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

MeiraGTx

Status and phase: Completed; Phase 2; Phase 1

Conditions: X-Linked Retinitis Pigmentosa

Treatments: Genetic: AAV5-RPGR

Study type: Interventional

Funder types: Industry

Identifiers

NCT03252847

MGT009

Details and patient eligibility

About

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.

Full 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.

Enrollment: 49 patients

Sex: Male

Ages: 5+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Key inclusion Criteria:

• Males aged 5 years or older
• 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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49 participants in 2 patient groups

Phase 1 (Part 1, Dose Escalation)

Experimental group

Description:

Participants receive one of three doses of AAV5-RPGR

Treatment:

Genetic: AAV5-RPGR

Phase 2 (Part 2; Expansion)

Experimental group

Description:

Participants receive one of two doses of AAV5-RPGR

Treatment:

Genetic: AAV5-RPGR