ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
ConditionX-linked Retinoschisis
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age6+
SponsorAtsena Therapeutics Inc.
About this trial
This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
Eligibility criteria
Qualifiers
Age ≥ 18 for Cohorts 1 through 4, and age ≥ 6 years and < 18 years for Cohort 5.
Male patients with clinical diagnosis of XLRS caused by mutations in RS1.
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).
Presence of foveal schisis and /or parafoveal/perifoveal schisis in the study eye on SD-OCT per the Principal Investigator.
Disqualifiers
Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection (eg, advanced glaucoma, optic neuropathy, uveitis, corneal transplants).
Any intraocular surgery (including laser treatment) in the study eye within 6 months prior to Screening or any intraocular surgery anticipated in the study eye during the first 12 months of the study.
Treatment in a prior ocular gene or cell therapy study.
Age ≥ 6 years.
Trial design
Treatments tested in this trial
- ATSN-201
Treatment groups
97 Participants
are divided into 9 treatment groups9
Treatment groupsSee each treatment group below.
Sponsors and collaborators
Source ClinicalTrials.gov