A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability … (NCT06942572) | Clinical Trial Compass
RecruitingPhase 1/2
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)
United States57 participantsStarted 2025-02-11
Plain-language 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.
Who can participate
Age range12 Years – 65 Years
SexALL
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Inclusion 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.
Exclusion criteria
✕. Have had any intraocular surgery (including cataract surgery) or thermal laser within 90 days of the Screening Visit or planned intraocular surgery (including cataract surgery) or thermal laser during the period of the study, in the study eye.
✕. Have had any major surgical procedure within 30 days of the Screening Visit or planned or anticipated major surgery during the period of the study.
. Have two pathogenic or likely pathogenic variants in IRD genes (other than ABCA4) or a single pathogenic or likely pathogenic variant in autosomal dominant or X-linked IRD genes.
✕. Have a history of amblyopia in the study eye.
✕. Are unwilling to stop taking the following products at Screening and throughout the study:
✕. Supplements containing vitamin A or beta-carotene, liver-based products.
✕. Prescription oral retinoids. Topical products containing vitamin A or retinoids are not exclusionary.
✕. Have any ophthalmic history of gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or intravitreal or sub-retinal or supra-choroidal injections.