Actively Recruiting
Phase 123 Study of ATSN-201 Gene Therapy for Patients 6 Years and Older With RS1-Associated X-linked Retinoschisis
Led by Atsena Therapeutics Inc. · Updated on 2026-08-10
97
Participants Needed
19
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the safety and effectiveness of ATSN-201 gene therapy in individuals aged 6 years and older who have RS1-associated X-linked retinoschisis XLRS, a genetic eye condition. The study is divided into three parts dose escalation, dose expansion, and a randomized controlled phase to assess different aspects of the therapy. Participants in the randomized phase will be assigned either to receive ATSN-201 or no treatment. Those treated will get a one-time injection of ATSN-201 under the retina in one or both eyes depending on eligibility. The study includes a Main Study Period lasting 1 year with regular assessments, followed by an Extension Study Period of 4 additional years. Control group participants may choose to receive the gene therapy after the first year if eligible. Throughout the study, participants will undergo various eye exams and visual function tests, including imaging with optical coherence tomography, visual acuity measurements, microperimetry, and questionnaires on visual function. Safety is monitored by tracking adverse events and treatment tolerability. The total participation can last up to 5 years, allowing thorough evaluation of both short- and long-term effects of ATSN-201.
CONDITIONS
Brief 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 adult cohorts, and between 6 and under 18 years for the pediatric cohort
- Genetically male patients with clinical diagnosis of X-linked retinoschisis caused by RS1 mutations
- Best corrected visual acuity in the study eye between 34 and 73 ETDRS letters (20/200 to 20/40 Snellen)
- Presence of foveal and/or parafoveal/perifoveal schisis in the study eye as seen on spectral domain optical coherence tomography
- For randomized phase, genetically female patients with biallelic RS1 mutations may also be eligible
- At least one eye must meet ocular inclusion criteria for dosing eligibility
You will not qualify if you...
- Pre-existing eye conditions that increase risk of vision loss from subretinal injection, such as advanced glaucoma, optic neuropathy, uveitis, or corneal transplants
- Intraocular surgery or laser treatment in the study eye within 6 months prior to screening or planned surgery during the first 12 months of the study
- Use of carbonic anhydrase inhibitors (oral or topical) within 1 month prior to screening
- Participation in prior ocular gene or cell therapy studies
- Absence of macular schisis
- Best corrected visual acuity better than 75 ETDRS letters (better than 20/32 Snellen)
Research Team
A
Atsena Therapeutics Clinical Trials
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