Study design & results
Available in the Gosset platform where reported.
Trial intelligence / Profile preview
Sepofarsen in children under 8 years with LCA10
2020-000535-45Phase 2Completed
- Sponsor
- ProQR Therapeutics
- Start
- Sep 2020
- 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) (3 arms), sponsored by ProQR Therapeutics. Primary endpoint: Safety and tolerability of sepofarsen. Started Sep 2020; status: completed.
02
Drugs
sepofarsen
03
Conditions
Leber's congenital amaurosis type 10 (LCA10)
Beyond the preview
Go deeper on 2020-000535-45.
Study design, results and competitive context are available in the Gosset platform where reported.
Competitive context
Available in the Gosset platform where reported.
Bring the full picture into focus.
See how Gosset can support your research on 2020-000535-45.