Purpose of this Study
We are doing this study to find out if an experimental drug called tinlarebant is a safe and effective option for people with Stargardt Disease.
Who Can Participate?
Eligibility
Children and adults ages 12-20 who are diagnosed with Stargardt Disease.
For more information, contact the study team at <a href= "mailto: sim15@duke.edu">sim15@duke.edu.</a>
For more information, contact the study team at <a href= "mailto: sim15@duke.edu">sim15@duke.edu.</a>
Age Range
12-20
Sex/Genders
Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No
What is Involved?
If you choose to join this study, you will get a random assignment (like a coin flip) to either take a 5 mg dose of the study drug or a placebo (inactive substance with no drug in it) every day for 24 months. There is a screening period that lasts about 4 weeks to make sure you are eligible to take part in the study. You will visit our clinic for check-ups about 10 times over the course of the study.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
No
Spanish Materials Available
No
Study Details
Full Title
A Phase 1b Open-laben Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Tinlarebant in Japanese Subjects with Stargardt Disease and a Phase 2/3 Randomized, Double-masked, and Placebo-controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Tinlarebant in Subjects with Stargardt Disease - DRAGON - LBS-008-CT07
Principal Investigator
Oleg
Alekseev
Protocol Number
PRO00115629
Phase
I
Enrollment Status
Pending Open to Enrollment