Study on Gene-Modified Donor T-Cells After Stem Cell Transplant for Blood Disorders
Sponsor: Bellicum Pharmaceuticals
ClinicalTrials ID:NCT03301168
This clinical trial is exploring whether specially modified immune cells from a family donor can help children and young adults recover faster after a stem cell transplant for blood disorders. The donor cells have a safety feature that allows them to be destroyed if they cause harmful reactions.
Patient Parameters
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Program Overview
This study is focused on children and young adults with blood disorders who are receiving a stem cell transplant from a partially matched family donor. The research aims to find out if adding specially grown immune cells, called T cells, from the donor can help the patient's immune system recover more quickly after the transplant. These T cells are modified in the lab to include a self-destruct switch, which can be activated if they start attacking the patient's body, a condition known as graft versus host disease (GvHD).
Description
This clinical trial is in Phase 1/2 and is testing the safety and feasibility of using BPX-501 T cells after a stem cell transplant that has been depleted of certain T cells. The goal is to see if these BPX-501 T cells can help the immune system recover in children and young adults with blood disorders. The study also looks at whether these cells can reduce the severity and duration of a condition called graft versus host disease (GvHD), which can occur after a transplant.
If GvHD does occur and does not respond to standard treatments, the study will evaluate the use of a special drug (AP1903/rimiducid) to treat it. This drug can activate the self-destruct switch in the modified T cells, potentially stopping the harmful reaction.
Eligibility Criteria
Inclusion Criteria
- Age between 1 month and 26 years.
- Expected to live more than 10 weeks.
- Eligible for a stem cell transplant.
- Have life-threatening blood cancers or disorders that can be treated with a stem cell transplant, such as:
- High-risk acute lymphoblastic leukemia (ALL) or acute myeloid leukemia (AML) in certain stages.
- Myelodysplastic syndromes or non-Hodgkin lymphomas in certain stages.
- Primary immune deficiencies, severe aplastic anemia, osteopetrosis, hemoglobin disorders like thalassemias and sickle cell anemia, and other hereditary blood disorders.
- No suitable conventional donor available or disease progressing too quickly to wait for a donor.
- A partial genetic match with the donor is required.
- Must have a certain level of physical functioning (Lansky/Karnofsky score > 50).
- Must provide signed informed consent.
Exclusion Criteria
- Severe graft versus host disease from a previous transplant.
- Currently receiving treatment for graft versus host disease from a previous transplant.
- Severe liver or kidney dysfunction.
- Serious heart disease.
- Active infections, including HIV.
- Other serious uncontrolled medical conditions.
- Pregnant or breastfeeding.
- If more than a certain amount of T cells were received with the transplant, approval from the sponsor is needed to participate.
Locations
| FACILITY | ZIP | CITY | STATE |
|---|---|---|---|
| Children's Hospital Los Angeles | 90027 | Los Angeles | California |
| Stanford University - Division of Pediatric Stem Cell Transplant & Regenerative Medicine | 94304 | Palo Alto | California |
| Children's National Medical Center | 20010 | Washington D.C. | District of Columbia |
| Children's Healthcare of Atlanta | 30322 | Atlanta | Georgia |
| Dana-Farber Boston Children's Cancer and Blood Disorders Center | 02215 | Boston | Massachusetts |
| Children's Hospital at Montefiore | 10467 | The Bronx | New York |
| Oregon Health Sciences University - Doernbecher Children's Hospital | 97239 | Portland | Oregon |
| University of Texas Southwestern-Children's Medical Center | 77390 | Dallas | Texas |
| Baylor College of Medicine/ Texas Children's Hospital | 77030 | Houston | Texas |
| Fred Hutchinson Cancer Research Center | 98109 | Seattle | Washington |
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