Thinking of joining a study?

Register your interest

Become a volunteer?

NCT05878860 | RECRUITING | X-linked Retinoschisis

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

Atsena Therapeutics Inc.

Brief 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).

Condition or disease

X-linked Retinoschisis

Intervention/treatment

ATSN-201

Phase

PHASE1

PHASE2

Detailed 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.

Study Type : INTERVENTIONAL
Estimated Enrollment : 21 participants
Masking : NONE
Masking Description : Cohort 3 will be partially masked.
Primary Purpose : TREATMENT
Official Title : 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
Actual Study Start Date : 2023-08-22
Estimated Primary Completion Date : 2025-10
Estimated Study Completion Date : 2029-10

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 6 Years
Sexes Eligible for Study: MALE
Accepts Healthy Volunteers:
Criteria
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.
ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

Location Details

NCT05878860

Please Choose a site

How to Participate

Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.

Locations

RECRUITING

United States, California

Children's Hospital of Los Angeles

Los Angeles, California, United States, 90027

RECRUITING

United States, Florida

Bascom Palmer Eye Institute

Miami, Florida, United States, 33136

RECRUITING

United States, Oregon

Oregon Health Sciences University

Portland, Oregon, United States, 97239

RECRUITING

United States, Pennsylvania

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104