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Sepofarsen in children under 8 years with LCA10
NL-OMON55235Phase 2Active, not recruiting
- Sponsor
- ProQR Therapeutics
- Start
- Mar 2021
- 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's congenital amaurosis type 10 (LCA10) (5 cohorts), sponsored by ProQR Therapeutics. Primary endpoints: Incidence and severity of ocular adverse events and Incidence and severity of non-ocular adverse events. Started Mar 2021; status: active, not recruiting.
02
Drugs
sepofarsen
03
Conditions
Leber's congenital amaurosis type 10 (LCA10)
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