An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.
Clinical trial NCT04855045 · UNKNOWN
Trial details
- NCT number
- NCT04855045
- Sponsor
- ProQR Therapeutics
- Status
- UNKNOWN
- Phase
- Phase 2, Phase 3
- Study type
- INTERVENTIONAL
- Start date
- 2021-03-23
- Primary completion
- 2023-12-01
- Enrollment
- 15
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