Purpose of this Study
We are doing this study to find out if an experimental gene transfer drug called OCU410ST is a safe and effective option for Stargardt Disease (STGD).
Who Can Participate?
Eligibility
Children and adults ages 6-65 who:<ul>
<li>Are diagnosed with STGD</li>
<li>Have documented evidence of macular atrophy</li>
<li>Have never received any of the following therapies: gene therapy, stem cell therapy, surgical implantation of retinal chips, intravitreal or sub-retinal injections</li></ul>
For more information about who can be in this study, please contact the study team at <a href= "mailto: DEC-RA@duke.edu">DEC-RA@duke.edu.</a>
<li>Are diagnosed with STGD</li>
<li>Have documented evidence of macular atrophy</li>
<li>Have never received any of the following therapies: gene therapy, stem cell therapy, surgical implantation of retinal chips, intravitreal or sub-retinal injections</li></ul>
For more information about who can be in this study, please contact the study team at <a href= "mailto: DEC-RA@duke.edu">DEC-RA@duke.edu.</a>
Age Range
6-75
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 are an adult and choose to join the study, you will receive:<ul>
<li>Either a low dose, or a medium dose, or a high dose of the study drug; OR</li>
<li>You will receive a placebo (inactive substance with no drug in it) and be in the control group</li></ul>
If you are a child and choose to join the study, you will receive a dose of the study drug or you will receive a placebo. Children in the study will all receive the same dose of the study drug.
You will be in touch with the study team for about 15 months and you will visit our clinic at the Duke Eye Center up to 8 times. At various visits, you will have blood tests, eye exams, and vision tests.
<li>Either a low dose, or a medium dose, or a high dose of the study drug; OR</li>
<li>You will receive a placebo (inactive substance with no drug in it) and be in the control group</li></ul>
If you are a child and choose to join the study, you will receive a dose of the study drug or you will receive a placebo. Children in the study will all receive the same dose of the study drug.
You will be in touch with the study team for about 15 months and you will visit our clinic at the Duke Eye Center up to 8 times. At various visits, you will have blood tests, eye exams, and vision tests.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
OCU410ST-101: A Phase 1/2 Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease.
Principal Investigator
Ramiro
Maldonado
Protocol Number
PRO00114399
NCT ID
NCT05956626
Phase
I/II
Enrollment Status
Open to Enrollment