NCT03252847

Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR)

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)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 49 (actual)
• Sponsor: MeiraGTx UK II Ltd · Industry
• Sex: Male
• Age: 5 Years
• Healthy volunteers: Not accepted

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.

Detailed description

This is an open-label phase 1/2 dose-escalation and cohort expansion trial to determine the safety and efficacy of subretinal administration of AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

Conditions

• X-Linked Retinitis Pigmentosa

Interventions

Type	Name	Description
GENETIC	AAV5-RPGR	Single, subretinal administration of AAV5-RPGR

Timeline

• Start date: 2017-07-31
• Primary completion: 2021-11-18
• Completion: 2021-11-18
• First posted: 2017-08-17
• Last updated: 2025-01-08
• Results posted: 2025-01-08

Locations

5 sites across 2 countries: United States, United Kingdom

Regulatory

• FDA-regulated drug study