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Research Studies

Cellular Therapy and Transplant Section Research Studies

Find research studies available to children cared for by the Cellular Therapy and Transplant Section team.

Existing patients or family members
New patients, referrals and second opinions

Recruitment Status

Clinical trial phase

CABA-201-002 for Myositis

The purpose of this study is to evaluate the safety and tolerability of CABA-201, an experimental form of therapy, in participants with active Idiopathic Inflammatory Myopathies (IIM) and Juvenile Idiopathic Inflammatory Myopathies (JIIM). CABA-201 is a CAR therapy drug, which uses T- cells (immune cells) and modifies them to find and remove the other immune cells (B cells) involved in causing this disease. The cells will be collected through a process called leukapheresis, then modified in a lab, the modified version will be infused into the body.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, Children

CHOP T-Cell Acute Lymphoblastic Leukemia Study (BEAM-201)

The purpose of this study is to test a cell therapy for T-Cell Lymphoblastic Leukemia or Lymphoma. The CAR T-cells are made by collecting T-cells from donors and modifying them to attach to cancer cells. CAR T-cells have been used to treat other cancers, but these cells, known as BEAM-201 cells, are an experimental treatment. The participants will be given BEAM-201 cells via an IV and then monitored for up to 12 months.

This study will assess the safety and effectiveness of BEAM-201 cells.

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Acute Lymphoblastic Leukemia (ALL)

EDIT-301: Phase 1/2 Sickle Cell Disease Gene Therapy Trial

Individuals with severe sickle cell disease, who are between 18 and 50 years old, may be able to participate. A number of screening tests will be done to determine if subjects can participate. These include review of your past medical history, physical exam, blood and urine tests, pregnancy test, heart ultrasound, electrocardiogram, pulmonary function test, kidney function test, questionnaires, and magnetic resonance imaging (MRI) of the heart and liver. If eligible, blood stem cells will be collected by apheresis.A gene in thesestem cells will thenbe edited to help the stem cells make more fetal hemoglobin. Higher levels of fetal hemoglobin can improve red blood cell sickling. You will then be hospitalized and chemotherapy will be given to empty out the bone marrow. The stem cells, with themodifiedgene, will then be given back to you. You will be followed for side effects and to see if themodifiedgene helps you make healthyred blood cells.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults, MRI/Imaging Studies

Conditions: Sickle Cell Disease

MICRO study

The purpose of this study is to collect and store data samples, such as stool, saliva, mouth swabs, and dental plaque specimens for research about the microbiome. The microbiome is a collection of the microbes, or very small forms of life, that live on and inside the human body.

Phase: N/A

Actively recruiting: Yes

Category: Adults, Children

PHOX2B CAR T-Cells for Relapsed Neuroblastoma

The purpose of this study is to test an experimental approach called cell therapy for neuroblastoma. This study will take a patient's own white blood cells (T cells) and change them to turn against the cancer. CART cells have been used to treat other cancers, but PHOX2B CART cells have not been administered to human patients prior to this clinical trial. Participants will be given PHOX2B cells via an IV and then monitored for up to 12 months.

Participants will also be asked to sign consent to a separate long term follow-up protocol to monitor their health for up to 15 years after their last PHOX2B CART treatment.

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Relapsed or Refractory Neuroblastoma

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