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Phase 1 Trial of Gene Therapy RP-L102 in Pediatric Fanconi Anemia Subtype A
NCT03814408Phase 1Unknown
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Jan 2019
- Primary completion
- Dec 2020
- Modality
- Gene Editing, Gene Silencing, Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of PGK-FANCA-Wpre* transduced CD34+ cells in Fanconi anemia (FA), sponsored by Rocket Pharmaceuticals, Inc. Primary endpoint: Number of participants with treatment-related adverse events at 3 years. Status: unknown.
02
Drugs
PGK-FANCA-Wpre* transduced CD34+ cells
03
Conditions
Fanconi anemia (FA)
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