Trials / NCT03252847
Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
Completed Phase 1 / Phase 2 Gene therapy
- Sponsor
- MeiraGTx UK II Ltd
- Treatment
- AAV5-RPGR
- Gene
- RPGR
- Conditions
- X-Linked Retinitis Pigmentosa
- Ages
- 5 Years to no upper limit
- Enrollment
- 49 participants
- Started
- 2017-07-31
- Main results expected
- 2021-11-18
- Results posted
- Yes
- Record last updated
- 2025-01-08
What the study is testing
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.
Where it runs
United Kingdom (2)
- Leeds Teaching Hospitals NHS Trust, Leeds
- Moorfields Eye Hospital NHS Foundation Trust, London
United States (3)
- Massachusetts Eye and Ear Institute, Boston, Massachusetts
- Kellogg Eye Center, Ann Arbor, Michigan
- UPMC Eye Center, Pittsburgh, Pennsylvania