Phase 2
N=27
Safety and Efficacy of rAAV-hRS1 in Patients With X-linked Retinoschisis (XLRS)
X-linked Retinoschisis
Bottom Line
View on ClinicalTrials.gov: NCT02416622 ↗Enrolled (actual)
27
Serious AEs
14.8%
Results posted
Jul 2020
Primary outcome: Primary: Number of Participants Experiencing Adverse Events — 6; 7; 12 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- rAAV2tYF-CB-hRS1 (Biological)
- Age
- Pediatric, Adult, Older Adult · 6+ yrs
- Sex
- Male
- Sponsor
- Beacon Therapeutics
- Primary completion
- Apr 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Experiencing Adverse Events |
6; 7; 12 | — |
| SECONDARY Change From Baseline in Best Corrected Visual Acuity (BCVA) |
38.7; 56.3; 50.4; 39.5; 59.9; 51.4 | — |
| SECONDARY Change From Baseline in Schisis Cavity Size on Optical Coherence Tomography (OCT) |
1.13; 1.12; 1.28; 1.33; 1.28; 1.04 | — |
| SECONDARY Change From Baseline in B-wave Amplitude in Electroretinogram (ERG) Responses |
107.3; 206.0; 169.9; 92.8; 142.2; 164.0 | — |
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.
Eligibility Criteria
Inclusion Criteria include:
- Retinal disease consistent with a diagnosis of XLRS and documented mutations in the RS1 gene
- Male individual at least 18 years of age (dose escalation phase) or at least 6 years of age (maximum tolerated dose phase),
- Able to perform tests of visual and retinal function,
- Visual acuity specified for each group
- Not treated with CAIs currently or within 3 months prior to study enrollment,
- Have acceptable laboratory parameters.
Exclusion Criteria include:
- Prior receipt of any AAV gene therapy product,
- Pre-existing eye conditions that would preclude the planned intravitreal injection or interfere with interpretation of study endpoints or complications of vector administration.
Data sourced from ClinicalTrials.gov (NCT02416622). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.