Clinical Trials

  • Principal Investigator:

    Nobuko Hijiya, MD
    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-asparaginase.The study consist of 3 periods: screening, treatment and end of study period.Participants with stable disease or better response at the end of the Induction Cycle will be offered an optional cycle of Consolidation chemotherapy.
  • Principal Investigator:

    Luca Szalontay, MD
    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 microsatellite-instability-high (MSI-H) solid tumor. Participant might be at least 6 months ( 3 years rrcHL) to less than 18 years old and able to provide the sample for testing the PD-L1 (programmed cell death ligand 1) purpose from an...
  • Principal Investigator:

    Wendy Kay Chung, MD
    Study examining the genetic causes of birth defects.
  • Principal Investigator:

    Wendy Kay Chung, MD
    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. DHREAMS (Diaphragmatic Hernia Research & Exploration; Advancing Molecular Science) study was created to improve the understanding of the molecular genetic basis of CDH. This National Institute of Health (NIH)-funded research team is composed of health care providers and researchers across the country, coordinated at Columbia University Medical Center. Multiple medical centers are enrolling individuals and families...
  • Principal Investigator:

    Vidhu Thaker, MD
    We will enroll children with severe early-onset obesity (BMI - 99th percentile) diagnosed prior to 6 years of age. 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 genetic study.
  • Principal Investigator:

    Brett Romeo Anderson, MD
    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 children with DS. In this research study, we want to learn whether having CHD and infant heart surgery affects the way children with DS develop and learn. We would like to compare children with DS who have had CHD and infant heart surgery...
  • Principal Investigator:

    Monica Bhatia, MD
    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.
  • Principal Investigator:

    Melissa S. Stockwell, MD
    We are doing this study to understand how COVID, the infection caused by a virus called SARS-CoV-2 affects the body, and why some people who got COVID are still sick many months after being infected. Help us to better understand the long-term effects of COVID. If you or your child has had COVID or is feeling the long-term effects of COVID, you might be able to help us understand more about it and treat it. Even if you or your child has never had COVID, you might be able to help. We are seeking volunteers who are under 25 to join the NIH RECOVER initiative. Once someone is enrolled, they will...
  • Principal Investigator:

    Melissa S. Stockwell, MD
    The purpose of this study is to assess the effectiveness of influenza and SARS-CoV-2 vaccines for preventing transmission of infections in households. We are seeking volunteers who have been recently diagnosed with COVID-19 or Influenza virus infection by either rapid diagnostic assay or RT-PCR in the last 7 days and live in a household with at least one other person. The study, also known as RVTN Sentinel Respiratory VirusTransmission Network will be looking at household transmission of SARS-CoV-2 and/or influenza. Once someone and their household contacts are enrolled, they will be followed...
  • Principal Investigator:

    Melissa S. Stockwell, MD
    The purpose of this study is to assess the effectiveness of influenza and SARS-CoV-2 vaccines for preventing transmission of infections in households. We are seeking volunteers who have been recently diagnosed with COVID-19 or Influenza virus infection by either rapid diagnostic assay or RT-PCR in the last 7 days and live in a household with at least one other person. The study, also known as RVTN Sentinel Respiratory VirusTransmission Network will be looking at household transmission of SARS-CoV-2 and/or influenza. Once someone and their household contacts are enrolled, they will be followed...