Actively Recruiting
A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal SB-007 in Subjects With Stargardt Disease Caused by ABCA4 Gene Mutations
Led by Splice Bio · Updated on 2025-04-29
57
Participants Needed
5
Research Sites
8 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate the safety, tolerability, and preliminary effectiveness of SB-007, a gene therapy delivered by subretinal injection, in people with Stargardt Disease type 1 (STGD1). STGD1 is an inherited retinal disorder caused by mutations in the ABCA4 gene leading to vision loss, typically starting in childhood or early adulthood. The study will enroll about 57 participants and follow them for 96 weeks after treatment to determine the best dose of SB-007. Participants will receive a single subretinal injection of SB-007. The study includes several groups receiving different doses: low, medium, high, and doses based on the maximum tolerated dose (MTD) identified in earlier phases. There is also a control group that will not receive the active treatment. SB-007 uses a novel dual AAV8 gene delivery method aiming to restore the full-length ABCA4 protein in the retina to potentially slow vision loss. During the study, participants will undergo regular safety checks and eye examinations over 96 weeks. Assessments include measuring changes in lesion size, retinal sensitivity, visual acuity under normal and low light, and quality of life through questionnaires. Researchers will monitor safety and treatment effects closely to understand how SB-007 works and its impact on disease progression. The total study duration includes treatment and follow-up.
CONDITIONS
Brief 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 and assent if under legal age
- Male or female adolescents and adults aged 12 to 65 years (older than 65 may be eligible with approval)
- Able to understand and comply with study procedures
- Diagnosis of Stargardt Disease type 1 caused by bi-allelic pathogenic or likely pathogenic variants in the ABCA4 gene confirmed by genetic testing
- Clinical evidence consistent with Stargardt Disease type 1
- Negative pregnancy test for women of child-bearing potential at screening and Day 0 if receiving treatment
- Agree to use strict abstinence or acceptable contraception for 3 months from Day 0 if sexually active
- Clear ocular media and adequate pupillary dilation in the study eye to allow good quality retinal imaging
- Meet visual acuity criteria based on ETDRS letter chart
- Meet baseline lesion size measurement as determined by the Reading Center
- Evidence of disease progression as determined by the Medical Monitor with Investigator consultation
You will not qualify if you...
- Had intraocular surgery or thermal laser within 90 days before screening or planned during the study in the study eye
- Had major surgery within 30 days before screening or planned during the study
- Have pathogenic variants in IRD genes other than ABCA4 or dominant/X-linked IRD genes
- History of amblyopia in the study eye
- Unwilling to stop vitamin A or beta-carotene supplements and prescription oral retinoids during the study
- Previous ophthalmic gene therapy, stem cell therapy, retinal chip implantation, or relevant intraocular injections
- Received investigational therapy within 90 days or 5 half-lives before screening
- Known serious allergies to fluorescein dye, ocular dilating drops, anesthetics, steroids, or SB-007 components
- Significant ocular or non-ocular disease that may pose risk or affect participation or results
- Immediate family member of the sponsor or study site personnel
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Single administration with follow-up assessments up to 96 weeks
Participants receive a subretinal injection of SB-007 at one of several dose levels to evaluate safety and preliminary efficacy.
Multiple visits including baseline and follow-ups up to 96 weeks
Duration - Up to 96 weeks post-treatment
Participants are monitored for safety, vision changes, and treatment effects after receiving SB-007.
Regular follow-up visits over 96 weeks
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
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
6
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here