Purpose of this Study
We are doing this study to find out if an experimental drug called SB-007 (the stud drug) is a safe and effective option for people with STGD1.
Who Can Participate?
Eligibility
Children and adults ages 12-65 who:
- Are diagnosed with STGD1
- Have no history of amblyopia (lazy eye)
Age Range
12-65
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 choose to join this study, you will get a single injection of the study drug directly into the eye.
Participation in the study will last for up to 8 years and involve about 11 visits to our clinic. During these visits, you will have eye exams, blood tests, and fill out surveys.
Locations
Duke University Hospital
Visit Timing
Weekdays
Compensation
Yes
Spanish Materials Available
No
Study Details
Full Title
A Phase 1/2, First-in-Human, Open-label, Assessor-Masked, Randomized, Controlled, Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects with Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene (ASTRA) - SB-007 CS-101
Principal Investigator
Ramiro
Maldonado
Protocol Number
PRO00116715
Phase
I/II
Enrollment Status
Open to Enrollment