4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)

Summary

This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retinitis pigmentosa (XLRP) due to mutations in the gene encoding retinitis pigmentosa GTPase regulator (RPGR).

Timeline

Start

2020-06-09

Primary completion

2026-06

Completion

2029-05

Drugs

Indications