- Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene — Active Not Recruiting • Phase III • Rare Diseases • NCT04794101.
- Gene therapy injection into the eye shows promise for treating inherited blindness caused by RPGR gene faults in men.
Verify eligibility, endpoints and current status on the original ClinicalTrials.gov registry before acting on this summary.
- Quick orientation before opening the registry record.
- Checking recruitment status, phase and sponsor at a glance.
- Connecting this trial to nearby guidelines, Drug Science and education.
A clinical trial of AAV5-hRKp.RPGR vector for participants with X-linked retinitis pigmentosa (XLRP) Conditions: X-Linked Retinitis Pigmentosa Interventions: Genetic: AAV5-hRKp.RPGR Intermediate Dose, Genetic: AAV5-hRKp.RPGR Low Dose Lead Sponsor: Janssen Research & Development, LLC Planned Enrollment: 97 participants