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Evaluation of SB-007 Safety and Efficacy in Patients with Stargardt Disease Type 1 (STGD1) Caused by ABCA4 Gene Mutations

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is investigating a treatment for Stargardt Disease type 1 (STGD1), a genetic eye disorder caused by mutations in the ABCA4 gene. Stargardt disease typically leads to progressive vision loss due to the degeneration of cells in the central part of the retina. The study will test a medication called SB-007, which is administered through a subretinal injection (an injection under the retina of the eye). The purpose of this study is to evaluate whether SB-007 is safe, well-tolerated, and potentially effective for people with genetically confirmed Stargardt disease.

The study is designed as a combined Phase 1/2 trial, which means it will first determine if the treatment is safe at different dose levels, and then assess whether it shows signs of effectiveness. Participants will receive either the SB-007 treatment in one eye or serve as a control group. Throughout the study, researchers will monitor participants' eye health using various imaging techniques such as fundus autofluorescence (FAF) and optical coherence tomography (OCT), which provide detailed pictures of the retina. They will also assess changes in vision using different vision tests that measure visual acuity and contrast sensitivity.

The study will follow participants for 96 weeks (about 2 years) after treatment to determine the long-term safety and potential benefits of SB-007. During this time, participants will attend regular check-ups where doctors will examine their eyes and assess any changes in their vision and the progression of the disease. The trial specifically targets adults between 18 and 65 years of age who have genetically confirmed Stargardt disease with mutations in both copies of the ABCA4 gene.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Screening and Initial Assessment

    You will undergo comprehensive eye examinations to confirm your Stargardt Disease Type 1 diagnosis and establish baseline measurements.

    Tests will include fundus autofluorescence (FAF) imaging to measure lesion size, spectral-domain optical coherence tomography (SD-OCT) to assess the retinal layers, microperimetry to measure retinal sensitivity, and various vision tests.

    Vision tests include best-corrected visual acuity (BCVA) using standard eye charts, low luminance visual acuity (LLVA), and contrast sensitivity assessments.

  2. Step 2

    Randomization

    You will be randomly assigned to either receive the SB-007 treatment or be in the untreated control group.

    This is an open-label study, which means you and your doctor will know which group you are in, but some assessors will be masked (unaware) of your treatment assignment.

  3. Step 3

    Treatment Day (Day 0)

    If assigned to the treatment group, you will receive a subretinal injection of SB-007, which contains an adeno-associated virus vector that delivers the ABCA4 gene to the retina.

    SB-007 consists of two components: adeno-associated virus vector serotype 8 encoding the ABCA4 protein N-region and C-region.

    The procedure will be performed under appropriate anesthesia and sterile conditions.

    Women of childbearing potential will have a pregnancy test on this day.

  4. Step 4

    Follow-up Period (96 Weeks)

    You will attend regular follow-up visits over 96 weeks (approximately 2 years) to monitor safety and assess the effectiveness of the treatment.

    During these visits, you will undergo repeated eye examinations similar to those performed at baseline.

    The primary focus will be on monitoring for any adverse events (AEs) related to the treatment or procedure.

    The doctors will track changes in your eye condition by measuring lesion size growth, retinal layer integrity, visual function, and other parameters compared to your baseline measurements.

  5. Step 5

    Safety Monitoring

    Throughout the study, you will be monitored for both ocular (eye-related) and non-ocular adverse events.

    Regular eye examinations will check for any changes in your eye health following the treatment.

    For safety reasons, you must use acceptable contraception for 4 months from Day 0 if you are sexually active (applies to both men and women of childbearing potential).

  6. Step 6

    Efficacy Assessments

    The study will measure treatment effectiveness by comparing changes in your eye condition from baseline to various timepoints over the 96-week period.

    Key measurements include changes in lesion size on fundus autofluorescence imaging, integrity of retinal layers on OCT, sensitivity of your retina using microperimetry, and various vision tests.

