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)

NCT06942572 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: SB-007 CS-1012024-519535-42
• Study Type: interventional
• Target: 57
• Locations: 1 country
• Sites: 5

Brief 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.

Conditions

Stargardt Disease; Stargardt Macular Degeneration; Stargardt Macular Dystrophy

Keywords

Gene Therapy; SB-007; Splicebio; Stargardt; Stargardts Disease; SGDT1

Outcome Measures

Primary Outcomes (1)

• Safety Measures

  Safety and tolerability assessed by incidence and/or clinically significant changes in ocular and non-ocular AEs

  96 weeks

Secondary Outcomes (6)

• Efficacy Measure: Change from baseline in lesion size using autofluorescence (FAF)

  96 weeks (Screening to 96 weeks post SB-007 administration)

• Other Efficacy Measure: Change from baseline in retinal sensitivity

  48 and 96 weeks (Screening to 48 and 96 weeks post SB-007 administration)

• Other Efficacy Measure: Change from baseline in BCVA (Best Corrected Visual Acuity)

  48 and 96 weeks (Screening to 48 and 96 weeks post SB-007 administration)

• Other Efficacy Measure: Change from baseline in LLVA (Low Luminance Visual Acuity)

  48 and 96 weeks (Screening to 48 and 96 weeks post SB-007 administration)

• Other Efficacy Measure: Change in Quality of Life measure using MRDQ

  48 and 96 weeks (Screening to 48 and 96 weeks post SB-007 administration)

Study Arms (6)

Phase 1 Dose Escalation - Low Dose

EXPERIMENTAL

Low Dose: Subjects will receive subretinal injection of SB-007 in the low dose group

Genetic: SB-007

Phase 1 Dose Escalation - Medium Dose

EXPERIMENTAL

Medium Dose: Subjects will receive subretinal injection of SB-007 in the medium dose group

Genetic: SB-007

Phase 1 Dose Escalation - High Dose

EXPERIMENTAL

High Dose: Subjects will receive subretinal injection of SB-007 in the high dose group

Genetic: SB-007

Phase 2 Dose Expansion: Dose 1 from Phase 1 Randomised Arm

EXPERIMENTAL

Subjects will receive a subretinal injection of SB-007 with Maximum tolerated dose (MTD) from Phase 1

Genetic: SB-007

Phase 2 Dose Expansion: Dose 2 from Phase 1 Randomised Arm

EXPERIMENTAL

Subjects will receive a subretinal injection of SB-007 with lower dose than Maximum tolerated dose (MTD) from Phase 1

Genetic: SB-007

Phase 2 No Intervention - Randomised Control Arm

NO INTERVENTION

No Intervention Control Arm: Subject will not receive any active study intervention

Interventions

SB-007 GENETIC

Subretinal Administration of SB-007

Phase 1 Dose Escalation - High Dose; Phase 1 Dose Escalation - Low Dose; Phase 1 Dose Escalation - Medium Dose; Phase 2 Dose Expansion: Dose 1 from Phase 1 Randomised Arm; Phase 2 Dose Expansion: Dose 2 from Phase 1 Randomised Arm

Eligibility Criteria

• Age: 12 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Subjects must be excluded from participating in the study if they:
• 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.
• 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.
• Have received any investigational therapy within 90 days of the Screening Visit or 5 half-lives, whichever is longer.
• Have known serious allergies to the fluorescein dye that might be used to measure IOP, ocular dilating drops, topical ocular anesthetic, steroid medication, or components of the SB-007 formulation.
• Have any significant ocular or non-ocular disease/disorder which, in the opinion of the Investigator and with concurrence of the Medical Monitor, may either put the subject at risk because of participation in the study, may influence the results of the study, or affect the subject's ability to participate in the study.
• Are an immediate family member (e.g., child, sibling) of the Sponsor or study site personnel.

Sponsors & Collaborators

• Splice Bio: lead

Study Sites (5)

UCHealth Sue Anschutz-Rodgers Eye Center,

Aurora, Colorado, 80045, United States

RECRUITING

Bascom Palmer Eye Institute

Miami, Florida, 33136, United States

RECRUITING

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, 02114, United States

RECRUITING

Oregon Health & Science University

Portland, Oregon, 97239, United States

RECRUITING

Retina Foundation of the Southwest

Dallas, Texas, 75261, United States

RECRUITING

Related Links

• SpliceBio Website

MeSH Terms

Conditions

Stargardt Disease

Condition Hierarchy (Ancestors)

Eye Diseases, Hereditary; Eye Diseases; Macular Degeneration; Retinal Degeneration; Retinal Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

SpliceBio

CONTACT

+34 934 02 04 56; clinicaltrials@splice.bio

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Non-Randomised Dose Escalation (Part A) is fully unmasked. Randomised Dose Expansion Cohort (Part B) masked to Study Dose.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Non-Randomised Dose Escalation (Part A) followed by Randomised Dose Expansion (Part B).

Study Record Dates

• First Submitted: February 18, 2025
• First Posted: April 24, 2025
• Study Start: February 11, 2025
• Primary Completion (Estimated): October 31, 2028
• Study Completion (Estimated): December 31, 2028
• Last Updated: April 29, 2025

Record last verified: 2025-04

Locations

US (5)