Actively Recruiting
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
Led by Atsena Therapeutics Inc. · Updated on 2026-04-30
97
Participants Needed
4
Research Sites
501 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
CONDITIONS
Official Title
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older for cohorts 1 to 4, or age 6 to under 18 years for cohort 5
- Male patients with a clinical diagnosis of XLRS caused by RS1 mutations (Parts A and B)
- Genetically male patients with XLRS caused by pathogenic RS1 mutations or genetically female patients with biallelic pathogenic RS1 mutations (Part C)
- Best corrected visual acuity in the study eye between 34 and 73 ETDRS letters (20/200 to 20/40 Snellen acuity)
- Presence of foveal schisis and/or parafoveal/perifoveal schisis in the study eye visible on SD-OCT
- At least one eye meeting ocular criteria for unilateral or bilateral dosing in Part C
You will not qualify if you...
- Pre-existing eye conditions in the study eye that increase risk of vision loss from subretinal injection, such as advanced glaucoma, optic neuropathy, uveitis, or corneal transplants
- Any intraocular surgery or laser treatment in the study eye within 6 months before screening or anticipated during the first 12 months of the study
- Prior treatment in an ocular gene or cell therapy study
- Treatment with any carbonic anhydrase inhibitor (oral or topical) within 1 month before screening (Part C)
- Absence of macular schisis (Part C)
- Best corrected visual acuity better than 75 ETDRS letters (20/32 Snellen acuity) (Part C)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Children's Hospital of Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
2
Bascom Palmer Eye Institute
Miami, Florida, United States, 33136
Actively Recruiting
3
Oregon Health Sciences University
Portland, Oregon, United States, 97239
Actively Recruiting
4
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
A
Atsena Therapeutics Clinical Trials
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
9
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