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
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- X-linked Retinoschisis
- Sponsor
- Applied Genetic Technologies Corp
- Enrollment
- 27
- Locations
- 9
- Primary Endpoint
- Number of Participants Experiencing Adverse Events
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
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.
Detailed Description
This will be a non-randomized, open label, Phase 1/2 dose escalation study. Up to 27 participants will be enrolled. Each participant will receive the study agent by intravitreal injection in one eye on a single occasion. Enrollment will begin with the lowest dose and will proceed to higher doses only after review of safety data by a Data and Safety Monitoring Committee (DSMC). Participants in the dose escalation phase will be ≥ 18 years of age. After the maximum tolerated dose is identified individuals ≥ 6 years of age will be enrolled. Safety will be measured by the number and proportion of participants experiencing adverse events and immune response to RS1. Efficacy will be measured by evaluation of changes in visual function and schisis cavity size.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Outcomes
Primary Outcomes
Number of Participants Experiencing Adverse Events
Time Frame: From Day 0 to Month 12 (12 months)
Number of participants with ocular adverse events, assessed by standard ophthalmic examination, including slit lamp biomicroscopy, tonometry, and indirect ophthalmoscopy.
Secondary Outcomes
- Change From Baseline in Schisis Cavity Size on Optical Coherence Tomography (OCT)(From Day 0 to Month 12 (12 months))
- Change From Baseline in B-wave Amplitude in Electroretinogram (ERG) Responses(From Day 0 to Month 12 (12 months))
- Change From Baseline in Best Corrected Visual Acuity (BCVA)(From Day 0 to Month 12 (12 Months))