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QR-110/Sepofarsen phase 2 trial in CEP290-related Leber congenital amaurosis
2018-003501-25Phase 2Completed
- Sponsor
- ProQR Therapeutics
- Start
- Nov 2018
- 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 2, randomised, double-blind trial of sepofarsen in Leber congenital amaurosis (LCA) (2 arms), sponsored by ProQR Therapeutics. Primary endpoint: Change from baseline in best corrected visual acuity. Started Nov 2018; status: completed.
02
Drugs
sepofarsen
03
Conditions
Leber congenital amaurosis (LCA)
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