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.
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…
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, please contact us to determine eligibility.
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…
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…
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.
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…
We will enroll children with severe early onset obesity (BMI greater than 99th percentile) diagnosed prior to 6 years 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…
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-…
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…
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, all children will be offered CBT, delivered by experts at no cost. This study will take place at…
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).
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…
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…
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…
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…
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…
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…
The purpose of this study is to understand more about the combination of amivantamab and lazertinib in non-small cell lung cancer (NSCLC) who have a specific mutation in the EGFR (epidermal growth factor receptor) gene and have not had prior treatment for their disease. The Food and Drug Administration (FDA) has not approved the combination of amivantamab…
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.
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…
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-…
The purpose of this study is to evaluate the safety and effectiveness of an experimental drug, BDTX1535, which is not yet approved by the United States Food and Drug Administration (FDA), intreating patients with advanced solid tumors with changes in a protein named epidermal growth factor receptor (EGFR), which may or may not help your condition during…
The purpose this study to learn more about if a medication called durvalumab will work and be safe for the treatment of non-small cell lung cancer (NSCLC) that has spread to other parts of the body (metastatic) with certain genetic changes when given in combination with tremelimumab and standard chemotherapy. Additionally, this study is being done to better…
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…
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.
This study will compare the effectiveness of osimertinib alone to the combination of osimertinib and chemotherapy (carboplatin and pemetrexed) in people with metastatic lung cancer that has a change (mutation) in the gene for the epidermal growth factor receptor (EGFR). Osimertinib alone is the usual treatment for metastatic EGFR-mutant lung cancer. This…
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.
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.
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.
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…
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…
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.
We are conducting a survey to gather feedback about lung cancer patients experience with treatment and the challenges you faced. Your insights can help shape better care for future patients and explore how technology can be of help! The survey takes 30-40 minutes, and eligible participants who complete it will receive a gift card. Interested in…
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…
This is a research study evaluating a new investigational drug, called CLN-081. CLN-081 is a type of drug called a tyrosine kinase inhibitor. It targets a mutation in the tumor cell called EGFR exon 20 insertion with the goal to slow down or stop cancer cell growth. It is taken orally (by mouth). There are several different parts to this study, each with…
The purpose of this study to learn more about whether the study drug osimertinib (TAGRISSOTM) is more effective than placebo in participants with epidermal growth factor receptor (EGFR) mutated stage IA2 or IA3 Non-Small Cell Lung Cancer (NSCLC) after complete surgical removal of the tumor. Stages IA2 and IA3 refer to tumor size. A placebo is an inactive…
The purpose of this study is to assess the benefits and risks of the study drug, adagrasib, when given either by itself or with another drug, nivolumab, to participants with non-small cell lung cancer and a specific cancer mutation, called KRAS G12C, and whether the study drug or the combination may treat or shrink tumors when given before cancer surgery.…
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.
The purpose of this study is to compare the drugs ramucirumab and pembrolizumab to standard-of-care treatment in patients whose lung cancer has progressed on immunotherapy. The study doctors hope to learn if the drug combination will help patients with stage IV or recurrent non-small cell lung cancer (NSCLC) live longer. The study is set up to find out if…
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.
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…
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…
We are doing this study because we want to find out if MRI brain scans alone is better or worse than MRI brain scans plus preventative brain radiation for patients with small lung cancer. The usual approach for patients with small cell lung cancer is prophylactic cranial irradiation. If you decide to take part in this study, you will either receive PCI for…
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…
The purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem cells, modifies them by CRISPR technology, and infuses them back to the…
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…
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.
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…
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…
The purpose of this study is to see whether the medication osimertinib (TAGRISSOTM) alone or when used in combination with chemotherapy is more effective than chemotherapy alone for the treatment of early stage Non-Small Cell Lung Cancer prior to surgery. Osimertinib is an EGFR TKI therapy that targets EGFR mutations. When a tumor is found to have this type…
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-…
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.
The purpose of this study is to compare the usual treatment for Non-Small Cell Lung Cancer (osimertinib) to a combination therapy (osimertinib plus ramucirumab). The combination therapy arm is testing if ramucirumab can help osimertinib control the abnormal EGRG protein for a longer time and keep the disease inactive longer. EGRF protein typically helps the…
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…
The purpose of this study is to assess the effects, good or bad, of atezolizumab and tiragolumab given with or without chemotherapy (in this study, chemotherapy will be a combination of either paclitaxel with carboplatin, pemetrexed with carboplatin or cisplatin, or gemcitabine with carboplatin or cisplatin) on patients who are able to have surgery to…
The purpose of the study is to see whether tomivosertib in combination with pembrolizumab treatment slows down the growth of cancers. The study drug, tomivosertib, works by inhibiting the activity of proteins in a pathway that allows tumor cells to grow and go unnoticed by your immune system. This study will evaluate the safety and antitumor activity of…
The purpose of this study is to gain a better understanding of why some patients with non-small cell lung cancer and an epidermal growth factor receptor (EGFR-sensitizing mutation, fail first-line treatment with the drug osermitinib. Additionally, the purpose is to explore different drug therapies for patients whose cancer has progressed (gotten worse)…
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.
