Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)
Sponsored by MeiraGTx UK II Ltd
About this trial
Last updated 3 years ago
Study ID
MGT009
Status
Completed
Type
Interventional
Phase
Phase 1/Phase 2
Placebo
No
Accepting
5+ Years
Male
Not accepting
Healthy Volunteers
Trial Timing
Ended 4 years ago
What is this trial about?
The Phase 1 part of the study is a dose escalation of subretinal administration of AAV2/5
vector to assess the safety of this vector in participants with XLRP caused by mutations in
RPGR.
The Phase 2 part of the study is a cohort expansion of subretinal administration of AAV2/5
vector to assess the safety and efficacy of this vector in participants with XLRP caused by
mutations in RPGR.
What are the Participation Requirements?
Key inclusion Criteria:
- Are aged 5 years or older male
- Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI)
Key exclusion Criteria:
• Have participated in another research study involving an investigational medicinal
therapy for ocular disease within the last 6 months