In short
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.
Key points
- Clinical trials are studying ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 ENCODING THE ABCA4 PROTEIN, C-REGION in Stargardt disease. The available trial is an interventional Phase 1/2 study called SB-007 and it is authorised. It focuses on people with Stargardt disease type 1 who have bi-allelic ABCA4 gene mutations confirmed by genetic testing. The study is looking at safety, tolerability, and early signs of benefit. The main endpoint is safety and tolerability through Week 96, measured by eye and body adverse events. The planned enrollment is 86 participants.
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. 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.
Who can participate
The trial is for subjects with Stargardt Disease, specifically Stargardt disease type 1 (STGD1). The study also requires genetic confirmation of bi-allelic ABCA4 gene mutations, which means both copies of the gene must have changes.
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.
What is being measured
The primary endpoint is safety and tolerability through Week 96. 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.
The study also aims to collect preliminary efficacy information, which means early signs of possible benefit rather than final proof that the treatment works. This information is being used to help determine dose selection in the study population.
Trial phase and status
This is a Phase 1/2 trial, which is an early stage of clinical research. Early phase studies usually focus first on safety and tolerability, while also collecting early signals about possible benefit.
The trial status is Authorised.
Study size and design
The planned enrollment is 86 participants. The study type is Interventional, meaning participants receive the study treatment as part of the research plan. The intervention listed is SB-007 (SUBRETINAL USE).
The source data do not provide more details about randomization, masking, or comparison groups, so those features cannot be described here.
Patient-friendly terms
Subretinal means under the retina, the light-sensing layer at the back of the eye. Adverse events are medical problems that happen during a study, and they may or may not be caused by the treatment.
Genetic confirmation means the study uses gene testing to make sure a person has the specific ABCA4 changes needed for the trial. Preliminary efficacy means the study is looking for early signs that the treatment may help, but the results are not yet final.