RecruitingPhase 1Phase 2NCT05878860
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
A Phase 1/2, Open-Label, Dose Escalation and Dose Expansion Study to Evaluate the Safety and Tolerability of ATSN-201 Gene Therapy in Male Subjects With RS1-Associated X-linked Retinoschisis
Sponsor
Atsena Therapeutics Inc.
Enrollment
21 participants
Start Date
Aug 22, 2023
Study Type
INTERVENTIONAL
Conditions
Summary
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).
Eligibility
Sex: MALEMin Age: 6 Years
Inclusion Criteria3
- Age ≥ 18 for Cohorts 1 through 3, and age ≥ 6 years and \< 18 years for Cohort 4.
- 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).
Exclusion Criteria3
- Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection.
- 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.
- Treatment in a prior ocular gene or cell therapy study.
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Interventions
BIOLOGICALATSN-201
AAV.SPR-hGRK1-hRS1syn
Locations(4)
View Full Details on ClinicalTrials.gov
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NCT05878860