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A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis Pigmentosa (XLRP)
Sponsor: Janssen Research & Development, LLC
Summary
The purpose of this study is to assess the safety and tolerability of subretinal delivery of Adeno-associated Virus Vector (AAV5 hRKp.RPGR) gene therapy in adults and children with X-linked retinitis pigmentosa.
Official title: An Open-label, Multicenter, Phase 2 Follow-on Study for Second Eye Treatment of Patients Previously Treated With a Recombinant Adeno-associated Virus Vector (AAV5 hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
Key Details
Gender
MALE
Age Range
5 Years - Any
Study Type
INTERVENTIONAL
Enrollment
24
Start Date
2024-10-10
Completion Date
2030-10-24
Last Updated
2026-03-13
Healthy Volunteers
No
Conditions
Interventions
AAV5-hRKp.RPGR
AAV5-hRKp.RPGR will be administered sub-retinally.
No intervention (Follow-Up assessment)
Participants will not receive any intervention and will undergo follow-up assessment.
Locations (3)
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States
University of Michigan Kellogg Eye Center
Ann Arbor, Michigan, United States
Moorfields Eye Hospital
London, United Kingdom