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PBKR03 in Early Infantile Krabbe Disease
NCT04771416Phase 2Suspended
- Sponsor
- GEMMA Biotherapeutics
- Start
- Feb 2022
- Primary completion
- Jan 2027
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 6
01
Overview
Phase 2, open-label trial of PBKR03 in Krabbe disease (2 arms), sponsored by GEMMA Biotherapeutics. Primary endpoints: Number of participants with treatment-related adverse events and serious adverse events at Grade 3 or higher at 24 months, Change from baseline in nerve conduction and velocity in motor nerve conduction studies at 5 years and 4 more. Started Feb 2022; status: suspended.
02
Drugs
PBKR03
03
Conditions
Krabbe disease
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