The purpose of this study is to learn about whether the study drug (PF-07209326) is safe and effective in people with sickle cell disease and learn about what the right dose is. We will look at drug levels in the blood, see how fast drug levels rise and fall, and what effects the drug has, all of which are known as the pharmacokinetics (PK) and…
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.
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…
The purpose of this study is to see if amivantamab in combination with administration of standard of care chemotherapy (carboplatin and pemetrexed) is safe and useful for treating patients with Non-Small Cell Lung Cancer (NSCLC), specifically in patients with a gene abnormality called EGFR Exon 20 insertions. There are two study arms for this trial.…
The purpose of this study is to see if the study drug, JNJ-61186372, is safe and useful for treating patients with advanced, metastatic, non-small cell lung cancer (NSCLC). There are 2 parts of this study. The first part is to determine the dose of JNJ-61186372 that is safe to give to patients with non-small cell lung cancer. The second part of the study is…
This study is aimed at finding patients who have small amounts of cancer DNA in their blood (called circulating tumor DNA or ctDNA) after surgery. This may indicate that you have minimal residual disease (MRD), meaning that even after surgery, there are still some remaining cancer cells releasing cancer DNA into your bloodstream. MRD after surgery may mean…
The purpose of this research study is to find out if a drug called capmatinib is safe and effective (can help people) in people who have non-small cell lung cancer (NSCLC) who are also eligible for surgery. Capmatinib is an oral drug that targets particular processes, which may not be working properly in the cancer cells (dysregulation) in the body. Prior…
The purpose of this study is to see if lazertinib and JNJ-61186372 are safe and useful for treating patients with a type of lung cancer called advanced non-small cell lung cancer (NSCLC) as well as looking at the effects that lazertinib and JNJ-61186372 have when taken together. Lazertinib is taken orally and JNJ-61186372 is an intravenous drug. Lazertinib…
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…
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…
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…
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…
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…
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…
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…
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…
The purpose of this research study is to see how effective durvalumab andtremelimumab is in patients with early stage non-small cell lung cancer (NSCLC). A a special blood test will be done to see if subjects have circulating tumor DNA (ctDNA) in their blood. ctDNA are pieces of genetic material shed from the tumor cells that can be detected in the blood,…
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…
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…
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…
The purpose of this study is to determine the doses of RO7121661 that can be administered safely to patients and what the side effects of RO7121661 are. In addition, how RO7121661 affects your body and, in particular, your tumor will be measured. The study also aims to investigate if RO7121661 can slow down or stop the growth of tumors in patients with…
The purpose of this master study is to test the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab (also known as BMS-936558) alone or in combination with standard of care therapies.
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…
The purpose of this study is to test the safety of the study drugs and how well your body tolerates it. The study will also test how well the study drugs work to slow down the growth of you tumor or stop your cancer. The study drugs are Cergutuzumab Amunaleukin and Atezolizumab. Atezolizumab is FDA approved for the treatment of patients with metastatic non-…
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.…
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…
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…
The purpose of this study is to compare the effects of using crizotinib (also known as Xalkori) after completion of surgery, and in some cases, after chemotherapy and/or radiation therapy for ALK-positive non-small cell lung cancer patients versus placebo. Participants will be randomized to crizotinib or placebo. Participants will take crizotinib/placebo…
The purpose of this study is to see if study drug AZD9291 is effective in treating non-small cell lung cancer (NSCLC) and if so, how it compares with chemotherapy. Only patients with NSCLC that has been confirmed as having the EGFR mutation are eligible to participate. Before patients can participate in the main study a tumor sample will be taken to check…
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…
The purpose of this study is to look at whether the combination of MEDI9447 and osimertinib or MEDI9447 and AZD4635 will improve the outcome in subjects with advanced epidermal growth factor receptor mutant (EGFRm) non-small cell lung cancer (NSCLC).
