myelofibrosis (MF). There are 2 parts planned for this study, the Phase 1b that will test different doses of TL-895 given with KRT-232 and it will also test KRT-232 alone, to identify the recommended dose for Phase 2. The Phase 2 (…
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of this study is to determine the optimal dose of oral CGT9486 in patients with Advanced Systemic Mastocytosis (AdvSM) and to determine the efficacy of CGT9486 at the selected optimal dose in patients with Advanced Systemic Mastocytosis (AdvSM). This includes Aggressive Systemic Mastocytosis, Advanced Systemic Mastocytosis with an Associated…
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
The purpose of this study is to determine the effectiveness of the study treatment- bendamustine and rituximab (BR) followed by venetoclax for 12 months. The combination of the drugs bendamustine and rituximab is a commonly used treatment for your disease. Venetoclax is an oral drug that blocks a protein called BCL-2 which is present on CLL cells. With this…
Condition: Skin Cancer / Melanoma
Investigator: Brian Marr, MD
Status: Closed
The purpose of this research study is to test the safety and efficacy of AU-011 at different doses and different number of doses of study drug when it is injected into the suprachoroidal space of the eye with one or two laser light applications and to find out what effects, if any, it has on research participants with a choroidal melanoma tumor or…
Condition: Skin Cancer / Melanoma
Investigator: Mariam El-Ashmawy, MD, PhD
Status: Closed
This study consists of two treatment groups, Part 1 and Part 2. The purpose of this study is to: Part 1 - Test the safety and tolerability of the study drug IN10018, see how well the drug works on your metastatic uveal melanoma, and see how your body handles the drug. Part 2 - Test the safety and tolerability of the study drug IN10018 with cobimetinib, see…
Condition: Cancer / Leukemia
Investigator: Ran Reshef, MD
Status: Closed
Patients who will be receiving immune effector cell (IEC) therapy for the treatment of their cancer (leukemia) are being asked to participate in this study. IEC can produce a harmful side effect called Cytokine Release Syndrome (CRS). CRS involves a release of a large amount of proteins into the bloodstream. This may cause changes in blood pressure and…
Condition: Pediatrics
Investigator: Jin Hee Kim, MD
Status: Closed
In this research study we want to learn more about a potential new therapy for pain associated with endometriosis. We want to determine if a non-hormonal medication, cabergoline, will help patients with endometriosis. Cabergoline is a medication that is approved by the Food and Drug Administration (FDA) for treatment of other medical conditions. In this…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to compare the effects, both good and bad, of magrolimab in combination with azacitidine, to those of azacitidine in combination with placebo, to find out which is better for treating patients with MDS (Myelodysplastic Syndrome). Other purposes of this study include determining the quantity of magrolimab in the blood, aspects of…
Condition: Pediatrics
Investigator: Doron Amsalem, MD
Status: Closed
We are conducting a research study to explore discrimination around depression and barriers to care in youth ages 14-18. We will use what we learn to create videos to reduce such discrimination. Parental permission is required for this study. Adolescents (ages 14-18) will participate in an individual interview that will be approximately 45 minutes long. We…
Condition: Cancer / Leukemia
Investigator: Andrew Lipsky, MD
Status: Closed
The purpose of this study is to assess the safety, effectiveness, pharmacokinetic (PK how long the drug is in your body and how it is metabolized) and pharmacodynamics (PD where does the drug go in your body and what is its biological effect) of the study drug, GFH009, in patients with relapsed or refractory hematologic malignancies including acute myeloid…
Condition: Pediatrics / Cancer
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find out the effects and safety of an investigational (experimental) new drug called copanlisib in pediatric patients from 6 months to 21 years of age. This study consists in two-phase, Phase I part will determine which is the right dose of copanlisib to be used in children/adolescents/adults with relapsed (has returned) or…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this clinical research study is to evaluate the safety (any good or bad effects) of CYNK-001 and to assess the effects of CYNK-001 on your Acute Myeloid Leukemia (AML). Also, the study plans to determine the maximum tolerated dose (highest safe dose) of CYNK-001 and assess whether CYNK-001 can eliminate your residual (leftover) disease.…
Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of Vincristine Sulfate Liposome Injection (Marqibo) in combination with UK ALL R3 induction chemotherapy. The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children with acute lymphoblastic leukemia (ALL) who have relapsed…
Condition: Pediatrics / Cancer
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the highest dose of Flotetuzumab that can be given safely to patients who have a certain type of leukemia called Acute Myeloid Leukemia (AML) that has relapsed (has come back after treatment) or is refractory (never responded to treatment). Flotetuzumab is an experimental treatment that works by targeting certain…
Condition: Cancer / Skin Cancer
Investigator: Richard Carvajal, MD
Status: Closed
The purpose of the study is to determine the long-term effectiveness and safety of cemiplimab in patients diagnosed with Cutaneous Squamous Cell Carcinoma (CSCC, a type of skin cancer).
