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

Description

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).

Conditions

X-linked Retinoschisis

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Study Overview

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Study Details

Study overview

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).

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

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

Condition
X-linked Retinoschisis
Intervention / Treatment

-

Contacts and Locations

Los Angeles

Children's Hospital of Los Angeles, Los Angeles, California, United States, 90027

Miami

Bascom Palmer Eye Institute, Miami, Florida, United States, 33136

Portland

Oregon Health Sciences University, Portland, Oregon, United States, 97239

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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).
  • 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.

Ages Eligible for Study

6 Years to

Sexes Eligible for Study

MALE

Accepts Healthy Volunteers

No

Collaborators and Investigators

Atsena Therapeutics Inc.,

Study Record Dates

2029-10