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.
Provide written consent. Subjects under legal age will also provide informed assent according to guidelines set forth by the same.
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 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.
Sequential
SB-007
GeneticSubretinal Administration of SB-007
6
Treatment groupsSee each treatment group below.
Safety Measures
Safety and tolerability assessed by incidence and/or clinically significant changes in ocular and non-ocular AEs
Efficacy Measure: Change from baseline in lesion size using autofluorescence (FAF)
Change from baseline in lesion size growth (mm\^2) on FAF imaging compared between SB-007 and untreated control at Week 96, adjudicated by a Central Reading Center
Other Efficacy Measure: Change from baseline in retinal sensitivity
Change from baseline in retinal sensitivity based on microperimetry at Weeks 48 and 96
Other Efficacy Measure: Change from baseline in BCVA (Best Corrected Visual Acuity)
Change from baseline in BCVA using the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA) letter score at Weeks 48 and 96
Other Efficacy Measure: Change from baseline in LLVA (Low Luminance Visual Acuity)
Change from baseline in LLVA using the Early Treatment Diabetic Retinopathy Study (ETDRS) chart at Weeks 48 and 96. A higher score represents better vision at low light levels.
