Clinical Trials

  • 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.
  • Principal Investigator

    Stergios Zacharoulis, MD
    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 study will be assigned talimogene laherparepvec. The study drug is a genetically modified virus. The virus' genes were modified in a laboratory to make the virus active in the tumor cell but not in normal cell.
  • Principal Investigator

    Alice Lee, MD
    This study will enroll children from 6 months to 17 years old that have solid tumor cancers or lymphoma (cancer of the cells in the lymph nodes, part of the body's immune system). The purpose of this study is to test the safety, tolerability and anti-tumor activity of the research study drug, pembrolizumab (MK-3475) as well as look for the highest dose of pembrolizumab that can be given safely without serious side effects. This study will also attempt to find out how pembrolizumab is absorbed and broken down and determine how treatment with pembrolizumab may benefit children with cancer.
  • 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 decreased and a similar number of patients will be evaluated for tolerability at this dose.
  • The purpose of this study is to find out whether a new drug, called apixiban, is safe to use in children, and whether it is helpful in children with blood clots. If you choose to have your child be part of this study, s/he will either receive the experimental drug, or he/she will receive one of the usual medications that we use for children with blood clots. Neither you nor your child's doctor will choose whether your child receives the usual medications or the experimental medication.
  • Principal Investigator

    Monica Bhatia, MD
    This study is being done to learn more about the safety and effects of CTX001 in patients with severe SCD. CTX001 is created by changing (editing) the DNA in your own blood stem cells near a gene called BCL11A which is stored in a solution called dimethyl sulfoxide (DMSO) to try to prevent cells from dying while they are frozen. BCL11A is the area in the gene that stops the blood cell from making Hemoglobin F, the normal type of hemoglobin that can carry oxygen. Editing this genemay stop it from working, which may lead to your blood cell to produce more Hemoglobin F.
  • Polycystic ovary syndrome (PCOS) is a common disorder. Young women with PCOS have irregular or absence of menstruation and excessive acne and or hair on their bodies. They may have multiple cysts on their ovaries. Adolescents and women affected with PCOS are at risk of developing abnormalities in their blood including high fat, low HDL cholesterol (good cholesterol), high blood pressure and prediabetes or diabetes; they also have increased fat in the abdominal area. This cluster of abnormalities is called the metabolic syndrome.
  • Principal Investigator

    Diane M. George, MD
    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 reason for this nationwide study is to help us learn if treatment with German cockroach extract, given as allergy shots, given along with standard asthma care makes children less likely to have asthma symptoms. The study will take up to 36 months. Study visits will include questionnaires, a physical examination, an allergy skin test, breathing tests, and blood, nasal collections, and allergy shots. You will be reimbursed for each completed study visit and your child will receive a gift.
  • Several children are born with birth defects every day. Through extensive genetic research, we have learned over the years that there are genetic factors that can contribute to the occurrence of some of these defects, especially in children with more than one congenital defect. However, the underlying genetic causes of many birth defects still remain unknown. The goal of this study is to use a sophisticated technology called whole exome/genome sequencing to identify underlying genetic causes of surgical birth defects.