Evaluating a new gene therapy for Stargardt Disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1/2 clinical trial aims to assess the safety, tolerability, and preliminary efficacy of a gene therapy called SB-007 administered via subretinal injection in patients with Stargardt Disease Type 1 (STGD1). The study will enroll approximately 57 participants and will follow them for 96 weeks after a single treatment to monitor outcomes. The trial is designed to determine the appropriate dosage and gather initial data on the therapy's effectiveness in slowing or reversing vision loss associated with this inherited retinal dystrophy.

Why it matters

Who can join: the full rules

Inclusion Criteria:

To be eligible for study participation, subjects must meet the following 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.

Exclusion Criteria:

Subjects must be excluded from participating in the study if they:

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.

Where it's running

• UCHealth Sue Anschutz-Rodgers Eye Center,
  Aurora, Colorado, United States Recruiting
• Bascom Palmer Eye Institute
  Miami, Florida, United States Recruiting
• Massachusetts Eye and Ear Infirmary
  Boston, Massachusetts, United States Recruiting
• Oregon Health & Science University
  Portland, Oregon, United States Recruiting
• Retina Foundation of the Southwest
  Dallas, Texas, United States Recruiting

Study contacts

• Study coordinator: SpliceBio
• Email: clinicaltrials@splice.bio
• Phone: +34 934 02 04 56

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Single intravitreal RTx-021 injection for Stargardt disease

  This will test whether a single injection of RTx-021 into one eye is safe and may help people aged 16 and older with Stargardt disease.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.