A Phase 1/2/3, Open-Label, Dose Escalation, Dose Expansion and Randomized, Controlled Study to Evaluate the Safety and Efficacy of ATSN-201 Gene Therapy in Subjects With RS1-Associated X-linked Retinoschisis (LIGHTHOUSE)
Atsena Therapeutics Inc.
Summary
This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
Description
The study is designed in three parts: a dose escalation phase (Part A), a dose expansion phase (Part B) and a randomized, controlled phase (Part C). In Part C of the study, eligible patients who enroll in this study will be randomly assigned to be treated with ATSN-201 or to have no treatment; subjects assigned to ATSN-201 will receive the drug as a one-time subretinal injection of ATSN-201 in one eye or both eyes, depending on whether only one or both eyes meet criteria for treatment. Subjects will have regular assessments for 1 year as part of the Main Study Period and additional assessment…
Eligibility
- Age range
- 6+ years
- Sex
- All
- Healthy volunteers
- No
Part A and B: Inclusion Criteria: 1. Age ≥ 18 for Cohorts 1 through 4, and age ≥ 6 years and \< 18 years for Cohort 5. 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). 4. Presence of foveal schisis and /or parafoveal/perifoveal schisis in the study eye on SD-OCT per the Principal Investigator. Exclusion Criteria: 1. Pre-existing eye conditions in the study eye that would contribute sign…
Interventions
- BiologicalATSN-201
AAV.SPR-hGRK1-hRS1syn
Locations (4)
- Children's Hospital of Los AngelesLos Angeles, California
- Bascom Palmer Eye InstituteMiami, Florida
- Oregon Health Sciences UniversityPortland, Oregon
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania