Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
NCT ID: NCT03252847
Last Updated: 2025-01-08
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE1/PHASE2
49 participants
INTERVENTIONAL
2017-07-31
2021-11-18
Brief Summary
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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.
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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Phase 1 (Part 1, Dose Escalation)
Participants receive one of three doses of AAV5-RPGR
AAV5-RPGR
Single, subretinal administration of AAV5-RPGR
Phase 2 (Part 2; Expansion)
Participants receive one of two doses of AAV5-RPGR
AAV5-RPGR
Single, subretinal administration of AAV5-RPGR
Interventions
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AAV5-RPGR
Single, subretinal administration of AAV5-RPGR
Eligibility Criteria
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Inclusion Criteria
* Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI)
Exclusion Criteria
5 Years
MALE
No
Sponsors
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Syne Qua Non Limited
INDUSTRY
Bionical Emas
INDUSTRY
MeiraGTx UK II Ltd
INDUSTRY
Responsible Party
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Principal Investigators
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James Bainbridge, Prof
Role: PRINCIPAL_INVESTIGATOR
University College, London
Locations
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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
Countries
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References
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Michaelides M, Besirli CG, Yang Y, DE Guimaraes TAC, Wong SC, Huckfeldt RM, Comander JI, Sahel JA, Shah SM, Tee JJL, Kumaran N, Georgiadis A, Minnick P, Zeldin R, Naylor S, Xu J, Clark M, Anglade E, Wong P, Fleck PR, Fung A, Peluso C, Kalitzeos A, Georgiou M, Ripamonti C, Smith AJ, Ali RR, Forbes A, Bainbridge J. Phase 1/2 AAV5-hRKp.RPGR (Botaretigene Sparoparvovec) Gene Therapy: Safety and Efficacy in RPGR-Associated X-Linked Retinitis Pigmentosa. Am J Ophthalmol. 2024 Nov;267:122-134. doi: 10.1016/j.ajo.2024.05.034. Epub 2024 Jun 12.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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MGT009
Identifier Type: -
Identifier Source: org_study_id
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