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Clinical Trials/2024-511411-25-00
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
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

Sponsor Class
Pharmaceutical company
Responsible Party
Principal Investigator
Principal Investigator

CTIS Point of Contact

Scientific

Janssen Cilag International

Study Sites (8)

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