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Clinical trial

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.

NCT04855045 is registered in ClinicalTrials.gov with ProQR Therapeutics as lead sponsor.

NCT NCT04855045
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Registry record

As filed in ClinicalTrials.gov.

Title
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.
Lead sponsor
ProQR Therapeutics
Phase
Phase 2/3
Overall status
Unknown status
Start date
March 23, 2021
Completion date
December 31, 2023
Enrollment
15 (estimated)
Sites
9

Conditions

As the registry lists them; a condition with its own page is linked.

blindness, eye diseases, eye disorders congenital, leber congenital amaurosis, leber congenital amaurosis 10, neurologic manifestations, retinal degeneration, retinal disease, retinal dystrophies, sensation disorders, vision disorders