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ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 ENCODING THE ABCA4 PROTEIN, C-REGION

Clinical trials are investigating ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 ENCODING THE ABCA4 PROTEIN, C-REGION in people with Stargardt disease. The study aims to assess safety, tolerability, and early signs of benefit, and it focuses on subjects with Stargardt disease type 1 and bi-allelic ABCA4 gene mutations.

Table of contents

Trial overview

The study titled A clinical study to Evaluate the Safety, Tolerability and Preliminary Efficacy of SB-007 in Subjects with Stargardt Disease is an interventional trial of ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 ENCODING THE ABCA4 PROTEIN, C-REGION.[1] It is designed to evaluate safety, tolerability, and preliminary efficacy in people with Stargardt disease type 1 who are confirmed to have bi-allelic ABCA4 gene mutations.[1]

Who can participate

The trial is for subjects with Stargardt Disease, specifically Stargardt disease type 1 (STGD1).[1] The study also requires genetic confirmation of bi-allelic ABCA4 gene mutations, which means both copies of the gene must have changes.[1]

This means the study is not for all people with eye disease; it is focused on a narrower group with a specific inherited form of Stargardt disease.[1]

What is being measured

The primary endpoint is safety and tolerability through Week 96.[1] The study measures this by looking at incidence and clinically significant changes in ocular and non-ocular adverse events, which are unwanted medical problems in the eye and elsewhere in the body.[1]

The study also aims to collect preliminary efficacy information, which means early signs of possible benefit rather than final proof that the treatment works.[1] This information is being used to help determine dose selection in the study population.[1]

Trial phase and status

This is a Phase 1/2 trial, which is an early stage of clinical research.[1] Early phase studies usually focus first on safety and tolerability, while also collecting early signals about possible benefit.[1]

The trial status is Authorised.[1]

Study size and design

The planned enrollment is 86 participants.[1] The study type is Interventional, meaning participants receive the study treatment as part of the research plan.[1] The intervention listed is SB-007 (SUBRETINAL USE).[1]

The source data do not provide more details about randomization, masking, or comparison groups, so those features cannot be described here.[1]

Patient-friendly terms

Subretinal means under the retina, the light-sensing layer at the back of the eye.[1] Adverse events are medical problems that happen during a study, and they may or may not be caused by the treatment.[1]

Genetic confirmation means the study uses gene testing to make sure a person has the specific ABCA4 changes needed for the trial.[1] Preliminary efficacy means the study is looking for early signs that the treatment may help, but the results are not yet final.[1]

Trial ID Phase Condition studied Status Enrollment
2024-519535-42-00 Phase 1/2 Stargardt Disease Authorised 86

FAQ

  • What is being studied in these trials? The trial is studying ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 ENCODING THE ABCA4 PROTEIN, C-REGION as SB-007 given by subretinal use. It is being tested to see whether it is safe, tolerable, and may show early signs of benefit in Stargardt disease.
  • Who can take part in the study? The study is for subjects with Stargardt disease type 1. People must also be confirmed by genetic testing to have bi-allelic ABCA4 gene mutations, which means changes in both copies of the ABCA4 gene.
  • What phase is the trial? This is a Phase 1/2 trial. Phase 1/2 studies usually look at safety first and also gather early information on whether the treatment may help.
  • What condition is the trial focused on? The condition studied is Stargardt Disease, specifically Stargardt disease type 1. This is the disease named in the trial record and the population the study is designed for.
  • What is the main outcome being measured? The main outcome is safety and tolerability through Week 96. The study looks at ocular and non-ocular adverse events, which means side effects in the eye and in the rest of the body.

Ongoing Clinical Trials on ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 ENCODING THE ABCA4 PROTEIN, C-REGION

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

    Not yet recruiting

    1 1
    Investigated diseases:
    Investigated drugs:
    Belgium Germany

Glossary

  • Stargardt disease: An inherited eye disease that affects the retina, the light-sensing part of the eye.
  • Stargardt disease type 1 (STGD1): A specific form of Stargardt disease named in the study. The trial focuses on this type.
  • ABCA4 gene: A gene that is checked in this study. The trial includes people with changes in both copies of this gene.
  • Bi-allelic mutation: A change in both copies of a gene. In this study, it means both ABCA4 gene copies must be affected.
  • Genotypically confirmed: Confirmed by genetic testing, not just by symptoms or eye exam.
  • Phase 1/2: An early study phase that checks safety and tolerability and also gathers early signs of whether the treatment may work.
  • Safety: How well a treatment can be given without causing harmful problems.
  • Tolerability: How well people can handle a treatment and its effects.
  • Preliminary efficacy: Early signs that a treatment may help, but not yet proof that it works.
  • Subretinal use: Given under the retina, which is the light-sensing layer at the back of the eye.
  • Adverse events (AEs): Unwanted medical problems that happen during a study. They may or may not be caused by the treatment.

References

  1. https://clinicaltrials.gov/study/2024-519535-42-00