Condition: Cancer / Pediatric Solid Tumors
Investigator: Nobuko Hijiya, MD
Status: Closed
We are testing new experimental drugs such as selinexor in the hopes of finding a treatment that may be effective against tumors that have come back or that have not responded to standard therapy. The goals of this study are: To find the highest safe dose of selinexor that can be given without causing severe side effects; To learn what kind of side effects…
Condition: Pediatrics
Investigator: Jennifer Vittorio, MD
Status: Closed
The purpose of this study is to see how effective and safe the drug maralixibat is when compared to placebo in infants with biliary atresia who have had Kasai surgery within 3 weeks before participation. This is a 26-week, multicenter, double-blind, placebo-controlled, randomized parallel-group study, followed by an open-label extension (OLE). Approximately…
Condition: Pediatrics / Cancer
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to find out if an investigational drug, called ADP-A2M4, is safe and how well it works in attacking Synovial Sarcoma or Myxoid/Round Cell Liposarcoma (MRCLS) cancer cells. The study involves taking certain types of immune cells from the blood, called T cells, and changing them in a laboratory before putting them back into the…
Condition: Skin Cancer / Squamous Cell Carcinoma
Investigator: Richard Carvajal, MD
Status: Closed
The purpose of this study is to test the good and bad effects of the combined treatment, using the drugs cetuximab and avelumab as well as avelumab by itself. Avelumab is a drug that works by triggering the immune system so it can try to attack the cancer again. Avelumab works in a similar way to cemiplimab, which is the usual drug for treatment of your…
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
This research study is evaluating an investigational drug, called cirmtuzumab, in combination with astandard drug, called ibrutinib (Imbruvica), as a possible treatment for patients with CLL/SLL or MCL. All cells in the body receive signals to grow and survive, but sometimes these signals get out of control, causing too much cell growth. When the growth of…
Condition: Pediatrics
Investigator: Jennifer Vittorio, MD
Status: Closed
The social determinants of health have a large impact on health. We have previously demonstrated that an index of neighborhood socioeconomic deprivation was associated with an increased risk of medication non-adherence in children after liver transplant and an increased risk of graft failure and death in children after transplant. The present proposal seeks…
Condition: Skin Cancer / Melanoma
Investigator: Richard Carvajal, MD
Status: Closed
The purpose of the study is to evaluate the effectiveness (how well the drug works) of the investigational drug called bempegaldesleukin when combined with drug nivolumab in comparison with nivolumab alone after complete resection (removal of all sites of known disease) of melanoma in participants at high risk of cancer returning after surgery (recurrence).
Condition: Skin Cancer / Melanoma
Investigator: Mariam El-Ashmawy, MD, PhD
Status: Closed
This research study is a phase II clinical trial that will study the combination of adjuvant nivolumab and ipilimumab for the treatment of adult subjects with completely treated high-risk ocular melanoma. This study is being done to see if the combination of the study drugs is safe and effective in preventing the return of their cancer. If you are eligible…
Condition: Pediatrics / Cancer
Investigator: Stergios Zacharoulis, MD
Status: Closed
The purpose of this study is to learn more about the treatment of children and young adults patient with relapsed (has returned) and progressive medulloblastoma (brain tumor). This study is being done to find out if bevacizumab can be safely given in combination with five study drugs given by mouth, thalidomide, fenofibrate, celecoxib and alternating every…
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of this study is to find out what dose of pacritinib is safest and works best for participants. Pacritinib is an experimental drug, which is a drug that is being tested and is not currently approved by the Food and Drug Administration (FDA) in the US. The study will be conducted at about 85 study clinics in North America, Europe, Israel and…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to: Test if the lifespan of study participants is increased when uproleselan is added to standard chemotherapy medicines compared to standard chemotherapy alone; test if the number of study participants achieving disease remission is increased when uproleselan is added to standard chemotherapy medicines compared to standard…
Condition: Skin Cancer / Melanoma
Investigator: Larisa Geskin, MD
Status: Closed
The purpose of this study is to determine how effective and safe the immune drug Nivolumab is for patients who have newly diagnosed cutaneous melanoma. Nivolumab is an anti-body, a type of human protein, that is being tested to see if it will allow the body's immune system to work against tumor cells. If you are eligible for the study, you will…
Study of sertraline (Zoloft) with ara-C in combination in patients with acute myeloid leukemia (AML)
Condition: Cancer / Leukemia
Investigator: Daniel Lee, MD
Status: Closed
The purpose of the study is to evaluate the safety, tolerability (the effect of the drug on your body), pharmacokinetics (the effect of your body on the drug) and maximum tolerated dose of sertraline in combination with cytosine arabinoside (ara-C, cytarabine) when given to patients with treatment-resistant myeloid leukemia. In addition, the effect of the…
Condition: Pediatrics / Cancer
Investigator: Luca Szalontay, MD
Status: Closed
This is a phase I, multicenter, dose escalation study of MK-1775 in combination with radiation. The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children and young adults with newly diagnosed Diffuse Intrinsic Pontine Glioma (DIPG). MK-1775 is an oral…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of the study is to see if a medicine called ASP2215 is both effective and safe as atreatment for AML patients with mutations in FLT3 gene identified by an investigational FLT3Mutation Assay companion diagnostic (a specific way to test your blood or bone marrow, which is used to see if you have a mutation of the FLT3 gene), compared to salvage…
Condition: Cancer / Pediatric Solid Tumors
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to learn what kind of side effects nivolumab as a single agent and nivolumab in combination with ipilimumab can cause and if these treatments are beneficial for solid tumors. Nivolumab (Dose Level 1) will be given as an intravenous infusion every 14 days of a 28 day cycle. If Dose Level 1 is not tolerable, then it will be…
Condition: Cancer / Skin Cancer
Investigator: Sewanti Limaye, MD
Status: Closed
The purpose of this study is to examine the effects of KPT-330 on patients with an advanced form of skin cancer called Squamous Cell Cancer (SCC) of the head and neck, lung, or esophagus. The study drug KPT-330 is an investigational drug that
Condition: Cancer / Pediatric Solid Tumors
Investigator: Stergios Zacharoulis, MD
Status: Closed
The purpose of this study is to find out more about how the study drug called entrectinib will be used in children, adolescents and young adults with treatment-resistant solid tumor in brain or body. This is a phase 1/1b study, divided into a dose escalation portion (different dose will be tested) and a dose expansion portion (one dose will be tested) in…
Condition: Cancer / Leukemia
Investigator: Daniel Lee, MD
Status: Closed
The purpose of this research study is to determine the effectiveness (how well something works) and safety of an investigational compound called alvocidib when used in a combination chemotherapy therapy regimen in patients with acute myeloid leukemia (AML) who test positive for NOXA priming.
