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APR-2020 in RPS19-Deficient Diamond-Blackfan Anemia
NCT07476183Phase 1Recruiting
- Sponsor
- Apriligen
- Start
- Apr 2026
- Primary completion
- Apr 2027
- Modality
- Gene Editing, Gene Silencing, Stem Cell Therapies
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of APR-2020 in Diamond-Blackfan anemia (DBA), sponsored by Apriligen. Primary endpoints: Incidence of protocol-defined dose limiting toxicities at 30 days, Incidence and severity of treatment emergent adverse events at 24 months and 3 more. Started Apr 2026; status: recruiting.
02
Drugs
APR-2020
03
Conditions
Diamond-Blackfan anemia (DBA)
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