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…
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…
The purpose of this study is to assess the overall safety and efficacy (how well the drug works) of study drug M7824 in the treatment of unresectable, non-small cell lung cancer. Another purpose is to determine whether use of the study drug in combination with chemoradiation (chemotherapy plus radiation therapy) followed by the study drug will result in…
This study is for patients who are being considered for surgical removal of a specific kind of lung cancer called "non-small lung cancer" (NSCLC), which is a description of the way it looks under a microscope. The purpose of this study is to determine whether adding an investigational treatment called atezolizumab (also called MPDL3280A) to…
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…
This is an open-label, Phase II study in patients with metastatic non-small cell lung cancer (NSCLC) whose cancer has gotten worse on an anti-programmed cell death-1/anti-programmed cell death ligand 1 (anti-PD-1/PD-L1) containing therapy. This study consists of a number of treatment cohorts to test the efficacy, safety, and tolerability of multiple…
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…
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…
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…
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…
The purpose of this study is to compare the effects, good or bad, of drugs alectinib and crizotnib on patients with lung disease to find out which is better for patients with a special type of non-small cell lung cancer (NSCLC) that is called anaplastic lymphoma kinase (ALK)-positive NSCLC. In this study, participants will receive either alectinib or…
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.
This purpose of this study is to determine whether the tumor cells of lung cancer patients contain changes to the DNA sequence of two proteins found on the cells surface- ALK and EGFR. If the tumor contains changes to the DNA of one of these proteins, the patient may be invited to participate on a trial that will look at drugs which may target tumors that…
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…
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…
This research study is testing two potential new drugs called AB928 and AB122. AB928 is an investigational drug that has been shown in animal studies to improve the functioning of the immune system, without causing any immune effects on its own. This research study will help us understand whether AB928, the study drug, can be safely given in combination…
This is a research study to test the effectiveness of pembrolizumab on cancer types having a known relationship to smoking carcinogen exposure. The study will enroll 120 subjects age 18 and above with advanced solid tumor related to smoking exposure. All patients will receive the treatment of pembrolizumab. The study aims to determine if tumor genetics can…
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…
This trial will look at how the study drug will work in combination with traditional chemotherapy at treating extensive-stage small cell lung cancer in the first-line setting. The study will also determine if these drugs cause any side effects compared to traditional chemotherapy alone. The study drug is Durvalumab by itself or with Tremelimumab. Both…
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…
The purpose of this research is to learn how to help parent youth pairs work together to help youth with sickle cell disease take hydroxyurea every day as a daily habit. This study is for children and teens ages 10-18 with Sickle Cell Anemia taking hydroxyurea, and their parents (or guardians). Many children and teens do not use hydroxyurea every day. Daily…
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 (…
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…
This randomized phase III trial studies how well management of a tobacco treatment intervention works in reducing surgical complications in patients with newly diagnosed lung cancer who smoke cigarettes. Management of a tobacco treatment intervention compares varenicline (a drug that reduces the craving and withdrawal symptoms that occur with abstinence…
The purpose of this study is to find out whether the drug, ruxolitinib, is effective, tolerable and safe in combination with two chemotherapy medicines called pemetrexed and cisplatin. The study will test if this combination has beneficial effects in people diagnosed with certain types of non-small cell lung cancer. Only participants with Stage IIIB,…
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…
The purpose of this study is to obtain information on the safety and effectiveness of NovoTTF-100M (study device) in subjects with brain metastases (tumors) as a result of non-small cell lung cancer (NSCLC).
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…
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…
The purpose of this study is to test whether local radiotherapy combined with Ipilimumab is safe. The study will try to find out if it can help patients with metastatic non-small cell lung cancer better than treatment with Ipilimumab and Nivolumab alone.
The purpose of this study is to evaluate the combination of MEDI4736 and tremelimumab in treating non-small cell lung cancer. This study will look to see whether MEDI4736 combined with tremelimumab or MEDI4736 alone are more effective than standard of care chemotherapy in treating lung cancer and to collect information on the safety of these drugs. Both…
The purpose of this study is to find if adding the study drug, nivolumab (also known as OPVIDO), will limit lung cancer from growing back in patients with early stage non-small cell lung cancer. Nivolumab is a drug that may turn on the body's immune system to attack any cancer cells that may remain after surgery. The addition of nivolumab may help…
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…
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…
The purpose of this study is to determine the amount (dose) of telaglenastat that is safe and tolerable when given together with the recommended treatment regimen of pembrolizumab, carboplatin, and pemetrexed and to determine if the combination of standard drugs with telaglenastat is more active against non-small cell lung cancer (NSCLC) than the standard…
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…
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).