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
The main goal of this study is to find out about how duvelisib acts in patients with CLL/SLL who were previously treated with a BTKi (Bruton Tyrosine Kinase inhibitor - commercially available ibrutinib or another investigational BTKi). Duvelisib is an investigational drug, meaning that it is not FDA approved, designed to try to stop cancer growth by…
Condition: Pediatrics
Investigator: Meyer Kattan, MD
Status: Closed
The Breathe Easy Early ORBEX Study is a three -year study to identify if a daily capsule of Broncho-Vaxom may help prevent or delay young children from developing wheezing or asthma- like symptoms. Other studies have found that Broncho-Vaxom can help prevent upper respiratory infections and stimulate immune defenses, and has been safely and effectively used…
Condition: Skin Cancer / Melanoma
Investigator: Richard Carvajal, MD
Status: Closed
This is a phase II research study for individuals with resectable, stage IIIB to IVM1a Melanoma. The research drug being studied is talimogene laherparepvec. Talimogene laherparepvec is still experimental and has not been approved by the Food and Drug Administration (FDA) for use in people with melanoma. The purpose of this study is to find out more about…
Condition: Pediatrics
Investigator: Leonardo Liberman, MD
Status: Closed
The purpose of this study is to investigate how mothers with babies with congenital heart disease feel about breastfeeding. We will ask you to fill out a survey while you are in the hospital at around the time of delivery and another survey approximately three months later to see how you feel about breastfeeding. In addition to the survey data, the study…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to see if a medicine called ASP2215 is both safe and effective as atreatment for patients with Acute Myeloid Leukemia (AML). ASP2215 is an experimental drug that is being studied to treat AML. In this study, ASP2215 will be given in addition to a standard chemotherapy regimen that patients could be receiving even if they were…
Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Claudia Chiriboga, MD, MPH
Status: Closed
The purpose of this study is to evaluate the safety, tolerability and effectiveness of a study drug called ISIS-396443 when using it to treat children with later-onset spinal muscular atrophy (SMA). SMA is a neuromuscular disease that causes a degeneration (a breakdown) of the motor neurons in the spinal cord, resulting in a weakening of the muscles in…
Condition: Cancer / Leukemia
Investigator: Daniel Lee, MD
Status: Closed
The purpose of this research study is to determine the preliminary safety and effectiveness (how well something works) of alvocidib when used after the standard drug decitabine in patients with MDS. The study will also gain information about the PK (the process by which a drug is absorbed, distributed, metabolized and eventually eliminated by the body),…
Condition: Pediatrics
Investigator: Meyer Kattan, MD
Status: Closed
Research funded by the NIH is being done to learn if an asthma medication, given along with standard asthma care, makes children less likely to have asthma attacks.
Condition: Skin Cancer / Melanoma
Investigator: Richard Carvajal, MD
Status: Closed
The purpose of this study is to find out which participants with melanoma respond best to nivolumab and ipilimumab treatment and to identify tumor and blood based markers that may predict response to the combination. This study is asking that participants submit tumor tissue samples from their initial tumor specimen and blood samples before they receive…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The main purpose of this study is to see how effective an investigational new drug (tipifarnib, the study drug) is to treat myelodysplastic syndrome (MDS). "Investigational" means that tipifarnib has not yet been approved by regulatory authorities such as the United States Food and Drug Administration (FDA) for use outside of clinical trials. You…
Condition: Pediatrics
Investigator: Ilene Fennoy, MD
Status: Closed
Your child has been asked to participate in this study because he/she may have or has growth hormone deficiency (i.e. not enough growth hormone in his/her body) and your doctor has already determined that your child may be suitable to be screened for this study. OPKO Biologics Ltd., the Sponsor of this clinical study, has developed an…
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
The purpose of the study is to see if a combination of idelalisib and BI 836826 can help control patients with Chronic Lymphocytic Leukemia (CLL) without serious side effects.
