ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
- Registration Number
- NCT05878860
- Lead Sponsor
- Atsena Therapeutics Inc.
- Brief Summary
This study will evaluate the safety and tolerability of ATSN-201 in male subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
- Detailed Description
Eligible patients who enroll in this study will receive a one-time subretinal injection of ATSN-201 in one eye. Safety and tolerability will be evaluated for 5 years.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- Male
- Target Recruitment
- 21
- Age ≥ 18 for Cohorts 1 through 3, and age ≥ 6 years and < 18 years for Cohort 4.
- Male patients with clinical diagnosis of XLRS caused by mutations in RS1.
- Best corrected visual acuity (BCVA) in study eye of 34 to 73 Early Treatment Diabetic Retinopathy Study (ETDRS) letters (corresponding to a Snellen acuity of 20/200 to 20/40).
- Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection.
- Any intraocular surgery (including laser treatment) in the study eye within 6 months prior or any intraocular surgery anticipated in the study eye during the first 12 months of the study.
- Treatment in a prior ocular gene or cell therapy study.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Cohort 1, Low Dose ATSN-201 - Cohort 4, High Volume ATSN-201 - Cohort 5, Pediatric ATSN-201 ATSN-201 at High Volume Cohort 3 ATSN-201 ATSN-201 at Mid Dose Cohort 1 ATSN-201 ATSN-201 at Low Dose Cohort 2 ATSN-201 ATSN-201 at High Dose Cohort 4, High Dose ATSN-201 ATSN-201 at High Volume Cohort 4, Low Dose ATSN-201 ATSN-201 at Low Volume Cohort 5, Pediatric ATSN-201 - Cohort 2, High Dose ATSN-201 - Cohort 3, Mid Dose ATSN-201 - Cohort 4, Low Volume ATSN-201 -
- Primary Outcome Measures
Name Time Method Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events From baseline to week 52 Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs).
- Secondary Outcome Measures
Name Time Method Visual function as assessed by full-field electroretinogram parameters From baseline to week 52 Change in full-field electroretinogram (ffERG) parameters.
Subject-reported visual function as assessed by the NEI VFQ-25 in adult subjects From baseline to week 52 Change in the National Eye Institute's Visual Function Questionnaire 25 (NEI VFQ-25) score for adult subjects with scores from 0 to 100 where a higher score indicates a better outcome.
Visual acuity as assessed by low-luminance visual acuity From baseline to week 52 Change in low-luminance visual acuity (LLVA).
Visual function as assessed by static perimetry From baseline to week 52 Change in static perimetry.
Visual function as assessed by microperimetry From baseline to week 52 Change in microperimetry.
Macular structure as assessed by spectral domain optical coherence tomography From baseline to week 52 Change in spectral domain optical coherence tomography (SD-OCT).
Visual acuity as assessed by best-corrected visual acuity From baseline to week 52 Change in best-corrected visual acuity (BCVA).
Visual function as assessed by contrast sensitivity From baseline to week 52 Change in contrast sensitivity.
Macular structure as assessed by fundus autofluorescence From baseline to week 52 Change in fundus autofluorescence (FAF).
Subject-reported visual function as assessed by the CVAQC in pediatric subjects From baseline to week 52 Change in the Cardiff Visual Ability Questionnaire for Children (CVAQC) score for pediatric subjects with scores from -3.00 to +2.80 where a higher score indicates a worse outcome.
Trial Locations
- Locations (4)
Children's Hospital of Los Angeles
🇺🇸Los Angeles, California, United States
Bascom Palmer Eye Institute
🇺🇸Miami, Florida, United States
Oregon Health Sciences University
🇺🇸Portland, Oregon, United States
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States