Clinical Trials

  • Principal Investigator:

    Rudolph L. Leibel, MD
    The Physiology of the Weight Reduced State (POWERS) is a national research study designed to discover and learn what happens to the human body after weight loss and to learn why some people regain their lost weight. Understanding why this happens could help health professionals improve strategies for successful weight loss maintenance after weight loss. POWERS is a research study with a goal to understand the changes in the body that occur after weight loss and learn how these changes affect the ability to keep the weight off. It involves universities, medical centers, researchers, and most...
  • Principal Investigator:

    Mercedes Martinez, MD
    This study is evaluating how well odevixibat, an approved medication for Alagille Syndrome (ALGS), works in real-world clinical practice and how safe it is for long-term use. Participants with ALGS who are receiving odevixibat as part of their usual medical care will be followed for about five years. During this time, information from routine clinic visits will be collected to understand how treatment affects liver health, growth, and development, and to identify any side effects. This research may help improve care and future treatment options for people living with ALGS. If you have any...
  • Principal Investigator:

    Melissa S. Stockwell, MD, MPH
    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 fill out a survey for us and take a home blood test. Then about half of people will be asked if they want to continue in the study to take some more surveys and come to the medical center for some...
  • Principal Investigator:

    Steven J. Lobritto, MD
    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-thymocyte globulin therapy improves survival for children with this rare, life-threatening condition, as compared to supportive care. Participants will be enrolled while they are hospitalized for acute liver failure, and will continue to be...
  • Principal Investigator:

    Steven J. Lobritto, MD
    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-thymocyte globulin therapy improves survival for children with this rare, life-threatening condition, as compared to supportive care. Participants will be enrolled while they are hospitalized for acute liver failure, and will continue to be...
  • Principal Investigator:

    Nobuko Hijiya, MD
    This study will enroll with children, adolescents and young adults that are less than 22 years old that have returned or 'relapsed' acute myeloid leukemia (AML) diagnoses. Within this group, the study is also targeting those are also unable to receive additional chemotherapy treatments containing anthracycline, which is a type of antibiotic that is used to treat many types of cancer and can cause damage to the heart in some children and young adults. The study doctors want to find out how safe the new study drug called venetoclax is when given with three other chemotherapy drugs called...

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