ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

Recruiting now · Phase 3

Conditions studied: X-linked Retinoschisis

In brief

This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).

Key facts

Study ID

NCT05878860

Run by

Atsena Therapeutics Inc.

People needed

97

Starts

2023-08-22

Expected to finish

2033-04-01

Last updated by the study team

2026-08-04

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.
• Part C:
• Inclusion Criteria:
• Note: For patients ineligible for bilateral dosing based on the ocular exclusion criteria, the same eye must meet all ocular inclusion criteria, but none of the ocular exclusion criteria, to be eligible for unilateral dosing.
• General All of the following criteria must be met for unilateral or bilateral dosing.
• 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.
• Ocular At least 1 eye must meet all of the following criteria for both unilateral and bilateral dosing.
• 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 Criteria:
• General None of the following criteria can be met for unilateral or bilateral dosing.
• 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.
• Absence of macular schisis.
• BCVA better than 75 ETDRS letters (corresponding to a Snellen acuity of 20/32).
• 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).
• 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.

Where it is running

• Associated Retina Consultants — Phoenix, Arizona, United States (enrolling)
• Children's Hospital of Los Angeles — Los Angeles, California, United States (enrolling)
• Bascom Palmer Eye Institute — Miami, Florida, United States (enrolling)
• Massachusetts Eye and Ear — Boston, Massachusetts, United States (enrolling)
• Mayo Clinic — Rochester, Minnesota, United States (enrolling)
• Oregon Health Sciences University — Portland, Oregon, United States (enrolling)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• Retina Foundation of the Southwest — Dallas, Texas, United States (enrolling)
• University of Michigan — Ann Arbor, Michigan, United States
• Oxford Eye Hospital — Oxford, England, United Kingdom
• Columbia University — New York, New York, United States
• Flaum Eye Institute, University of Rochester — Rochester, New York, United States
• Shiley Eye Institute — La Jolla, California, United States
• Duke University — Durham, North Carolina, United States
• Stanford University — Palo Alto, California, United States
• UC San Francisco — San Francisco, California, United States
• Moran Eye Center — Salt Lake City, Utah, United States
• University of Wisconsin — Madison, Wisconsin, United States
• Boston Children's Hospital — Boston, Massachusetts, United States

Full record on ClinicalTrials.gov

Trial information comes from ClinicalTrials.gov and is refreshed daily. TrialsForMe does not provide medical care and does not run the studies it lists.