Clinical Trials

  • Genetic Study of Obesity

    Principal Investigator

    Wendy K. Chung, MD, PhD
    The goal of this study is to better understand the genetic causes of weight gain and identify individuals who may benefit from a new treatment to help them maintain a healthy weight. Your participation will help us understand how why some people gain more weight and how individuals can stay at a healthy weight. We will ask you questions about your weight and your height to determine if you are eligible. If you are eligible, we will invite you to participate.

  • ICON3: A Phase 3 Study of Eltrombopag vs. Standard Therapy for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children

    Principal Investigator

    Cindy Neunert, MD
    The purpose of this study is 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.

  • MLN4924 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome (1 month to 17 years old)

    Principal Investigator

    Alice Lee, MD
    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.

  • MLN4924 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome (18 to 21 years old)

    Principal Investigator

    Alice Lee, MD
    The purpose of this study is to find the best dose of pevonedistat that can be given safely with chemotherapy in young adults 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.

  • Off the shelf T-cells therapy for viral infection post HCST

    In this trial, we will evaluate whether partially-HLA matched allogeneic multivirus-specific VSTs, will be safe and produce anti-viral effects in immunodeficient recipients infected with one of more of the targeted viruses that are persistent despite conventional anti-viral therapy. This will be in patients who have received a hematopoietic stem cell transplant or patients with primary immunodeficiency conditions who have persistent viral infections and have not undergone HSCT.

  • Palbociclib and Chemotherapy in Treating Children With Relapsed Acute Lymphoblastic Leukemia (ALL) or Lymphoblastic Lymphoma (LL)

    The purpose of this study is to find the best dose of Palbociclib that can be given safely with chemotherapy in children and young adults with Acute Lymphoblastic Leukemia (ALL) or Lymphoblastic Lymphoma (LL) which has come back or has not responded to standard therapy. Palbociclib is considered experimental because it has not been approved by the United States (US) Food and Drug Administration (FDA) for treating children with Acute Lymphoblastic Leukemia (ALL) and Lymphoblastic Lymphoma (LL). Palbociclib works by targeting proteins that are necessary for cell growth.

  • Program for Overweight Education and Reduction (POWER)

    Principal Investigator

    Ilene Fennoy, MD
    We are doing this research study to find out about 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.

  • PVDOMICS: Defining the Future Fingerprints of Pulmonary Vascular Disease

    This research study is being conducted to study pulmonary hypertension and right heart dysfunction based on clinical, imaging, physiological and pathological characteristics. We hope to develop a new classification system and new methods to diagnose and treat pulmonary hypertension. Diagnostic testing includes (listed subsequently): full medical work-up, minimal blood collection, right heart catheterization, echocardiogram, ekg, take home sleep oximetry study, six-minute walk, exercise test, Pulmonary function test, Lung CT, Questionnaires, ventilation/perfusion scan and cardiac MRI.

  • Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients (6 months-17 years)

    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 refractory (did not respond to standard treatment) solid tumor or lymphoma.

  • Safety, Tolerability, Efficacy and Pharmacokinetics of Copanlisib in Pediatric Patients (ages 18-21)

    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 refractory (did not respond to standard treatment) solid tumor or lymphoma.

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