ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis (NCT05878860)
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)
🩺 Plain-English Summary (8th-Grade Level)
This study will evaluate the safety and efficacy of ATSN-201 in subjects ≥ 6 years of age with RS1-associated X-linked retinoschisis (XLRS).
Who can join: Review the eligibility criteria below.
Location: Associated Retina Consultants — Phoenix, Arizona
Age range: 6 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (19 Sites)
- Associated Retina Consultants — Phoenix, Arizona
- Shiley Eye Institute — La Jolla, California
- Children's Hospital of Los Angeles — Los Angeles, California
- Stanford University — Palo Alto, California
- UC San Francisco — San Francisco, California
- Bascom Palmer Eye Institute — Miami, Florida
- Massachusetts Eye and Ear — Boston, Massachusetts
- Boston Children's Hospital — Boston, Massachusetts
- University of Michigan — Ann Arbor, Michigan
- Mayo Clinic — Rochester, Minnesota
- Columbia University — New York, New York
- Flaum Eye Institute, University of Rochester — Rochester, New York
- Duke University — Durham, North Carolina
- Oregon Health Sciences University — Portland, Oregon
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania
- Retina Foundation of the Southwest — Dallas, Texas
- Moran Eye Center — Salt Lake City, Utah
- University of Wisconsin — Madison, Wisconsin
- Oxford Eye Hospital — Oxford, England
📞 Contact the Study Coordinator
Phone: (984) 261-2001
Email: clinicaltrials@atsenatx.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
Explore by condition: All X-linked Retinoschisis clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.