Purpose of this Study
Participants will receive the study drug ASP2020 in one eye during an outpatient surgery and will not need to stay in the hospital. Participation will last about 13 to 15 months and includes 12 study visits for eye exams, tests, and other study procedures.
Who Can Participate?
Eligibility
Participants may be able to join if they are at least 12 years old in one part of the study or at least 6 years old in another part and have Stargardt disease or a similar macular eye condition. They cannot have received gene therapy or stem cell therapy in the past, received treatment for thyroid eye disease, or had eye surgery within the past 3 months.
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 ASP2020 for people with Stargardt disease and similar eye conditions. Researchers want to learn if the treatment is safe and whether it can help improve or protect vision.
Locations
Duke University Hospital
Visit Timing
Weekends
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
A Phase 1b, Open-label, Multicenter Dose-escalation Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of a Single Subretinal Dose of ASP2020 in Participants with Macular Dystrophies with a Stargardt-type Clinical Presentation (2020-CL-0101)
Principal Investigator
Oleg
Alekseev
Protocol Number
PRO00121007
Phase
I
Enrollment Status
Pending Open to Enrollment