Condition: Neuromuscular / Spinal Muscular Atrophy
Investigator: Darryl De Vivo, MD
Status: Closed
This study is for infants that have been genetically diagnosed with Spinal Muscular Atrophy (SMA) and are currently not showing any signs or symptoms of the disease. The purpose of this study is to see whether ISIS 396443 has any effects (good or bad) on infants with SMA (who do not yet show signs or symptoms of the disease) and to see if ISIS 396443 can…
Condition: Skin Cancer / Melanoma
Investigator: Gary Schwartz, MD
Status: Closed
The purpose of this study is to find out if the drugs LEE011 and MEK162, administered in combination, are safe and effective in treating people who have melanoma. Specifically, this study will evaluate the effects of the combination of the two drugs, LEE011 and MEK162, in melanoma, carrying specific mutation in a gene called NRAS. The changes in the NRAS…
Condition: Cancer / Leukemia
Investigator: Daniel Lee, MD
Status: Closed
The purpose of this research study is to find out if the study drug MBG453 in combination with other drugs (decitabine or azacitidine) already commonly used to treat MDS is safe and has beneficial effects in people who have some types of MDS like you (intermediate, high, or very high risk MDS). Subjects will be administered the study treatment as indicated…
Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
This is a phase 1/2 multicenter study of AZD1775 (MK-1775) that will be given in combination with irinotecan. The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children and young adults with solid tumors which have come back or have not responded to standard…
Condition: Cancer / Leukemia
Investigator: Todd Rosenblat, MD
Status: Closed
The goal of this research study is to find the safest highest dose of SL-401 that can be given to patients with AML or BPDCN. This study will also look at how SL-401 stops or slows leukemia or BPDCN growth and how SL-401 enters and leaves the body. This study will also look at certain proteins in the blood and bone marrow and how the amounts of them might…
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
We are doing this study to find out if an investigational drug called CPI-0610 can help with myelofibrosis. An investigational drug is a drug that is not approved for sale in the United States by the Food and Drug Administration (FDA). This study has two groups (arms) one group of patients will be treated with CPI-0610 alone (monotherapy arm) and the other…
Condition: Pediatrics / Cancer
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of temsirolimus in combination with etoposide and cyclophosphamide. The study treatment is considered experimental because it is not approved by the Unites States (US) Food and Drug Administration (FDA) for treating children with acute lymphoblastic leukemia (ALL) and non-Hodgkins lymphoma (NHL) who have relapsed. The…
A study for children with refractory solid tumors, including CNS tumors using study drug ramucirumab
Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
The goals of this study are to find the highest safe dose of ramucirumab that can be given without causing severe side effects, to learn what kind of side effects ramucirumab can cause, to learn more about the effects of ramucirumab on cells and proteins in the blood and to determine whether ramucirumab is a beneficial treatment for patients with solid…
Condition: Skin Cancer / Squamous Cell Carcinoma
Investigator: Sewanti Limaye, MD
Status: Closed
This study is for adult subjects with head and neck cancer (oral cavity, oropharynx middle part of the throat, hypopharynx bottom part of the throat, or larynx part of throat between the base of the tongue and the windpipe) that has either returned after treatment (recurrent) or is newly diagnosed. If newly diagnosed, the cancer must be metastatic,…
Condition: Cancer / Leukemia
Investigator: Ran Reshef, MD
Status: Closed
The purpose of this research study is to obtain information on the safety and effectiveness of PBCAR20A to treat certain types of cancers. PBCAR20A is a new investigational treatment for certain types of cancer of the blood, such as leukemia and lymphoma. It is made from a type of blood cells known as T cells. The T cells in PBCAR20A came from healthy…
Condition: Pediatrics
Investigator: Sharon Oberfield, MD
Status: Closed
We are recruiting boys between 3-9 years old and girls between 3-8 years old to participate in a research study. Children who would like to participate must be healthy and not showing signs of puberty. The goal of the study is to understand the relationship between early body development, metabolism (sugars and fats processing), and the saliva microbiome (…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to determine if oral azacitidine (CC-486) is safe and effective as maintenance therapy in continuing the response patients had with their last acute myeloid leukemia (AML) treatment and improving the quality of that response. Currently, there is no standard treatment to maintain or improve patient response and patients are…
Condition: Cancer / Pediatric Solid Tumors
Investigator: Julia Glade Bender, MD
Status: Closed
The purpose of this study is to assess the safety and effectiveness of a study drug called axitinib on treating tumors that have returned (recurrent) or have not responded to standard therapy (refractory). Children will take axitinib twice a day for 28 days (1 cycle). Children may take study drug for up to 24 cycles (2 years).
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to test the safety and effectiveness of avapritinib (also known as BLU-285) in individuals who have been diagnosed with aggressive systemic mastocytosis (ASM), Systemic mastocytosis with associated hematologic neoplasm (SM-AHN), or mast cell Leukemia (MCL). Avapritinib is an experimental drug means that is is not yet known if…
Condition: Pediatrics / Cancer
Investigator: Alice Lee, MD
Status: Closed
This is a Phase 1 multicenter study of LY2606368, a CHK1/2 inhibitor, in pediatric patients with refractory solid tumors, including CNS tumors. The study treatment is considered experimental because LY2606368 is not approved by the United States (US) Food and Drug Administration (FDA) for treating pediatric patients with refractory solid tumors, including…
Condition: Skin Cancer / Melanoma
Investigator: Brian Marr, MD
Status: Closed
The purpose of this research study is to test the safety of Light-activated AU-011 at three different doses of the study drug and repeat dose regimens (two or three doses of highest safe dose) to find out what effects, if any, it has on research participants with a choroidal melanoma tumor. This study will have a high dose, a medium dose, and a low dose of…
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of this study is to determine how well a drug called rigosertib works on people with MDS and to study the safety of rigosertib when it is given to people with MDS. Rigosertib is currently being tested in other clinical research studies.
