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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

22CT012: GPC2 CAR T Cell Trial

The purpose of this study is to test an experimental approach called cell therapy for neuroblastoma or retinoblastoma. This study will take a patient's own white blood cells (T cells) and change them to turn against the cancer. These cells are an experimental treatment and have not been approved by the FDA. CAR T cells have been used to treat other cancers, but GPC2 CAR T cells have not been administered to human patients prior to this clinical trial.

If they choose to participate in this study, active participation will last approximately 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 GPC2 CAR T treatment.

This study will assess the safety and effectiveness of GPC2 CAR T cells. Overall, approximately 45 subjects will be enrolled on this study.

Phase: Phase I

Actively recruiting: Yes

Category: Adults, Children

Conditions: Retinoblastoma (Eye Cancer in Children), Relapsed and Refractory Neuroblastoma

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

CAR T-Cells for Severe Lupus

The purpose of this study is to test an experimental approach called cell therapy for Systemic Lupus Erythematosus (SLE).CAR T-cells have been used to treat some cancers, but these cells, known as CART19 cells, are an experimental treatment for lupus. The CAR T-cells are made by collecting T-cells from patients and changing them to find and remove B-cells, including ones that are involved in causing lupus. Participants will be given CART19 cells through an IV and then monitored for up to 24 months.

Participants will also be asked to participate in a separate, long term follow-up study to monitor their health for up to 15 years after their last CART19 treatment.

Phase: Phase II

Actively recruiting: Yes

Category: Adults, Children

Conditions: Pediatric Lupus

CD22 Redirected Autologous T Cells for Acute Lymphoblastic Leukemia (ALL)

The purpose of this study is to test an experimental approach called cell therapy for Leukemia that involves B cells (tumor cells). This is a study for people who have been previously treated for Leukemia. This study will take the participant's white blood cells (T cells) and then modify them in a lab to recognize and target the cancer cells. These modified cells are then put back into the body through infusion. If you have previously received a similar cell therapy, you may still be eligible for this study.


Phase: Pilot

Actively recruiting: Yes

Category: Adults, Children

Conditions: Acute Lymphoblastic Leukemia (ALL), Relapsed/Refractory Acute Lymphoblastic Leukemia (ALL)

CD33 CART AML

This phase 1/2 study will determine the safety, feasibility, and efficacy of CD33 CART cells following lymphodepleting chemotherapy in patients with acute myeloid leukemia (AML). Patients who are between ages 1 to 30 years with relapsed or refractory AML will be eligible for this study.

Phase: Phase I, Phase II

Actively recruiting: No

Category: Adults, Children

Conditions: Acute Myelogenous Leukemia (AML)

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)

CHOP-ALS20 Lentiviral gene Therapy for Beta Thalassemia

This clinical trial aims to test a new gene therapy in adults (18 to 39 years old) with beta thalassemia who require regular red cell transfusions. The study will evaluate the safety and efficacy of genetically engineered blood stem cells using a novel lentiviral vector that produces a high levels of a healthy beta globin. Several tests and procedures such as review of medical history, blood and urine tests, bone marrow aspirate/biopsy, heart ultrasound, electrocardiogram, magnetic resonance imaging (MRI) of the heart and liver, and lung tests will be done to find out if subjects are eligible to participate in this trial. If eligible, blood stem cells will be collected by apheresis. These stem cells will then have a healthy beta globin gene inserted. Subjects will then be hospitalized, and chemotherapy will be given to empty out the bone marrow. The stem cells, with the inserted gene (CHOP-ALS20), will then be given back to the subject. Subjects will be followed for side effects and to see if the inserted gene helps make hemoglobin and reduce or stop blood transfusions.

Phase: Phase I, Phase II

Actively recruiting: Yes

Category: Adults

Conditions: Beta Thalassemia (Cooley's Anemia)

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: No

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 and Refractory Neuroblastoma

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