At a glance
ClinicalIndex Comparison Record- ✓Males aged 5 years or older
- ✓X-linked retinitis pigmentosa confirmed by a retinal specialist
- ✕Participation in another investigational medicinal therapy for ocular disease within the last 6 months
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/5-hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)
In Brief
A Phase 2 clinical trial evaluating AAV5-RPGR for X-Linked Retinitis Pigmentosa. Completed, enrolled 49 participants across 5 sites in 2 countries.
Detailed Summary
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.
Study Details
Timeline
Interventions
Single, subretinal administration of AAV5-RPGR