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Displaying 1 - 107 of 107
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: 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: 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: 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: 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: 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
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 / 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: 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: 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: Endocrine & Metabolic Disease / Cushing's Disease
Investigator: Pamela Freda, MD
Status: Closed
This study is for individuals who have Cushings disease and want to join voluntarily in this study to find out if a study drug called osilodrostat (LCI699) is safe and has beneficial effects in people who have Cushings disease. Cushings disease is a rare but debilitating disease. Cushings disease is caused by a benign tumor of the pituitary gland. The tumor…
Condition: Endocrine & Metabolic Disease / Cushing's Disease
Investigator: Amanda Tsang
Status: Closed
The purpose of this study is to determine if measuring blood levels of proteins called POMC, AgRP, and chromogrannin A (CGA) can be used to diagnose and track tumor growth and recurrence in patients that have ACTH-dependent Cushing's syndrome, non-secreting pituitary tumors, and other neuroendocrine tumors. We invite you to participate if you have: a…
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: 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: Pediatrics
Investigator: Brooke Aggarwal, EdD, MS
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: 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 / 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: 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 / 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: 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: 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: 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: 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: 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: 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: 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 / 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: 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: 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: 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 / 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 / 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: 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: 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: 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: 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: 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: 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: 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: 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: 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 / 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: 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: 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: 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: 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: 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: Endocrine & Metabolic Disease / Cushing's Disease
Investigator: Pamela Freda, MD
Status: Closed
The purpose of this study is to confirm the effectiveness and safety of LCI699 in treating patients with Cushing's disease. Patients will be treated with the invesitigational drug LCI699 at doses up to 60 mg a day, and possibly for a short period of time with placebo. A placebo is a dummy drug ("pill") with no active medicine inside.
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.
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: 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: 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: 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: 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: 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: 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: 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: 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 / 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: 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: 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 / 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: 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: 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: 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…
Condition: Pediatrics / Cancer
Investigator: Luca Szalontay, MD
Status: Closed
The purpose of this study is to find out more about the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab (also known as BMS-936558) when given alone or when combined with ipilimumab in children/ adolescents/ young adults with high grade primary central nervous system (CNS) malignancies (certain…
Condition: Pediatrics / Cancer
Investigator: Ran Reshef, MD
Status: Closed
Epstein-Barr Virus (EBV) is the virus that causes mononucleosis, also known as mono. Almost all adults have had EBV. For most people, EBV never causes a health problem. However, when their immune system is not working properly (people who have had a transplant and those who have immune disorders), EBV can cause certain diseases where cells grow abnormally.…
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 talimogene laherparepvec will be used in children/adolescents/young adults with advanced solid tumors not involving the central nervous system that are available for direct injection. This is a phase 1 study, multicenter, open-label study, that means all subject enrolled in the…
Condition: Pediatrics
Investigator: Monica Bhatia, MD
Status: Closed
The overall purpose of this research is to treat diseases that are not cancer but are improved by bone marrow transplantation with mild and safer chemotherapy as pretransplant conditioning. This treatment is chosen because it is milder and is likely to have fewer long and short term side effects unlike older chemotherapy and radiation treatments. Yet, it…
Condition: Cancer / Pediatric Solid Tumors
Investigator: Julia Glade Bender, MD
Status: Closed
The purpose of this study is to examine the effectiveness and tolerability of a study drug called PF-02341066. Additional goals of this study are to learn how the body handles the study drug as well as determine whether or not PF-02341066 is a beneficial treatment for your tumor This study is for children with a recurrent or progressive tumor or lymphoma…
Condition: Cancer / Pediatric Solid Tumors
Investigator: Alice Lee, MD
Status: Closed
This is a Phase 1 multicenter study of a drug called ABI-009 (nab-Rapamycin) in combination withtemozolomide and irinotecan in children and young adults with solid tumors (including CNS tumors)which have come back or have not responded to standard therapy. The study is considered experimental because ABI-009 (nab-Rapamycin) is not approved by the United…
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