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Clinical trial

Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene

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Last updated:9th Mar 2021
Status: Not yet recruiting
Identifier: NCT04671433
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

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