The LIGHTHOUSE Study: A Gene Therapy Clinical Trial for X-Linked Retinoschisis

The LIGHTHOUSE Study: A Gene Therapy Clinical Trial for X-Linked Retinoschisis

Contact

 If you are interested in participating in the study or want to learn more, please contact: Contact us
This study is now recruiting. Learn more about enrolling here.

Description
This is a Phase ½ study testing an experimental gene therapy developed by Atsena Therapeutics for X-Linked Retinoschisis (XLRS). This study will be testing the safety and the efficacy of different doses of the gene therapy in males diagnosed with XLRS. The LIGHTHOUSE study is looking for approximately 21 volunteers to participate. It will test different doses of the gene therapy to find out if it is safe and effective for people with XLRS. Participating in this study will include a total of 16 visits to the clinic over a 5-year span with most visits taking place in the first year. The procedure includes a one-time administration of the gene therapy in one eye through a surgical procedure called vitrectomy with subretinal injection.

Eligibility and criteria
IRB Number:
23-021220
Eligible age range:
18 years - 65 years
Clinical trial phase:
Phase I
Phase II
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 (LIGHTHOUSE)

What to expect

Related specialties
Smiling CHOP patient holding shopping bag

We need families like you

Your time and participation make a difference in supporting the work of the Research Institute and children in our community. Learn more about opportunities to participate in our research.