Condition: Pediatrics / Cancer
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to compare two different combinations of treatment plans to a standard transplant procedure in order to see whether one or both of them are better at reducing the occurrence and severity of chronic GVHD. The research portion of this study involves manipulation of your stem cell product by removing T-cells, which cause chronic…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
This is a clinical research study for subjects with Acute Myeloid Leukemia (AML) who have not received any treatment for their disease and who are over the age of 60. These patients will be treated with low-does cytarabine in combination with Lintuzumab-Ac225. Low dose cytarabine has been investigated in elderly AML patients for at least two decades and is…
Study for subjects with untreated Melanoma using trial drugs Talimogene Laherparepvec and Ipilimumab
Condition: Cancer / Skin Cancer
Investigator: Bret Taback, MD
Status: Closed
The purpose of this study is to find out more about talimogene laherparepvec (formerly known as OncoVEXGM-CSF), in people with advanced melanoma when given in combination with another drug called ipilimumab. Melanoma is a type of skin cancer. Advanced melanoma means the tumor is not removable by surgery or has spread to other parts of the body. Phase 1 of…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to test the safety and effectiveness of an experimental medication, FT-2102, in the treatment of acute myeloid leukemia or myelodysplastic syndrome. Patients will receive either FT-2102 alone or in combination with cytarabine or azacitidine, an approved drug commonly used in the treatment of AML and MDS. Subjects will remain on…
Condition: Pediatrics / Neuromuscular
Investigator: Thomas Brannagan, MD
Status: Closed
Moving forward in Charcot-Marie-Tooth disease. A clinical research study assessing the safety and effectiveness of an investigational product in people with Charcot-Marie-Tooth (CMT) disease experiencing muscle cramps is now enrolling. Participation is entirely voluntary. Contacting us does not mean that you are required to take part in this study. If you…
Condition: Pediatrics
Investigator: Tina Leone, MD
Status: Closed
Babies born prematurely frequently have difficulty breathing because of immature lung development. A substance called surfactant is usually present in mature lungs to help keep the lungs inflated normally. The treatment for breathing problems in premature infants can include giving surfactant as a medicine. When surfactant is given as a medicine a…
Condition: Skin Cancer / Squamous Cell Carcinoma
Investigator: Richard Carvajal, MD
Status: Closed
This study is for patients with melanoma, non-small cell lung cancer (NSCLC), or squamous cell cancer of the head and neck (SCCHN) that has a protein called B7-H3 on the tumor cells or blood vessels in thetumor. The purpose of this study is to test the safety, tolerability, and most effective dose ofMGA271 in combination with ipilimumab (also called Yervoy…
Condition: Cancer / Leukemia
Investigator: Azra Raza, MD
Status: Closed
The purpose of the study is to evaluate the efficacy and safety of imetelstat for the treatment of patients with myelodysplastic syndrome (MDS). All patients participating in Part 1 of this study will get 7.5 mg/kg of imetelstat as a 2-hour intravenous infusion once every 4 weeks. The dose of imetelstat may be increased, held or decreased depending on how…
Condition: Pediatrics / Neuromuscular
Investigator: Darryl De Vivo, MD
Status: Closed
Our clinic is currently participating in the Brave Trial, a clinical research study for Duchenne muscular dystrophy (DMD). This study is being conducted to learn whether a study drug can improve motor functions in boys with DMD. If your child takes part, he'll receive at no cost: study drug or placebo, study-related procedures, reimbursement for…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to evaluate the safety, and effectiveness of ABT-199 in combination with decitabine and ABT-199 in combination with azacitidine in subjects who have not been treated for their AML. In addition, substances (biomarkers) found in the blood and bone marrow that may indicate the effects or progress of the leukemia and the activity of…
Condition: Pediatrics
Investigator: Wei Shen, MD
Status: Closed
This is a study of body changes that take place in teenagers who undergo bariatric surgery as a treatment for obesity and obesity-related conditions. Changes will be measured by serial MRI andmetabolomic profiling.
Condition: Skin Cancer / Melanoma
Investigator: Richard Carvajal, MD
Status: Closed
The purpose of this study is to compare the efficacy and safety of cobimetinib plus atezolizumab with the efficacy and safety of pembrolizumab in patients with a type of skin cancer called melanoma that has spread to other parts of the body (metastatic), cannot be removed by surgery (unresectable), and have not received any prior treatment.
Condition: Cancer / Leukemia
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to compare two different combinations of treatment plans to a standard transplant procedure in order to see whether one or both of them are better at reducing the occurrence and severity of chronic GVHD. The research portion of this study involves manipulation of your stem cell product by removing T-cells, which cause chronic…
Condition: Pediatrics
Investigator: Rakesh Sahni, MD
Status: Closed
The purpose of this study is to see if the use of bubble nasal continuous positive airway pressure (NCPAP) at a pressure of 6-7 centimeters of water (cm H2O) in very low birth weight (VLBW) infants is associated with improved gas exchange without any related changes in vital signs.
