ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)

Atsena Therapeutics

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: X-linked Retinoschisis

Treatments: Biological: ATSN-201

Study type: Interventional

Funder types: Industry

Identifiers

NCT05878860

ATSN-201-1

Details and patient eligibility

About

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).

Full description

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.

Enrollment: 21 estimated patients

Sex: Male

Ages: 6 to 64 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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21 participants in 7 patient groups

Cohort 1, Low Dose

Experimental group

Treatment:

Biological: ATSN-201

Cohort 2, High Dose

Experimental group

Treatment:

Biological: ATSN-201

Cohort 3, Mid Dose

Experimental group

Treatment:

Biological: ATSN-201

Cohort 4, Low Volume

Experimental group

Treatment:

Biological: ATSN-201

Cohort 4, High Volume

Experimental group

Treatment:

Biological: ATSN-201

Cohort 4, Control

No Intervention group

Cohort 5, Pediatric

Experimental group

Treatment:

Biological: ATSN-201