2024-511411-25-00RecruitingPhase 3
Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene
Janssen Cilag International8 sites in 6 countries31 target enrollmentStarted: June 21, 2024Last updated:
Drugs
Trial Snapshot
- Phase
- Phase 3
- Status
- Recruiting
- Sponsor
- Janssen Cilag International
- Enrollment
- 31
- Locations
- 8
- Primary Endpoint
- Efficacy: Change from baseline in binocular vision-guided mobility assessment (VMA) after bilateral subretinal delivery of AAV5-hRKp.RPGR Adverse events Laboratory Assessment
Study Overview
Brief Summary
To assess the long-term safety and tolerability of AAV5-hRKp.RPGR in individuals with RPGR-XLRP
To assess the long-term efficacy of treatment with AAV5-hRKp.RPGR in individuals with RPGR-XLRP based on assessments of functional vision as measured by vision-guided mobility assessment (VMA).
Study Design
- Study Type
- Interventional
Eligibility Criteria
- Ages
- 0 years to 64 years (0-17 Years, 18-64 Years)
- Accepts Healthy Volunteers
- Yes
Inclusion Criteria
- •1.Previously completed participation in Study MGT-RPGR-
- •2.Must reconfirm that he or she understands the purpose of, and procedures required for, the study and is willing to participate in the study.
Exclusion Criteria
- •There are no specific exclusion criteria.
Outcomes
Primary Outcomes
Efficacy: Change from baseline in binocular vision-guided mobility assessment (VMA) after bilateral subretinal delivery of AAV5-hRKp.RPGR Adverse events Laboratory Assessment
Efficacy: Change from baseline in binocular vision-guided mobility assessment (VMA) after bilateral subretinal delivery of AAV5-hRKp.RPGR Adverse events Laboratory Assessment
Secondary Outcomes
No secondary outcomes reported
Investigators
CTIS Point of Contact
Scientific
Janssen Cilag International
Study Sites (8)
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