Now Enrolling
LIGHTHOUSE Trial of ATSN-201 Gene Therapy for X-Linked Retinoschisis (XLRS) – Phase 3
This website provides information about the LIGHTHOUSE trial of ATSN-201 investigational gene therapy for treatment of X-linked retinoschisis (XLRS). It is intended for people with XLRS and their families. Contact us to learn more or if you are interested in participating in this clinical trial.
The LIGHTHOUSE Phase 3 Trial
What is ATSN-201?
ATSN-201 is an investigational gene therapy for the treatment of X-linked retinoschisis (XLRS). XLRS is caused by a mutation (variation) in the RS1 gene. ATSN-201 is delivered by a one-time injection to the eye underneath the retina. The gene therapy is designed to deliver a functional copy of the RS1 gene to the eye.
What is the purpose of the LIGHTHOUSE Phase 3 trial?
The focus of the LIGHTHOUSE Phase 3 trial is to measure how ATSN-201 affects vision in people with XLRS and how safe it is. The trial also aims to understand the following about ATSN-201:
Does it improve the structure of the macula*?
Does it improve visual function, such as visual acuity?
Does it make visual activities, such as reading, easier?
*The macula is located at the center of the retina and is used for high-resolution color vision
Who can take part in the LIGHTHOUSE Phase 3 trial?
The Phase 3 portion of the LIGHTHOUSE trial is open to people:
- Age 6 years or older*
- Who have a diagnosis of XLRS caused by mutations in the RS1 gene
These are not all the eligibility criteria.
Click here for more information about the trial’s eligibility criteria and current sites.
*As Part C begins, the study will primarily enroll participants who are 12 years of age or older. There will be a limited number of openings for children ages 6-11.
The trial is currently only enrolling people with XLRS who live outside of Europe. Updates will be shared when European countries become eligible.
What to expect in the LIGHTHOUSE Phase 3 trial?
LIGHTHOUSE Phase 3 involves about 5-6 years of participation, including:
~3 months for screening and baseline assessments
12 months of participation in the main study period
~4-5 years of additional follow up with around 1 visit per year
Click here to download a brochure that provides an overview of LIGHTHOUSE Phase 3 trial visits and procedures.
Phase 3 of the LIGHTHOUSE trial is now recruiting and enrolling participants.
For more information about the LIGHTHOUSE Phase 3 trial, talk to your doctor, fill out this form, or contact [email protected].
You can also:
- Visit ClinicalTrials.gov and enter identifier number NCT05878860
- Share this website with your doctor
About the LIGHTHOUSE Trial
The LIGHTHOUSE trial is a Phase 1/2/3 clinical trial of investigational ATSN-201 gene therapy for treatment of X-linked retinoschisis (XLRS). It is the first trial to test ATSN-201 in humans.
Phase 1 (Part A) and Phase 2 (Part B)
The Phase 1 (Part A, Dose Escalation) and Phase 2 (Part B, Dose Expansion) portions of the LIGHTHOUSE trial tested different concentrations and amounts of ATSN-201 to find the safest and most effective dose.
Phases 1 and 2 are now fully enrolled.
Phase 3 (Part C)
The Phase 3 (Part C) portion is now recruiting and enrolling participants. This part of the trial is designed to understand how ATSN-201 affects vision in people with XLRS, including assessing the structure of the macula, visual function, and ease of visual activities such as reading.
Now Enrolling for Phase 3.
About XLRS
X-linked retinoschisis (XLRS), sometimes referred to as juvenile retinoschisis, is an inherited eye disease caused by mutations (variations) in the RS1 gene. The RS1 gene mutation causes schisis, or abnormal splitting of the retinal layers, which leads to impaired vision that is not correctable with glasses. XLRS leads to progressive vision loss. Currently, there are no approved treatments for XLRS.
About Atsena
Atsena Therapeutics is a clinical-stage biopharmaceutical company focused on developing treatments for inherited retinal diseases such as X-linked retinoschisis (XLRS).
Click here for more information about Atsena.
Contact Us
For more information about the LIGHTHOUSE trial, complete this form or contact our Patient Advocacy team by email at [email protected].
Please note that this trial is currently only enrolling people with XLRS who live outside of Europe. Updates will be shared when European countries become eligible.
