Actively Recruiting
A Phase 1/2/3, Open-Label, Dose Escalation, Dose Expansion and Randomized, Controlled Study to Evaluate the Safety and Efficacy of ATSN-201 Gene Therapy in Subjects With RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)
Led by Atsena Therapeutics Inc. · Updated on 2026-04-30
97
Participants Needed
4
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating 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 includes three parts: dose escalation, dose expansion, and a randomized controlled phase, designed to explore different doses and assess the treatment's impact on visual function and safety. In the randomized phase, participants will be randomly assigned to receive a one-time subretinal injection of ATSN-201 in one or both eyes if they meet treatment criteria, or to no treatment initially. After one year, those in the control group may choose to receive the gene therapy if eligible. The study period includes a main phase with regular assessments for one year, followed by an extension phase with additional evaluations lasting four years. Participants will undergo various eye exams and vision tests such as best-corrected visual acuity, low-luminance visual acuity, contrast sensitivity, microperimetry, and macular imaging using spectral domain optical coherence tomography. Patient-reported visual function will also be collected using questionnaires appropriate for adults and children. Safety will be closely monitored through treatment-emergent adverse events and dose-limiting toxicities throughout the study and extension periods, with total follow-up lasting up to five years.
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 Cohorts 1 through 4, and age 6 years to less than 18 years for Cohort 5
- Male patients with a clinical diagnosis of XLRS caused by mutations in the RS1 gene (Parts A and B)
- Best corrected visual acuity in the study eye between 34 to 73 ETDRS letters (Snellen equivalent 20/200 to 20/40)
- Presence of foveal schisis and/or parafoveal or perifoveal schisis in the study eye on spectral domain optical coherence tomography
- Age 6 years or older (Part C)
- Genetically male patients with XLRS caused by pathogenic or likely pathogenic RS1 mutations, or genetically female patients with biallelic pathogenic or likely pathogenic RS1 mutations (Part C)
- At least one eye meeting all ocular inclusion criteria for unilateral or bilateral dosing (Part C)
You will not qualify if you...
- Pre-existing eye conditions in the study eye that increase risk of visual loss from subretinal injection (e.g., advanced glaucoma, optic neuropathy, uveitis, corneal transplants)
- Any intraocular surgery or laser treatment in the study eye within 6 months prior to screening or planned during 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 prior to screening (Part C only)
- Absence of macular schisis (Part C only)
- Best corrected visual acuity better than 75 ETDRS letters (Snellen equivalent 20/32) (Part C only)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - One-time injection at start of the study
Participants assigned to receive ATSN-201 will get a one-time subretinal injection in one or both eyes depending on eligibility. Participants in the control group will not receive treatment initially.
1 treatment visit (in-person)
Duration - 1 year
Participants will have regular assessments for 1 year after treatment in the Main Study Period.
Regular visits during 1-year main study period
Duration - 4 years
Additional assessments occur over the next 4 years as part of the Extension Study Period to monitor long-term effects.
Periodic visits during 4-year extension period
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
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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