OCU410ST-101 (Stargardt Disease) - Clinical Trial

What is the 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).

What is the Condition Being Studied?

Stargardt Disease (STGD)

Who Can Participate in the Study?

Children and adults ages 6-65 who:

  • Are diagnosed with STGD
  • Have documented evidence of macular atrophy
  • Have never received any of the following therapies: gene therapy, stem cell therapy, surgical implantation of retinal chips, intravitreal or sub-retinal injections

For more information about who can be in this study, please contact the study team at DEC-RA@duke.edu.

Age Group
Adults, Children

What is Involved?

If you are an adult and choose to join the study, you will receive:

  • Either a low dose, or a medium dose, or a high dose of the study drug; OR
  • You will receive a placebo (inactive substance with no drug in it) and be in the control group

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.

Study Details

Full Title
OCU410ST-101: A Phase 1/2 Study to Assess the Safety and Efficacy of OCU410ST for Stargardt Disease.
Principal Investigator
Protocol Number
IRB: PRO00114399
NCT: NCT05956626
Phase I/II
Enrollment Status
Open for Enrollment