Gene therapy ATSN-201 for X-linked retinoschisis, an inherited eye disease
Tests ATSN-201 gene therapy in people with RS1-linked retinoschisis, an inherited eye disease.
Plain-language title generated from the registry record
Registry title: A Phase 1/2/3 Study of ATSN-201 Gene Therapy in Subjects with RS1-Associated X linked Retinoschisis
Key facts
- Where
- Munich and Tuebingen, GermanySee the 2 sites
- Phase
- Phase 3
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for people aged 6 or older with X-linked retinoschisis caused by changes in the RS1 gene, with reduced vision and fluid-filled splits in the retina.
- Age
- 6 years or older
- Sex
- Genetically male people with an RS1 gene change; genetically female people only if they have RS1 gene changes on both copies of the gene
- Disease stage
- Clinical diagnosis of X-linked retinoschisis, vision of 34 to 73 ETDRS letters (Snellen 20/200 to 20/40), and retinal schisis at the fovea seen on SD-OCT
- Earlier treatment
- Cannot have had carbonic anhydrase inhibitor treatment (oral or topical) within 1 month before screening, or treatment in an earlier eye gene or cell therapy study
- Biomarker or test result
- Pathogenic or likely pathogenic mutation in the RS1 gene (on both copies in genetically female people)
Main reasons for not taking part
- Carbonic anhydrase inhibitor treatment within 1 month before screening
- Treatment in a prior eye gene or cell therapy study
- No macular schisis
- Vision better than 75 ETDRS letters (Snellen 20/32)
- Eye surgery or laser treatment within 6 months before screening, or eye surgery expected in the first 12 months of the study
Read by machine on 9 November 2026.
Where in Germany
- Ludwig-Maximilians-Universitaet MuenchenMunich, GermanyNot yet recruiting
- Universitaetsklinikum Tuebingen AöRTuebingen, GermanyNot yet recruiting
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- Atsena Therapeutics Inc.
- Trial code
- NCT05878860EU 2026-525886-38-00
- Record updated
- 19 October 2026
- Original description
To determine the effect of ATSN-201 on visual function.Read full original descriptionShow less
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)
To determine the effect of ATSN-201 on visual function.
Inclusion: Age ≥ 6 years; Genetically male patients with clinical diagnosis of XLRS caused by pathogenic or likely pathogenic mutations in RS1 OR Genetically female patients with clinical diagnosis of XLRS caused by biallelic pathogenic or likely pathogenic mutations in RS1; BCVA of 34 to 73 ETDRS letters (corresponding to a Snellen acuity of 20/200 to 20/40); Presence of foveal schisis on SD-OCT.
Exclusion: Treatment with any carbonic anhydrase inhibitor (oral or topical) within 1 month prior to Screening; Treatment in a prior ocular gene or cell therapy study; Any intraocular surgery (including laser treatment) within 6 months prior to Screening or any intraocular surgery anticipated during the first 12 months of the study; Pre-existing eye conditions that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants); Absence of macular schisis; BCVA better than 75 ETDRS letters (corresponding to a Snellen acuity of 20/32).