A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations

Brief Summary:
This study will evaluate and compare the safety, efficacy, and tolerability of 2 doses of a recombinant adeno-associated virus vector (AGTC-501) to an untreated control group in male patients with X-linked retinitis pigmentosa caused by RPGR mutations.

Detailed Description:
This Phase 2/3 study is a randomized, controlled, masked, multi-center study evaluating and comparing 2 doses of AGTC-501 to an untreated control group. A single subretinal injection of AGTC-501 Dose 1(low dose) or Dose 2(high dose) will be administered in subjects in 2 treatment groups (N=~42), while subjects in the untreated control group (N=~21) will be followed and evaluated up to Month 12, after which they will be eligible to receive treatment with AGTC-501 Dose 2.

Approximately 63 eligible male subjects between 8 and 50 years of age (inclusive) will be randomized in a 1:1:1 ratio to 1 of 3 groups (low dose, high dose, untreated control).


Study Type: Interventional (Clinical Trial)
Estimated Enrollment: 63 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Subjects are randomized in a 1:1:1 ratio to 1 of 3 groups (low dose, high dose, untreated). After 12 months, subjects in the untreated group are eligible to receive the high dose of AGTC-501.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Masking Description: For groups 1 and 2 (AGTC-501 dosing groups), both the subjects and the Investigators are masked to dose assignment, and only the unmasked staff who prepare the dose are unmasked to the subject's dose assignment. Due to the need to perform the subretinal injection to administer the study drug, both subjects and Investigators will know whether the subject was assigned to a treatment group or to the control group. To minimize bias of the treated and control study eye evaluations, starting at Month 3, microperimetry, mobility testing, and BCVA assessments will be conducted by appropriately qualified masked evaluators who do not know whether the subject underwent surgery. Subjects will be instructed not to disclose whether they had surgery to the examiner administering the test.

Primary Purpose: Treatment
Official Title: A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene
Estimated Study Start Date: August 2021
Estimated Primary Completion Date: January 2024
Estimated Study Completion Date: March 2029

Arm:
- Active Comparator: Group 1: Low Dose
- Active Comparator: Group 2: High Dose
- Group 3: Control

View full details