Status:
TERMINATED
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases
Lead Sponsor:
Bellicum Pharmaceuticals
Conditions:
Primary Immune Deficiency Disorders
Hemophagocytic Lymphohistiocytosis
Eligibility:
All Genders
4-55 years
Phase:
PHASE1
Brief Summary
The purpose of this study is to determine a safe dose of BPX-501 gene modified T cells infused after a haplo-identical stem cell transplant to facilitate engraftment and the safety of Rimiducid (AP190...
Detailed Description
This is a single arm dose finding study evaluating the safety and efficacy of a BPX 501 infusion (T cells genetically modified with the inducible Caspase 9 suicide gene) of 3x10E6 to 1X10E7 cells/kg f...
Eligibility Criteria
Inclusion
- Patient must meet eligibility criteria for allogeneic transplantation
- Lack of suitable conventional donor (10/10 allele matched related or unrelated donor) or presence of rapidly progressive disease not permitting time to identify an unrelated donor
- Males or females
- Age \< 55 years old and \> 4 months
- Diagnosis of a nonmalignant disorder considered treatable by HCT.
- HLA typing will be performed at high resolution (allele level) for the HLA-A, -B, Cw, DRBl, and DQB1 loci.
- i. A minimum match of 5/10 is required. ii. The donor and recipient must be identical, as determined by high resolution typing, in at least one allele of each of the following
- If capable of reproduction, patient must agree to use contraception or abstinence to prevent pregnancy during the first year of enrollment and treatment.
- Informed consent signed by patient (if ≥18 years old) or parent/guardian (if \<18 years old).
- Fanconi anemia patients ONLY i) Patients must meet one of the following criteria to be eligible for this study:
- Any patient with Fanconi anemia and bone marrow failure involving 2 of the following 3 lineages: granulocyte count \<0.5 x 109/L, platelet count \<20 x 109/L, or hemoglobin \<8 g/dL.
- Any patient with Fanconi anemia who requires red blood cell or platelet transfusions because of marrow failure
- Any patient with Fanconi anemia who has a life-threatening bone marrow failure involving a single hematopoietic lineage.
Exclusion
- Serious organ dysfunction
- Pregnant or breast-feeding
- Evidence of HIV infection
- Bovine product allergy
- Patients with an active infectious disease
- Patients with Fanconi anemia with AML/MDS.
Key Trial Info
Start Date :
February 1 2015
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2018
Estimated Enrollment :
1 Patients enrolled
Trial Details
Trial ID
NCT02231710
Start Date
February 1 2015
End Date
January 1 2018
Last Update
March 25 2024
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Fred Hutchinson Cancer ResearchCenter
Seattle, Washington, United States, 98109