An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. (BRIGHTEN)
An Open-Label, Dose Escalation and Double-Masked, Randomized, Controlled Study to Evaluate the Safety and Tolerability of Sepofarsen in Pediatric Subjects <8 Years of Age With Leber Congenital Amaurosis Type 10 (LCA10) Due to the c.2991 +1655A>G (p.Cys998X) Mutation.
Sponsor: ProQR Therapeutics
A PHASE2/PHASE3 clinical study on Blindness and Eye Diseases, this trial is ongoing. The trial is conducted by ProQR Therapeutics and has accumulated 6 data snapshots since 2021. Longitudinal tracking of this trial contributes to a broader understanding of treatment development timelines.
Status Flow
Change History
6 versions recorded-
Sep 2025 — Present [monthly]
Unknown PHASE2/PHASE3
-
Sep 2024 — Sep 2025 [monthly]
Unknown PHASE2/PHASE3
-
Jul 2024 — Sep 2024 [monthly]
Unknown PHASE2/PHASE3
Status: Unknown Status → Unknown · Phase: PHASE2_PHASE3 → PHASE2/PHASE3
-
Apr 2024 — Jul 2024 [monthly]
Unknown Status PHASE2_PHASE3
Status: Recruiting → Unknown Status
-
Apr 2022 — Apr 2024 [monthly]
Recruiting PHASE2_PHASE3
▶ Show 1 earlier version
-
May 2021 — Apr 2022 [monthly]
Recruiting PHASE2_PHASE3
First recorded
Mar 2021
Trial started
Per CT.gov start date — pre-dates our first snapshot
Eligibility Summary
No eligibility information available.
Contact Information
- ProQR Therapeutics
For direct contact, visit the study record on ClinicalTrials.gov .