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
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?
Trial 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).