Actively Recruiting
A Phase 1/2, First-in-Human 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)
Led by Splice Bio · Updated on 2025-04-29
57
Participants Needed
5
Research Sites
202 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This Phase 1/2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1). This is a multicenter study which will enroll approximately 57 subjects, followed up over a 96 week period post treatment after a single administration of SB-007.
CONDITIONS
Official Title
A Phase 1/2, First-in-Human 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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Provide written consent or assent if under legal age
- Male or female aged 18 to 65 years in Part A; 12 to 65 years in Part B (older than 65 may be eligible with approval)
- Able to understand and comply with study procedures
- Diagnosed with Stargardt Disease type 1 caused by bi-allelic pathogenic or likely pathogenic ABCA4 gene variants confirmed by genetic testing
- Clinical signs consistent with Stargardt Disease type 1
- Women of child-bearing potential must have a negative pregnancy test at screening and Day 0 if treated
- Agree to strict abstinence or use acceptable contraception for 3 months after Day 0 for both sexes
- Clear ocular media and adequate pupil dilation in the study eye without allergy to dilating drops
- Meet visual acuity criteria based on ETDRS letter chart
- Meet baseline lesion size criteria as measured by the Reading Center
- Show evidence of disease progression as determined by the Medical Monitor after consultation
You will not qualify if you...
- Had intraocular surgery or thermal laser in study eye within 90 days before screening or planned during study
- Had major surgery within 30 days before screening or planned during study
- Have pathogenic variants in other IRD genes besides ABCA4 or a single dominant/X-linked IRD gene variant
- History of amblyopia in the study eye
- Unwilling to stop taking vitamin A or beta-carotene supplements, liver-based products, or prescription oral retinoids during study
- History of ophthalmic gene therapy, stem cell therapy, retinal chip implantation, or intraocular injections
- Received investigational therapy within 90 days before screening or 5 half-lives, whichever is longer
- Known serious allergies to fluorescein dye, dilating drops, ocular anesthetic, steroid medication, or SB-007 components
- Significant ocular or non-ocular disease that may risk participation or affect study results
- Immediate family member of sponsor or study site personnel
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 5 locations
1
UCHealth Sue Anschutz-Rodgers Eye Center,
Aurora, Colorado, United States, 80045
Actively Recruiting
2
Bascom Palmer Eye Institute
Miami, Florida, United States, 33136
Actively Recruiting
3
Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, United States, 02114
Actively Recruiting
4
Oregon Health & Science University
Portland, Oregon, United States, 97239
Actively Recruiting
5
Retina Foundation of the Southwest
Dallas, Texas, United States, 75261
Actively Recruiting
Research Team
S
SpliceBio
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
6
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