Purpose of this Study
If you join this study, you will have a procedure where the study medicine is placed into the back of your eye. After this, you will return for follow up visits for up to five years so doctors can check how your eyes are doing. There will be about seventeen visits in total. At these visits, doctors will test your vision and examine your eyes to watch for changes and make sure the treatment is safe.
Who Can Participate?
Eligibility
People can join this study if they are older than 6 years and have a diagnosis of X linked retinoschisis, which is a condition that affects vision. They cannot have taken part in another study that used gene therapy or cell therapy for the eyes before.
Age Range
6-110
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
Description
This study is testing a new treatment called ATSN 201 for people who have a rare eye condition called X linked retinoschisis. This condition affects vision because of a change in a gene. The study drug is a type of gene therapy, which is designed to fix or replace the problem gene. Researchers want to learn if this treatment is safe and if it can help improve vision or slow down vision loss.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
No
Study Details
Full 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)
Principal Investigator
Lejla
Vajzovic
Protocol Number
PRO00120184
NCT ID
NCT05878860
Phase
III
Enrollment Status
Pending Open to Enrollment