Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
Completed · Phase 1/Phase 2
Conditions studied: X-Linked Retinitis Pigmentosa
In brief
Phase 1 of the study is a dose escalation of the subretinal administration of AAV5-hRKp.RPGR vector to assess the safety of this vector in participants with XLRP caused by mutations in RPGR. Participants enrolled in Phase 1 were assigned to a dose group based on when they enrolled (i.e., sequential assignment). Phase 2 of the study is a cohort expansion of the subretinal administration of AAV5-hRKp.RPGR vector to assess the safety and efficacy of this vector in participants with XLRP caused by mutations in RPGR. Participants enrolled in Phase 2 were randomized to immediate or deferred treatment.
Key facts
- Study ID
- NCT03252847
- Run by
- MeiraGTx UK II Ltd
- People needed
- 49
- Starts
- 2017-07-31
- Expected to finish
- 2021-11-18
- Last updated by the study team
- 2025-01-08
Who can join
Age: 5 and older. Sex: male. Healthy volunteers: not accepted.
Where it is running
- Massachusetts Eye and Ear Institute — Boston, Massachusetts, United States
- Kellogg Eye Center — Ann Arbor, Michigan, United States
- UPMC Eye Center — Pittsburgh, Pennsylvania, United States
- Leeds Teaching Hospitals NHS Trust — Leeds, United Kingdom
- Moorfields Eye Hospital NHS Foundation Trust — London, United Kingdom
Full record on ClinicalTrials.gov
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