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
This study is for patients with Richter's transformation (RT). The purpose of this study is to see if a new type of investigational drug called PNT2258 that may be effective in blocking the "on" signal to cancer cells, eventually causing them to die. We are trying to see if PNT2258 provides evidence of anti-tumor effect by measuring what…
Evaluating the safety and effectiveness of study drug AMG 228 in patients with Advanced Solid Tumors
Condition: Cancer / Pediatric Solid Tumors
Investigator: Yvonne Saenger, MD
Status: Closed
This study is for patients with the following cancers: advanced non-small cell lung cancer, head and neck cancer, melanoma, colon cancer, or bladder cancer. The purpose of this study is to find out more about the effects of AMG 228 in people and on their cancer. AMG 228 is an experimental drug that is being developed to stimulate the bodys immune system to…
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
You are being asked to take part in this study because you have been diagnosed with Myeloproliferative neoplasm (MPN) Associated Myelofibrosis (your disease) and have received ruxolitinib. The purpose of this study is to confirm how well this study treatment works to reduce your spleen volume, spleen size, what effect the study treatment has on you and…
Condition: Pediatrics / Cancer
Investigator: Monica Bhatia, MD
Status: Closed
The purpose of this study is to: 1) Test the safety and tolerability of the research study drug, MK-5592 posaconazole (POS), intravenously (IV) and in powder for oral suspension (PFS), 2) Evaluate the pharmacokinetics (how the study drug is absorbed and broken down in the body), of POS IV solution and POS PFS, and 3) Evaluate the taste and reaction to the…
Condition: Skin Cancer / Melanoma
Investigator: Richard Carvajal, MD
Status: Closed
The purpose of this research study is to determine if high-dose versus standard-dose regimens of encorafenib and binimetinib are safe and have positive effects in people who have melanoma brain metastasis and also have been found by their doctor to have a mutated gene called BRAFV600. The time frame for the study is approximately 2 years.
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to test the safety of DCLL9718S at different dose levels, alone or in combination with azacitidine. DCLL9718S is an experimental drug manufactured by Genentech that is designed to inhibit tumor growth. The drug is being tested in clinical trials and has not been approved by the U.S. Food and Drug Administration (FDA) and/or any…
Condition: Pediatrics
Investigator: Alexis Boneparth, MD
Status: Closed
Juvenile fibromyalgia (JFM) is a cause of chronic, whole-body pain. The cause of JFM is unknown. One of the possible causes of JFM is a problem with nerves called "small fiber neuropathy." This is a problem where small nerve fibers in the skin are not working properly and patients can have pain as a result. Recent studies have found evidence of…
Condition: Cancer / Childhood Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in children with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) that has either come back or has not responded to the standard therapy. Pevonedistat works by blocking some of the enzymes that are needed for cell growth.
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of this study is to find the dose of imetelstat that best treats myelofibrosis. (A doseis a measured amount of a drug taken at one time.) The effects of imetelstat on myelofibrosis as well as any side effects will be explored.
Condition: Pediatrics / Cancer
Investigator: Alice Lee, MD
Status: Closed
The purpose of this research study is to collect information about the percentage of people that develop HACA when they are treated with Unituxin and determine if HACA affects the blood levels of Unituxin. Unituxin is a type of antibody. Antibodies are made by the body to attack tumors and fight infections. Unituxin is a chimeric antibody which means it is…
Condition: Skin Cancer / Squamous Cell Carcinoma
Investigator: Sewanti Limaye, MD
Status: Closed
This study is for patients with recurrent or metastatic Squamous Cell Carcinoma of the Head and Neck (SCCHN). This study will test the safety of an experimental anti-cancer drug (MEDI4736). MEDI4736 is an antibody (a protein produced by the defense system of the body immune system) that has been made in the laboratory. MEDI4736 boosts the ability of your…
Condition: Pediatrics / Cardiology
Investigator: Marc Richmond, MD
Status: Closed
Over time, children and young adults who have had a Fontan operation may find they are more tired, have difficulty breathing and cannot exercise as well as before. Currently, there are no approved medicines to prevent this decline. The purpose of this research study is to learn whether children who have had the Fontan operation can improve their ability to…
Condition: Cancer / Leukemia
Investigator: Daniel Lee, MD
Status: Closed
You are being asked to take part in this study because you have acute myeloid leukemia (AML) that has come back after initial treatment (relapsed) with venetoclax combination therapy or because you did not get a complete response from your initial treatment (refractory) with venetoclax combination therapy. The purpose of this research study is to determine…
Condition: Pediatrics
Investigator: Wendy Chung, MD, PhD
Status: Closed
The purpose of this research is to determine whether a new investigational drug called TAK-935 is safe and whether it may help treat epilepsy in children with 15q duplication syndrome or CDKL5 deficiency disorder.
