Clinical Trials
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Principal Investigator:
James Hinkley Garvin Jr, MDChildren and young adults with diffuse midline glioma will be treated with Focused Ultrasound (FUS) with DEFINITY microbubbles and neuro-navigator-controlled sonication. The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known cure exists, and determine whether there are any clinical benefits of the study treatment. Children with progressive diffuse midline gliomas (DMGs) have very poor outcomes and have very limited options. One of the main obstacles in the effectiveness of medical therapy in... -
Principal Investigator:
James Hinkley Garvin Jr, MDChildren and young adults with diffuse midline glioma will be treated with Focused Ultrasound (FUS) with DEFINITY microbubbles and neuro-navigator-controlled sonication. The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known cure exists, and determine whether there are any clinical benefits of the study treatment. Children with progressive diffuse midline gliomas (DMGs) have very poor outcomes and have very limited options. One of the main obstacles in the effectiveness of medical therapy in... -
Principal Investigator:
James Hinkley Garvin Jr, MDChildren and young adults with diffuse midline glioma will be treated with Focused Ultrasound (FUS) with DEFINITY microbubbles and neuro-navigator-controlled sonication. The overall goal of this study is to see if the study treatment plan will improve the outcome of participants with diffuse midline glioma, for which no known cure exists, and determine whether there are any clinical benefits of the study treatment. Children with progressive diffuse midline gliomas (DMGs) have very poor outcomes and have very limited options. One of the main obstacles in the effectiveness of medical therapy in... -
Principal Investigator:
Steven J. Lobritto, MDTReatment 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, MDTReatment 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, MDThis 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... -
Principal Investigator:
Mercedes Martinez, MDThe purpose of this clinical research study is to learn more about the use of the investigational study drug, volixibat, for the treatment of pruritus (itching) associated with primary sclerosing cholangitis or PSC. Volixibat is a drug that is designed to lower circulating bile acid levels may lead to the itching in patients with PSC. Eligible patients will be randomly assigned to volixibat or to placebo for 28 weeks. This is a double-blind study, so the study doctors and participants will not know to which arm the participants are assigned. After completing 28 weeks of treatment, if eligible...