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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

21 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-08-22

Study Completion Date

2029-10-31

Brief Summary

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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).

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Detailed Description

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Eligible patients who enroll in this study will receive a one-time subretinal injection of ATSN-201 in one eye. Safety and tolerability will be evaluated for 5 years.

Conditions

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X-linked Retinoschisis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Cohort 3 will be partially masked.

Study Groups

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Cohort 1, Low Dose

Group Type EXPERIMENTAL

ATSN-201

Intervention Type BIOLOGICAL

AAV.SPR-hGRK1-hRS1syn

Cohort 2, High Dose

Group Type EXPERIMENTAL

ATSN-201

Intervention Type BIOLOGICAL

AAV.SPR-hGRK1-hRS1syn

Cohort 3, Mid Dose

Group Type EXPERIMENTAL

ATSN-201

Intervention Type BIOLOGICAL

AAV.SPR-hGRK1-hRS1syn

Cohort 4, Low Volume

Group Type EXPERIMENTAL

ATSN-201

Intervention Type BIOLOGICAL

AAV.SPR-hGRK1-hRS1syn

Cohort 4, High Volume

Group Type EXPERIMENTAL

ATSN-201

Intervention Type BIOLOGICAL

AAV.SPR-hGRK1-hRS1syn

Cohort 4, Control

Group Type NO_INTERVENTION

No interventions assigned to this group

Cohort 5, Pediatric

Group Type EXPERIMENTAL

ATSN-201

Intervention Type BIOLOGICAL

AAV.SPR-hGRK1-hRS1syn

Interventions

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ATSN-201

AAV.SPR-hGRK1-hRS1syn

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion Criteria

1. Age ≥ 18 for Cohorts 1 through 3, and age ≥ 6 years and \< 18 years for Cohort 4.
2. Male patients with clinical diagnosis of XLRS caused by mutations in RS1.
3. 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 Criteria

1. Pre-existing eye conditions in the study eye that would contribute significantly to an increased risk of visual loss from a subretinal injection.
2. 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.
3. Treatment in a prior ocular gene or cell therapy study.

Minimum Eligible Age

6 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No