Status:
ACTIVE_NOT_RECRUITING
Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant
Lead Sponsor:
Bellicum Pharmaceuticals
Conditions:
Acute Lymphoblastic Leukemia
Leukemia, Acute Myeloid (AML), Child
Eligibility:
All Genders
1-26 years
Phase:
PHASE1
PHASE2
Brief Summary
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that ...
Detailed Description
This is a Phase 1/2 study evaluating the safety and feasibility of BPX-501 T cells infused after partially mismatched, related, TCR alpha beta T cell depleted hematopoietic stem cell transplant (HSCT)...
Eligibility Criteria
Inclusion
- Age \> 1 month and \< 26 years
- Life expectancy \> 10 weeks
- Subjects deemed eligible for allogeneic stem cell transplantation.
- Subjects with life-threatening hematological malignancies (high-risk ALL in 1st CR, ALL in 2nd or subsequent CR, AML in 1st CR, AML in 2nd or subsequent CR, myelodysplastic syndromes, non-Hodgkin lymphomas in 2nd or subsequent CR, other hematologic malignancies eligible for stem cell transplantation per institutional standard);
- Non-malignant disorders amenable to cure by an allograft:
- primary immune deficiencies,
- severe aplastic anemia not responding to immune suppressive therapy,
- osteopetrosis,
- hemoglobinopathies, (thalassemias, and sickle cell anemia, and Diamond-Blackfan anemia among others)
- congenital/hereditary cytopenia, including Fanconi Anemia before any clonal malignant evolution (MDS, AML) Note: Subjects will be eligible if they meet either item 4 OR item 5.
- Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically identical relative or 10/10 unrelated donor evaluated using high resolution molecular typing) or presence of rapidly progressive disease not permitting time to identify an unrelated donor
- A minimum genotypic identical match of 5/ 10 is required.
- The donor and recipient must be identical, as determined by high resolution typing, at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA- DRB1 and HLA-DQB1.
- Lansky/Karnofsky score \> 50
- Signed written informed consent
Exclusion
- Greater than Grade II acute GVHD or chronic extensive GVHD due to a previous allograft at the time of inclusion
- Subject receiving an immunosuppressive treatment for GVHD treatment due to a previous allograft at the time of inclusion
- Dysfunction of liver (ALT/AST \> 5 times normal value, or bilirubin \> 3 times normal value), or of renal function (creatinine clearance \< 30 mL / min)
- Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive heart failure or left ventricular ejection fraction \< 40%)
- Current active infectious disease (including positive HIV serology or viral RNA)
- Serious concurrent uncontrolled medical disorder
- Pregnant or breastfeeding subject
- For subjects who have received more than 1 x 10E5 alpha/beta T cells/kg with the graft infusion the clinical trial site must contact the sponsor for approval to be eligible to receive BPX-501 infusion.
Key Trial Info
Start Date :
April 1 2014
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
May 1 2034
Estimated Enrollment :
120 Patients enrolled
Trial Details
Trial ID
NCT03301168
Start Date
April 1 2014
End Date
May 1 2034
Last Update
July 12 2022
Active Locations (10)
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1
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
2
Stanford University - Division of Pediatric Stem Cell Transplant & Regenerative Medicine
Palo Alto, California, United States, 94304
3
Children's National Medical Center
Washington D.C., District of Columbia, United States, 20010
4
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30322