    Your results will be compared to either your baseline measurements or to the untreated control group to determine if SB-007 is providing any benefit.

  7. Step 7

    Study Completion

    After completing the 96-week (approximately 2-year) follow-up period, you will have a final comprehensive assessment.

    This final assessment will document your eye condition and overall health status at the end of the study period.

    The study is expected to run until September 2028, though your individual participation will depend on when you join the study.

Who can join the trial?

8 criteria

  • You must provide written consent to participate in the study.
  • You must be between 18 and 65 years old (including ages 18 and 65).
  • You need to be able to understand and follow the study procedures.
  • You must have a diagnosis of Stargardt disease type 1 (STGD1).
  • You must show clinical signs that are consistent with Stargardt Disease type 1.
  • If you are a woman who can become pregnant, you must have a negative pregnancy test during screening and on Day 0 if you're receiving active treatment.
  • If you are a woman who can become pregnant or a man (or have a female partner who can become pregnant), you must agree to either strict abstinence or use acceptable birth control for 4 months from Day 0 if sexually active.
  • Your study eye must have clear eye tissue and adequate pupil dilation, including no allergies to dilating eye drops, to allow for good quality retinal imaging (pictures of the back of your eye).

Who cannot join the trial?

14 criteria

  • Having ophthalmic conditions other than Stargardt Disease Type 1 that may affect vision
  • Not having confirmed mutations in both copies of the ABCA4 gene (which causes Stargardt Disease)
  • Having had an eye surgery within 6 months before the study
  • Having uncontrolled glaucoma (high pressure in the eye that can damage the optic nerve)
  • Having an active eye infection or inflammation
  • Having a history of certain eye conditions like retinal detachment (when the retina pulls away from its normal position)
  • Being pregnant or breastfeeding
  • Having a history of cancer in the past 5 years (except certain skin cancers)
  • Having serious medical conditions that would make participation unsafe
  • Having a history of drug or alcohol abuse
  • Having participated in another clinical trial within 30 days
  • Being unable to comply with study procedures or follow-up visits
  • Having known allergies to the study medication or its components
  • Using medications that could interfere with the study treatment
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Investigated drugs

SB-007 is a treatment administered through subretinal injection (an injection given under the retina of the eye). It is being studied for people with Stargardt disease type 1, which is a genetic eye disorder caused by mutations in the ABCA4 gene. This treatment is experimental and aims to address the underlying genetic cause of the disease, which affects vision.

What is already known about the treatment

SB-007 – This is an investigational therapy administered via subretinal injection directly into the eye for treatment of Stargardt Disease Type 1 (STGD1), a genetic eye disorder caused by mutations in the ABCA4 gene. Currently in Phase 1/2 clinical trials, SB-007 is being evaluated for safety, tolerability, and preliminary effectiveness in patients with confirmed bi-allelic ABCA4 gene mutations. While the exact molecular mechanism has not been fully disclosed in the provided information, this therapy represents a potential targeted approach for this rare inherited retinal disease that typically leads to progressive vision loss. The ASTRA study is assessing different dose levels to determine the optimal therapeutic dose for future development.

Investigated diseases

Stargardt Disease Stargardt disease is an inherited retinal disorder characterized by progressive vision loss due to degeneration of the macula, the central part of the retina responsible for sharp, central vision. It typically begins in childhood or adolescence, resulting in gradual deterioration of central vision while peripheral vision usually remains intact. The condition is caused by mutations in the ABCA4 gene, which lead to the accumulation of lipofuscin (a fatty substance) in the retinal pigment epithelium cells. As the disease progresses, characteristic yellowish flecks appear in the retina, and the macula develops atrophic lesions visible on imaging. Patients experience increasing difficulty with reading, recognizing faces, and other tasks requiring detailed vision.
Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase I/IITrial ID2024-519535-42-00Protocol codeSB-007 CS-101Estimated enrolment86 patientsSponsorSplicebio S.L.

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