BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study
NCT03639844 · Status: NO_LONGER_AVAILABLE · Type: EXPANDED_ACCESS
Last updated 2020-10-05
Summary
Providing access of BPX-501 gene modified T cells and rimiducid to pediatric patients who do not meet the eligibility criteria of the BP-U-004 study.
Conditions
- Hurler Syndrome
- Inherited Metabolic Disorder
- Lysosomal Storage Disorder
- Metachromatic Leukodystrophy
- Inborn Errors of Metabolism
Interventions
- BIOLOGICAL
-
rivogenlecleucel
BPX-501 T cells are genetically modified with a suicide safety switch. The cells are infused after T cell-depleted HSCT to potentially enhance immune reconstitution while reducing severity and duration of GVHD.
- DRUG
-
rimiducid
Rimiducid induces activation of the Caspase 9 suicide gene in BPX-501 T cells inducing apoptosis of the modified T cells in case of GVHD
Sponsors & Collaborators
-
Bellicum Pharmaceuticals
lead INDUSTRY
Principal Investigators
-
Bellicum Pharmaceuticals · Bellicum Pharmaceuticals, Inc.
Eligibility
- Min Age
- 3 Months
- Max Age
- 21 Years
- Sex
- ALL
- Healthy Volunteers
- No
Timeline & Regulatory
Countries
- United States
Study Locations
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