OCU410ST-101 (Stargardt Disease)

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:
  • 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 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?

Description

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.

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
ClinicalTrials.gov