Clinical Trials

  • Principal Investigator:

    Erika S Berman Rosenzweig, MD
    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 the growth and/or function of the arteries that supply blood to the lungs. This is a growing problem in the world today. Despite the serious nature of this disorder, the medical field does not yet know the best first treatment for infants,...
  • Principal Investigator:

    Erika S Berman Rosenzweig, MD
    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 the growth and/or function of the arteries that supply blood to the lungs. This is a growing problem in the world today. Despite the serious nature of this disorder, the medical field does not yet know the best first treatment for infants,...
  • Principal Investigator:

    Steven J. Lobritto, MD
    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 the patient population. Vaccine-preventable illness continues to have resurgence in the United States and globally. Specifically, mumps and measles outbreaks have occurred with increasing frequency and distribution in the past several...
  • Principal Investigator:

    Steven J. Lobritto, MD
    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 the patient population. Vaccine-preventable illness continues to have resurgence in the United States and globally. Specifically, mumps and measles outbreaks have occurred with increasing frequency and distribution in the past several...
  • Principal Investigator:

    Nobuko Hijiya, MD
    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 uproleselan works when given to children and young adults with relapsed or refractory leukemia. Uproleselan is experimental because it has not been proven to work in this type of situation. We are using uproleselan because it seems to work...
  • 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:

    Ilene Fennoy, MD
    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 treatment affects the physical and clinical features of overweight or obesity. To achieve this aim we want to formalize our ongoing clinical chart review into a database for our patients.
  • Principal Investigator:

    Steven J. Lobritto, MD
    The purpose of this study is to find out if a new laboratory blood test for patients with Wilson's disease can accurately and reliably measure so-called free copper, also known as non-ceruloplasmin copper (NCC). Participation will last about 12 months. If you have any questions regarding this study, please contact the Transplant Clinical Research Center (TCRC).
  • Principal Investigator:

    Melissa S. Stockwell, MD, MPH
    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:

    Steven J. Lobritto, MD
    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 liver transplants. If you have any questions regarding this study, please contact the Transplant Clinical Research Center (TCRC).

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