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

Status: Recruiting
Location: See all (5) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 1/Phase 2
SUMMARY

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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 12
Maximum Age: 65
Healthy Volunteers: f
View:

• To be eligible for study participation, subjects must meet the following criteria:

• Provide written consent. Subjects under legal age will also provide informed assent according to guidelines set forth by the same.

• Are male or female adolescents and adults, aged as follows:

∙ In Part A, subjects will be ≥18 to ≤65\* years (inclusive)

‣ In Part B, subject age is planned as ≥12 to ≤65\* years (inclusive) \*Subjects aged \>65 years may be eligible in Parts A and B, following discussion with, and approval by, the Medical Monitor.

• Are able to understand and comply with the study procedures.

• Have a diagnosis of STGD1 caused by bi-allelic pathogenic, or likely pathogenic, variants in the ABCA4 gene confirmed genotypically by an accredited genetic testing laboratory

• Clinical evidence consistent with Stargardt Disease type 1.

• For women of child-bearing potential (WOCBP), have a negative pregnancy test at Screening and, if due to receive active treatment, at Day 0.

• For both WOCBP and male subjects (or their female partners who are of child-bearing potential), agree to either strict abstinence or, if sexually active, use an acceptable contraception measure for 3 months from Day 0

• Must have clear ocular media and adequate pupillary dilation in the study eye, including no allergy to dilating eyedrops, to permit good quality retinal imaging.

• Fulfil visual acuity criteria based on ETDRS letter chart

⁃ Fulfil baseline lesion size measurement, as measured by the Reading Center

⁃ Evidence of disease progression as determined by the Medical Monitor following consultation with the Investigator.

Locations
United States
Colorado
UCHealth Sue Anschutz-Rodgers Eye Center,
RECRUITING
Aurora
Florida
Bascom Palmer Eye Institute
RECRUITING
Miami
Massachusetts
Massachusetts Eye and Ear Infirmary
RECRUITING
Boston
Oregon
Oregon Health & Science University
RECRUITING
Portland
Texas
Retina Foundation of the Southwest
RECRUITING
Dallas
Contact Information
Primary
SpliceBio
clinicaltrials@splice.bio
+34 934 02 04 56
Time Frame
Start Date: 2025-02-11
Estimated Completion Date: 2028-12-31
Participants
Target number of participants: 57
Treatments
Experimental: Phase 1 Dose Escalation - Low Dose
Low Dose: Subjects will receive subretinal injection of SB-007 in the low dose group
Experimental: Phase 1 Dose Escalation - Medium Dose
Medium Dose: Subjects will receive subretinal injection of SB-007 in the medium dose group
Experimental: Phase 1 Dose Escalation - High Dose
High Dose: Subjects will receive subretinal injection of SB-007 in the high dose group
Experimental: Phase 2 Dose Expansion: Dose 1 from Phase 1 Randomised Arm
Subjects will receive a subretinal injection of SB-007 with Maximum tolerated dose (MTD) from Phase 1
Experimental: Phase 2 Dose Expansion: Dose 2 from Phase 1 Randomised Arm
Subjects will receive a subretinal injection of SB-007 with lower dose than Maximum tolerated dose (MTD) from Phase 1
No_intervention: Phase 2 No Intervention - Randomised Control Arm
No Intervention Control Arm: Subject will not receive any active study intervention
Sponsors
Leads: Splice Bio

This content was sourced from clinicaltrials.gov

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