Clinical Trials
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Principal Investigator:
Prakash Satwani, MDAre you undergoing a stem cell transplant? You may be at high risk for infections after the transplant. This study is looking at the safety and effectiveness of an investigational medication for those who have undergone a stem cell transplant. Recovery after a stem cell transplant can already be challenging without the added worry of life-threatening viral reinfections. It may be worth exploring the possibilities and peace of mind a clinical trial can offer. This is a Phase 2/3 study to evaluate the efficacy and safety of ALVR105 (also known as Viralym-M and formerly known as ALVR-105) for... -
Principal Investigator:
Monica Bhatia, MDThe purpose of this study is to find out if the study medicine called EDIT-301 will help in the treatment of Severe Sickle Cell Disease (SCD) and how safe it is to be used in people. EDIT-301 (study medicine) is a new investigational therapy which uses the patient's own stem cells, modifies them by CRISPR technology, and infuses them back to the patient to treat SCD. The CRISPR technology is a method to selectively cut and modify DNA (the genetic material in all cells). This is called genome editing. Genes can be added, removed, or interrupted. In this study, the DNA of your own blood-forming... -
Principal Investigator:
Nobuko Hijiya, MDThe purpose of this study is to find out if the drugs called Nivolumab and 5-Azacytidine can be given safely in children and young adults with Acute Myeloid Leukemia (AML) which has come back after treatment or has not responded to standard therapy. These drugs are thought to work by turning on genes that limit the growth of cancer cells. The study drugs will be given for 2 courses of chemotherapy duration based on how well the participants will tolerate the study drug and response, each course is 29-36 days long. Treatment for the 2 courses of therapy will last about 2-3 months. -
Principal Investigator:
Nobuko Hijiya, MDThe purpose of this study is to find out if the drugs called Nivolumab and 5-Azacytidine can be given safely in children and young adults with Acute Myeloid Leukemia (AML) which has come back after treatment or has not responded to standard therapy. These drugs are thought to work by turning on genes that limit the growth of cancer cells. The study drugs will be given for 2 courses of chemotherapy duration based on how well the participants will tolerate the study drug and response, each course is 29-36 days long. Treatment for the 2 courses of therapy will last about 2-3 months. -
Principal Investigator:
Dana Goldner, MDThis study evaluates whether odevixibat is safe and effective compared to placebo in children with biliary atresia who have recently undergone a Kasai procedure. This is a double-blind study, so the study doctors and the child/parent will not know whether the child is receiving odevixibat or placebo. Participation will involve several research visits over approximately a 2 year period. After completion of the study, participants may be eligible to join an extension study to receive "open-label" odevixibat through the study. If you have any questions regarding this study, please contact the... -
Principal Investigator:
Dana Goldner, MDThis study evaluates whether odevixibat is safe and effective compared to placebo in children with biliary atresia who have recently undergone a Kasai procedure. This is a double-blind study, so the study doctors and the child/parent will not know whether the child is receiving odevixibat or placebo. Participation will involve several research visits over approximately a 2 year period. After completion of the study, participants may be eligible to join an extension study to receive "open-label" odevixibat through the study. If you have any questions regarding this study, please contact the... -
Principal Investigator:
Luca Szalontay, MDThe purpose of this study is to find the best dose of Palbociclib that can be given safely with chemotherapy in children with solid tumor which has come back or has not responded to the standard therapy. Palbociclib works by targeting proteins that are necessary for cell growth. Palbociclib will be given by mouth on Days 1-14 of a 21-day cycle. It will be given in combination with chemotherapy drugs Irinotecan and Temozolomide. -
Principal Investigator:
Luca Szalontay, MDThe purpose of this study is to find the best dose of Quizartinib that can be given safely with chemotherapy in children and young adults with acute myeloid leukemia (AML) which has come back or has not responded to the standard therapy. Quizartinib will be given by mouth once a day from Day 6 through Day 28. It will be given in combination with chemotherapy drugs cytarabine and fludarabine. -
Principal Investigator:
Luca Szalontay, MDThe purpose of this study is to find the best dose of Quizartinib that can be given safely with chemotherapy in children and young adults with acute myeloid leukemia (AML) which has come back or has not responded to the standard therapy. Quizartinib will be given by mouth once a day from Day 6 through Day 28. It will be given in combination with chemotherapy drugs cytarabine and fludarabine. -
Principal Investigator:
Nobuko Hijiya, MDThis study will utilize an investigational drug combination (also known as the study drugs) called relatlimab and nivolumab as a possible treatment for recurrent/relapsed (returned) or refractory (treatment-resistant) cHL and NHL. An investigational drug combination is one that has not been approved by regulatory agencies, such as the United States (US) Food and Drug Administration (FDA), European Union (EU) European Medicines Agency (EMA), or others. The purpose of this study is to learn about the safety and effectiveness of nivolumab and relatlimab in participants with recurrent/relapsed or...