A Multiple-Site, Phase 1/2, Safety and Efficacy Trial of a Recombinant Adeno-associated Virus Vector Expressing Retinoschisin (rAAV2tYF-CB-hRS1) in Patients With X-linked Retinoschisis

AGTC-RS1-001

This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector expressing retinoschisin (rAAV2tYF-CB-hRS1) in patients with X-linked retinoschisis. Up to 27 participants will be enrolled and 3 dose levels will be evaluated in a dose escalation format.

2015-05-01

2019-04-01

2023-05-09

Completed

Early phase I

27

Inclusion Criteria include: 1. Retinal disease consistent with a diagnosis of XLRS and documented mutations in the RS1 gene 2. Male individual at least 18 years of age (dose escalation phase) or at least 6 years of age (maximum tolerated dose phase), 3. Able to perform tests of visual and retinal function, 4. Visual acuity specified for each group 5. Not treated with CAIs currently or within 3 months prior to study enrollment, 6. Have acceptable laboratory parameters. Exclusion Criteria include: 1. Prior receipt of any AAV gene therapy product, 2. Pre-existing eye conditions that would preclude the planned intravitreal injection or interfere with interpretation of study endpoints or complications of vector administration.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 27, 'type': 'ACTUAL'}}

2023-06-12