Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
A clinical trial of AAV5-RPGR vector for patients with X-linked retinitis pigmentosa (XLRP)
Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 48 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Bilateral, subretinal administration of AAV5-RPGR - immediate treatment group
Masking: Single (Outcomes Assessor)
Masking Description: No intervention - deferred treatment group (Bilateral, subretinal administration of AAV5-RPGR to be administered in the follow-up study)
Primary Purpose: Treatment
Official Title: Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene
Estimated Study Start Date: March 1, 2021
Estimated Primary Completion Date: July 5, 2022
Estimated Study Completion Date: July 5, 2022
Arm:
- Experimental: Experimental
- Deferred Treatment
| Category | Value |
|---|---|
| Study type(s) | Interventional |
| Estimated enrolment | 48 |
| Estimated Study start date | 01 March 2021 |
| Estimated Study Completion Date | 05 July 2022 |