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
The purpose of this study is to evaluate how well the study drug ABT-199 works and safety of ABT-199 in subjects with Chronic Lymphocytic Leukemia (CLL) whose cancer has come back (relapsed) after treatment or no response (refractory) to treatment with B-cell receptor signaling pathway inhibitor therapy, namely Ibrutinib and/or Idelalisib. In addition,…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to test the safety of the combination of the experimental drugs called atezolizumab and guadecitabine (not yet approved by the U.S. Food and Drug Administration FDA or any other regulatory authority outside the United States) to find out what effects, good or bad, the combination of atezolizumab and guadecitabine has on patients…
Determining Changing in Cardiovascular and Metabolic Risks in Patients with Chronic Myeloid Leukemia
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
This study will see how the treatment you will receive for your chronic myeloid leukemia affects cardiovascular and metabolic risk factors. The treatments that are being studied are called BCR-ABL1 tyrosine kinase inhibitors (TKIs) (i.e., dasatinib, imatinib, nilotinib). Subjects who are eligible for the study will be starting treatment for chronic myeloid…
Condition: Pediatrics
Investigator: Steven Lobritto, MD
Status: Closed
Open-labeled hepatitis C pediatric treatment trial with a two drug direct acting viral oral combination (already approved in adults) across all genotypes for naive or treatment experienced subjects.
Condition: Pediatrics
Investigator: Joel Lavine, MD
Status: Closed
Researchers at Columbia University are conducting a clinical research study of children with inammatory bowel disease (IBD). This study will examine the eectiveness and safety of an investigational medication inchildren with IBD.
Condition: Cancer / Leukemia
Investigator: Daniel Lee, MD
Status: Closed
The purpose of this study is to evaluate the efficacy (how well the study drug works) and safety of venetoclax in combination with azacitidine in subjects who have not been treated for their acute myeloid leukemia (AML). The tolerability (the degree to which evident adverse effects can be tolerated by the patient) of venetoclax will also be evaluated.
Condition: Pediatrics
Investigator: Diane George, MD
Status: Closed
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a non-malignant condition.
Condition: Pediatrics / Cardiology
Investigator: Irene Lytrivi, MD
Status: Closed
This is a Phase 3 multi-center open label randomized clinical trial. The purpose of this study is to learnmore about which anti-rejection (or immunosuppressive) medications best protect infants, children,adolescents, and young adults after they have had a heart transplant. Two different drug regimens will bewill be compared. All of these drugs are FDA-…
Condition: Cancer / Leukemia
Investigator: Todd Rosenblat, MD
Status: Closed
The purpose of the study is to test how effective and safe the study drug SGI-110 is compared with current standard treatment for AML. The study drug, SGI-110, is changed in the body into a compound called decitabine. Decitabine is an approved drug in the US for treatment of patients with myelodysplastic syndrome (MDS) and in Europe for treatment of…
Condition: Pediatrics / Neuromuscular
Investigator: Jacinda Sampson, MD
Status: Closed
The purpose of this study is to find out if eteplirsen can help people with the muscle disorder called Duchenne Muscular Dystrophy (DMD) and find out if eteplirsen is safe to take without causing too many side effects. Eteplirsen is an investigational (experimental) drug and can only be used in research studies. DMD is caused by a mutation (a change) in the…
Condition: Cancer / Leukemia
Investigator: Daniel Lee, MD
Status: Closed
The main purposes of this study are to: 1) understand the safety of atezolizumab given in combination with Hu5F9-G4 and 2) understand the way the body processes atezolizumab and Hu5F9-G4 (pharmacokinetics). This study will also be testing if the combination therapy is effective at treating relapsed or refractory acute myeloid leukemia (R/R AML).…
Condition: Cancer / Leukemia
Investigator: Daniel Lee, MD
Status: Closed
The purpose of this study is to measure the effectiveness and safety of an investigational drug called "alovicidib" in combination with cytarabine and daunorubicin in the treatment of AML. Investigational means that this drug has not been approved by the FDA. Cytarabine and daunorubicin are both standard of care treatments for patients with AML.…
Condition: Cancer / Pediatric Solid Tumors
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of pevonedistat in combination with Temozolomide and Irinotecan in pediatric patients with refractory solid tumors, including CNS tumors and lymphoma. The study treatment is considered experimental because pevonedistat is not approved by the United States (US) Food and Drug Administration (FDA) for treating pediatric…
Condition: Cancer / Childhood Leukemia
Investigator: Nobuko Hijiya, MD
Status: Closed
This is a Phase 1 multicenter study of Decitabine (DEC) and Vorinostat (VOR) followed by the standard chemotherapy drugs (Fludarabine, Cytarabine and G-CSF (FLAG)). The study treatment is considered experimental because it is not approved by the United States (US) Food and Drug Administration (FDA) for treating children with acute myeloid leukemia (AML)…
Condition: Cancer / Leukemia
Investigator: Richard Carvajal, MD
Status: Closed
This study is for patients with Acute Myeloid Leukemia (AML). The purpose of this study is to determine if decitabine plus JNJ-56022473 can be safely combined and is active for your disease. The study drugs used in this study are JNJ-56022473, an experimental drug and DACOGEN ( also known generic as decitabine), a drug already used to treat your type of…
Condition: Pediatrics / Cancer
Investigator: Alice Lee, MD
Status: Closed
This study is for children with relapsed or refractory acute myeloid leukemia (rrAML). Acute myeloid leukemia is a fast growing cancer of the blood and bone marrow. This study examines the effects of study drug Lenalidomide on children with rrAML. Lenalidomide is an anti-cancer drug that belongs to a group of drugs known as immunomodulating drugs (IMiDs), a…
Condition: Skin Cancer / Squamous Cell Carcinoma
Investigator: Sewanti Limaye, MD
Status: Closed
The purpose of this study is to determine the effectiveness and safety of study drug MEDI4736 monotherapy, tremelimumab monotherapy, and MEDI4736 and tremelimumab combination therapy in the treatment of patients with recurrent or metastatic PD-L1-negative squamous cell carcinoma of the head and neck who have progressed during or after treatment with…
Condition: Pediatrics
Investigator: Ali Mencin, MD
Status: Closed
This research study is for children age 12 to 16. In this research study, we will evaluate the safety, effectiveness and tolerability of two different doses of a bowel cleansing medication referred to as BLI-800. One group of study participants will receive the larger dose (6 oz.), which dose has been approved by the FDA for use by adults. A second group of…
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
The purpose of this study, which involves research, is to determine if giving an experimental drug called acalabrutinib in combination with venetoclax with and without obinutuzumab is safe and effective for treating people with CLL compared to a combination of bendamustine and rituximab (BR) or a combination of Fludarabine, Cyclophosphamide and Rituximab (…
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
This study is being done to see how well tolerated the investigational drug TGR-1202 is in comparison to other similar therapeutics agents in treating patients with chronic lymphocytic leukemia (CLL).
