Clinical Research Directory
Browse clinical research sites, groups, and studies.
Long-term Follow-up Gene Therapy Study for RPGR- XLRP
Sponsor: MeiraGTx Ocular UK Ltd
Summary
This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.
Official title: Long-term Follow-up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV5- hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
Key Details
Gender
MALE
Age Range
5 Years - Any
Study Type
OBSERVATIONAL
Enrollment
42
Start Date
2017-07-31
Completion Date
2025-09-17
Last Updated
2026-09-25
Healthy Volunteers
No
Conditions
Interventions
AAV5-hRKp.RPGR
Participants will not receive any intervention in this study. Participants from the retrospective (MGT009-NCT03252847) study will be follow-up.
Locations (5)
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States
Univ of Michigan Medical Center
Ann Arbor, Michigan, United States
University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, United States
Leeds Teaching Hospitals NHS Trust
Leeds, United Kingdom
Moorfields Eye Hospital
London, United Kingdom