Research has shown that early involvement of a team of clinicians that specialize in lessening (or palliating) many of these distressing physical and emotional symptoms and in helping patients and their family cope with a serious illness improves patients and their loved ones experience with their cancer. This team is called palliative care, and consists of…
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.
This study is for adults who have non-small cell lung cancer and have had surgery to remove their lung tumor. People who have lung cancer and undergo surgery still have a significant risk of their cancer returning outside of the lung. The purpose of this study is to determine if a new type of immune-based therapy can increase your chance of being cured.…
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.
The purpose of this study is to compare any good and bad effects of avoiding the hippocampus during whole brain radiation to the usual whole-brain radiation. The hippocampus is part of the brain that is important for memory. Avoiding the hippocampus during whole-brain radiation could decrease the chance of side effects on memory and thinking. It also is…
The FLAURA Study is a new clinical research study underway in NSCLC and we are looking for people who would like to join. This study will examine an investigational drug, called AZD9291, and compare it tostandard, approved drugs that are already used to treat lung cancer. These drugs are called gefitinib and erlotinib. When we talk about investigational, it…
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…
The purpose of this study is to compare the effects of atezolizumab, an immunotherapy drug, with chemotherapy or placebo with chemotherapy in patients with resectable NSCLC to find out which is better. In this study, you will receive treatment before undergoing surgery to remove the tumor.
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…
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.
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…
The purpose is to study the safety and the effect of AB122, AB154, and AB928 in participants with stage IV non-small cell lung cancer. Researchers will study the treatment response of AB122 alone as well as in combination with AB154 and AB928. There are three possible groups a participant can be randomly assigned to. Group 1 will include AB122 alone, Group…
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…
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.
This study is being done to determine if REGN2810 (anti-PD-1) in combination with ipilimumab (anti-CTLA-4) plus initial platinum-based doublet chemotherapy, or REGN2810 in combination with ipilimumab will prolong how long you live without your disease progressing compared to the standard of care pembrolizumab (anti-PD-1) monotherapy treatment. Neither…
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.
This study will investigate the safety and effectiveness of the study drug SGI-110 in combination with two immunotherapy drugs named durvalumab and tremelimumab in patients with small cell lung cancer who have progressed on other treatments. SGI-110 is a form of chemotherapy known as a "hypomethylating agent" and may help the immunotherapy work…
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.
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-…
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…
This research study is being done to test the combination of two new drugs, GSK3359609 and Tremelimumab, in certain cancers. These two drugs are experimental which means they have not been fully tested yet and are not yet approved to treat any disease. There are two parts to this study. Part 1 determines the best dose for the study drugs and part 2 compares…
The purpose of this study is to evaluate the combination of study drug MEDI4736 and tremelimumab in treating advanced non-small cell lung cancer that is resistant to or returned after receiving standard therapies, for which there is no available standard therapies, or for individuals who have declined standard therapies. MEDI4736 and tremelimumab are fully…
The purpose of this study is to determine if the study drug is safe and evaluate whether adjuvant therapy with the study drug will improve overall survival. The study drug is crizotinib (also known as XALKORI). The study will also collect tumor tissue and blood samples for future research.
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…
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)…
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…
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…
This study is being done to see if giving your child a lower dose of chemotherapy (drug therapy), than has been used historically in bone marrow transplant, to kill his/her own diseased cells before he/she receives a bone marrow transplant, will possibly result in the permanent control of your child's sickle cell disease. Patients will receive busulfan…
The purpose of this study screening step is to perform genetic tests on your tumor sample for certain features that investigational targeted agents used in this study are specifically designed to work against. Then the purpose is to assign you to a treatment sub-study that is chosen based on the results (genes and proteins called biomarkers) of this genetic…
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…
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.
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.
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…
The purpose of this study is to look at the effects of treating Patients with Completely Resected Epidermal Growth Factor Receptor (EGFR) Mutant Non-small Cell Lung Cancer with the standard treatment or standard treatment in addition to Erlotinib. Subjects will be randomized to one of the two study groups. Subjects will be on the study for up to two years.
The purpose of this study is to test the effectiveness (how well the drug works), safety, and tolerability of an investigational drug called nivolumab on patients with advanced or metastatic non-small cell lung cancer . Nivolumab is an antibody (a type of human protein) that is being tested to see if it will allow the bodys immune system to work against…
This study is being done to test the safety and efficacy of immunotherapy based treatment combinations in patients with metastatic non-small cell lung cancer. Participants will be assigned to one of two groups, Cohort 1 or Cohort 2. Each cohort will be split into Stage 1 and Stage 2 phases.