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Sepofarsen paired-eye placebo-controlled study in CEP290-associated Leber congenital amaurosis
2024-518378-14-00Phase 3Authorised
- Sponsor
- Laboratoires Théa
- Start
- Apr 2025
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 9
01
Overview
Phase 3, randomised, double-blind trial of sepofarsen in Leber congenital amaurosis (LCA) and Leber's congenital amaurosis type 10 (LCA10) (2 arms), sponsored by Laboratoires Théa. Primary endpoint: Change from baseline in best-corrected visual acuity. Planned start Apr 2025; status: authorised.
02
Drugs
sepofarsen
03
Conditions
Leber congenital amaurosis (LCA)Leber's congenital amaurosis type 10 (LCA10)
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