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of this study is to provide samples for biomarker testing for the different MPD-RC treatment studies. As part of the treatment study you currently participating in, this is an additional research study in which you we will ask for you to donate blood and/or bone marrow samples, two nail clippings, and buccal (cheek) swab. In order to…
Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
This study is being carried out by the Childrens Oncology Group (COG) Phase 1 Consortium. COG is an international research group that consists of more than 200 hospitals that treat children with cancer in the United States, Canada, Australia, and Switzerland. The Phase 1 Consortium is the group within COG that consists of 21 hospitals, and participation in…
Condition: Cancer / Leukemia
Investigator: Alice Lee, MD
Status: Closed
This study is for children and teens who have been diagnosed with Acute Myeloid Leukemia (AML). AML is a cancer of the bone marrow, the spongy tissue inside the large bones of the body where blood cells are made. In AML, the bone marrow makes large numbers of immature white blood cells called blasts. These blast cells crowd out the normal cells of the bone…
Condition: Pediatrics / Cancer
Investigator: Diane George, MD
Status: Closed
The purpose of this study is to determine the incidence and severity of acute GVHD following CD34+ selection in children, adolescents and young adults receiving an allogeneic peripheral blood stem cell transplant from a family member or unrelated adult donor for a malignant condition.
Condition: Cancer / Leukemia
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in young adults with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) that has either come back or has not responded to the standard therapy. Pevonedistat works by blocking some of the enzymes that are needed for cell growth.
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
This is a clinical research study for subjects with Myelofibrosis (Primary, Post-Polycythemia Vera, and Post-Essential Thrombocytopenia) and thrombocytopenia. The purpose of this study is to determine if pacritinib is better at reducing spleen size and a reduction in symptoms than the best available therapy. This is a randomized, controlled study.…
Condition: Pediatrics / Cancer
Investigator: Maria Sulis, MD
Status: Closed
The purpose of this study is to determine the effectiveness and safety of the study drug moxetumomab pasudotox in treating children with relapsed or refractory pALL or Lymphoblastic Lymphoma of B-cell origin. Moxetumomab pasudotox is made up of two parts: 1) a modified mouse antibody that attaches to a protein from the immune system, called CD22, which can…
Condition: Cancer / Leukemia
Investigator: Joseph Jurcic, MD
Status: Closed
The purpose of this study is to find if PTC299 is safe to treat people with relapsed/refractory acute myeloid leukemia (AML) as well to determine the dose of PTC299 that can be given without causing severe side effects. AML is a cancer of the blood and bone marrow (spongy tissue inside bones where blood cells are made). PTC299 is being developed to…
Condition: Skin Cancer / Melanoma
Investigator: Richard Carvajal, MD
Status: Closed
The purpose of the first part (Phase I) of the study is to test the safety of navitoclax in combination with dabrafenib and trametinibat different doses to find out what effects, if any, it has on people. There will be about 18 people treated in this part of the study. The purpose of the second part of this study (Phase II) is to compare the good and bad…
Condition: Cancer / Leukemia
Investigator: Mark Heaney, MD, PhD
Status: Closed
The purpose of this study is to compare the safety, tolerability, and effectiveness of ruxolitinib, an investigational drug, compared to anagrelide, an FDA approved drug. Investigational means that the study drug has not been approved by the FDA for treatment of ET. Patients will be assigned to one of two groups. Group A will receive ruxolitinib and placebo…
Condition: Cancer / Leukemia
Investigator: Nicole Lamanna, MD
Status: Closed
The purpose of this study, which involves research, is to determine if an experimental drug: acalabrutinib is safe and effective in the treatment of this patients with High Risk Chronic Lymphocytic Leukemia (CLL). Acalabrutinib is an investigational drug, which means that it is still being tested in people and has not been approved by government agencies in…
Condition: Pediatrics
Investigator: Ali Mencin, MD
Status: Closed
This study is evaluating an investigational medication to see if it is safe and if it works well in children andadolescents who have mild to moderate ulcerative colitis (UC) or are in remission. Ulcerative colitis (UC) is a chronic disease of the last section of the intestine, which is also known as the colon. If you have UC, the lining of your colon…
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