Clinical trial
A Phase 1/2 Open-Label, Multicenter Dose-Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects With Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy)
NCT04771416 is registered in ClinicalTrials.gov with Gemma Biotherapeutics as lead sponsor.
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Registry record
As filed in ClinicalTrials.gov.
- Title
- A Phase 1/2 Open-Label, Multicenter Dose-Ranging and Confirmatory Study to Assess the Safety, Tolerability and Efficacy of PBKR03 Administered to Pediatric Subjects With Early Infantile Krabbe Disease (Globoid Cell Leukodystrophy)
- Lead sponsor
- Gemma Biotherapeutics
- Phase
- Phase 1/2
- Overall status
- Suspended
- Start date
- February 24, 2022
- Completion date
- January 31, 2030
- Enrollment
- 24 (estimated)
- Sites
- 9
Conditions
As the registry lists them; a condition with its own page is linked.