ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis
brief summary
This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 5 years of age with RS1-associated X-linked retinoschisis (XLRS).
detailed 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 assessments over the next 4 years as part of the Extension Study Period.
Some subjects may have all their study visits at a surgery site.
Some subjects may go to one study site (a medical site) to determine eligibility and another study site (a surgery site) to have surgery - including pre-operative care and approximately 1-week of post-operative care per treated eye. After the surgery, they will go back to the other study site (the medical site) to complete the follow-up visits.
Subjects who do not receive treatment as part of the control group can choose to receive ATSN-201 in one or both eyes after the 1-year Main Study Period if eligible. If the subject is not eligible at the Month 12 visit, eligibility may be reassessed at the Month 18 visit.
official title
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)