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Trial intelligence / Profile preview
Phase 3 paired-eye trial of sepofarsen for CEP290-associated Leber congenital amaurosis
NL-OMON58804Phase 3Recruiting
- Sponsor
- Laboratoires Théa
- Start
- Jul 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
- 1
01
Overview
Phase 3, randomised, double-blind trial of sepofarsen in Leber congenital amaurosis (LCA) and Leber's congenital amaurosis type 10 (LCA10), sponsored by Laboratoires Théa. Primary endpoint: Change from baseline in best-corrected visual acuity. Started Jul 2025; status: recruiting.
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Drugs
sepofarsen
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Conditions
Leber congenital amaurosis (LCA)Leber's congenital amaurosis type 10 (LCA10)
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