RECRUITING

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)

Conditions

Stargardt Disease Stargardt Macular Degeneration Stargardt Macular Dystrophy Gene Therapy SB-007 Splicebio Stargardt Stargardts Disease SGDT1

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

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

Quick Facts

Study Start:2025-02-11

Study Completion:2028-12-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06942572

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Provide written consent. Subjects under legal age will also provide informed assent according to guidelines set forth by the same. 2. Are male or female adolescents and adults, aged as follows: 1. In Part A, subjects will be ≥18 to ≤65\* years (inclusive) 2. 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. 3. Are able to understand and comply with the study procedures. 4. 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 5. Clinical evidence consistent with Stargardt Disease type 1. 6. For women of child-bearing potential (WOCBP), have a negative pregnancy test at Screening and, if due to receive active treatment, at Day 0. 7. 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 8. 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. 9. Fulfil visual acuity criteria based on ETDRS letter chart 10. Fulfil baseline lesion size measurement, as measured by the Reading Center 11. Evidence of disease progression as determined by the Medical Monitor following consultation with the Investigator.	1. 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. 2. 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. 3. 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. 4. Have a history of amblyopia in the study eye. 5. Are unwilling to stop taking the following products at Screening and throughout the study: 1. Supplements containing vitamin A or beta-carotene, liver-based products. 2. Prescription oral retinoids. Topical products containing vitamin A or retinoids are not exclusionary. 6. 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. 7. Have received any investigational therapy within 90 days of the Screening Visit or 5 half-lives, whichever is longer. 8. 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. 9. 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. 10. Are an immediate family member (e.g., child, sibling) of the Sponsor or study site personnel.

Inclusion Criteria

Exclusion Criteria

1. Provide written consent. Subjects under legal age will also provide informed assent according to guidelines set forth by the same.
2. Are male or female adolescents and adults, aged as follows:
1. In Part A, subjects will be ≥18 to ≤65\* years (inclusive)
2. 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.
3. Are able to understand and comply with the study procedures.
4. 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
5. Clinical evidence consistent with Stargardt Disease type 1.
6. For women of child-bearing potential (WOCBP), have a negative pregnancy test at Screening and, if due to receive active treatment, at Day 0.
7. 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
8. 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.
9. Fulfil visual acuity criteria based on ETDRS letter chart
10. Fulfil baseline lesion size measurement, as measured by the Reading Center
11. Evidence of disease progression as determined by the Medical Monitor following consultation with the Investigator.

1. 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.
2. 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.
3. 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.
4. Have a history of amblyopia in the study eye.
5. Are unwilling to stop taking the following products at Screening and throughout the study:
1. Supplements containing vitamin A or beta-carotene, liver-based products.
2. Prescription oral retinoids. Topical products containing vitamin A or retinoids are not exclusionary.
6. 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.
7. Have received any investigational therapy within 90 days of the Screening Visit or 5 half-lives, whichever is longer.
8. 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.
9. 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.
10. Are an immediate family member (e.g., child, sibling) of the Sponsor or study site personnel.

Contacts and Locations

Study Contact

SpliceBio

CONTACT

+34 934 02 04 56

clinicaltrials@splice.bio

Study Locations (Sites)

UCHealth Sue Anschutz-Rodgers Eye Center,

Aurora, Colorado, 80045

United States

Bascom Palmer Eye Institute

Miami, Florida, 33136

United States

Massachusetts Eye and Ear Infirmary

Boston, Massachusetts, 02114

United States

Oregon Health & Science University

Portland, Oregon, 97239

United States

Retina Foundation of the Southwest

Dallas, Texas, 75261

United States

Collaborators and Investigators

Sponsor: Splice Bio

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-02-11

Study Completion Date2028-12-31

Study Record Updates

Study Start Date2025-02-11

Study Completion Date2028-12-31

Terms related to this study

Keywords Provided by Researchers

Gene Therapy
SB-007
Splicebio
Stargardt
Stargardts Disease
SGDT1

Additional Relevant MeSH Terms

Stargardt Disease
Stargardt Macular Degeneration
Stargardt Macular Dystrophy