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RP-L102 gene therapy in pediatric Fanconi anemia subtype A
NCT04248439Phase 2Active, not recruiting
- Sponsor
- Rocket Pharmaceuticals, Inc.
- Start
- Jul 2020
- Primary completion
- May 2026
- Modality
- Gene Editing, Gene Silencing, Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of PGK-FANCA-Wpre* transduced CD34+ cells in Fanconi anemia (FA), sponsored by Rocket Pharmaceuticals, Inc. Primary endpoint: Bone Marrow Colony-Forming Cell Mitomycin-C Resistance at 21 months. Started Jul 2020; status: active, not recruiting.
02
Drugs
PGK-FANCA-Wpre* transduced CD34+ cells
03
Conditions
Fanconi anemia (FA)
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