NCT05878860 · Atsena Therapeutics Inc.
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
(LIGHTHOUSE)
What this study is about
This study will evaluate the safety and how well patients handle the treatment of ATSN-201 in male subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
View original scientific description
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).
Interventions
BIOLOGICAL
ATSN-201
AAV.SPR-hGRK1-hRS1syn
Primary outcome measures
Part A (Dose Escalation) and Part B (Dose Expansion): Safety and tolerability as assessed by dose-limiting toxicities and treatment-emergent adverse events
Time frame: From baseline to week 52
Incidence of dose-limiting toxicities (DLTs) and treatment-emergent adverse events (TEAEs).
Part C (Phase 3, Randomized, Controlled) Effect of ATSN-201 on visual function.
Time frame: From baseline to week 52
Proportion of subjects ≥12 years of age with improvement ≥ 7dB from baseline in microperimetry across prespecified loci in the study eye.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
Inclusion criteria
- Age ≥ 18 for Cohorts 1 through 3, and age ≥ 6 years and \< 18 years for Cohort 4. 2. Male patients with clinical diagnosis of XLRS caused by mutations in RS1. 3. 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).
Exclusion criteria
- Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection. 2. 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. 3. Treatment in a prior ocular gene or cell therapy study.
Where
- Los Angeles, California
- Miami, Florida
- Portland, Oregon
- Philadelphia, Pennsylvania
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Apr 30, 2026 · Source of record for eligibility and locations