In short
Clinical trials are investigating ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 ENCODING THE ABCA4 PROTEIN, N-REGION in people with Stargardt disease. The studies aim to assess safety, tolerability, and early signs of benefit in patients with confirmed bi-allelic ABCA4 gene mutations.
Key points
- Clinical trials are studying ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 ENCODING THE ABCA4 PROTEIN, N-REGION in people with Stargardt disease. The available trial is a Phase 1/2 study and is authorised. It focuses on subjects with Stargardt disease type 1 who have confirmed bi-allelic ABCA4 gene mutations. The study aims to assess safety, tolerability, and preliminary efficacy after subretinal administration. The main endpoint is safety and tolerability through Week 96, measured by ocular and non-ocular adverse events.
Trial overview
The available study is a clinical trial of ADENO-ASSOCIATED VIRUS VECTOR SEROTYPE 8 ENCODING THE ABCA4 PROTEIN, N-REGION in people with Stargardt disease. The trial is titled as a study to evaluate the safety, tolerability, and preliminary efficacy of SB-007 in subjects with Stargardt disease.
This is an interventional study, which means the research team gives a treatment and then measures what happens. The study has an enrollment of 86 and is listed as authorised.
Who can join
The trial is for subjects with Stargardt disease type 1 (STGD1). People must be confirmed by genetic testing to have bi-allelic ABCA4 gene mutations, which means changes in both copies of the ABCA4 gene.
The source data does not list age limits, vision requirements, or other eligibility rules, so only the criteria above can be confirmed from the trial record.
What is being measured
The main endpoint is safety and tolerability through Week 96. Safety means the study checks whether unwanted medical problems happen, and tolerability means how well participants can handle the treatment.
The study measures this by looking at ocular and non-ocular adverse events, including any clinically significant changes. Ocular adverse events are problems in the eye, while non-ocular adverse events are problems elsewhere in the body.
The brief summary also says the study is looking at preliminary efficacy, which means early signs that the treatment may help, and it is being used to help determine dose selection.
Trial phase and status
This study is in Phase 1/2. Phase 1/2 trials are early studies that usually focus on safety first and also look for early evidence of benefit.
The trial status is Authorised. The study is also described as a subretinal administration trial, meaning the treatment is given under the retina, which is the light-sensitive layer at the back of the eye.
Study details
The intervention listed in the source is SB-007 (SUBRETINAL USE). The trial title and brief summary show that the study is designed to evaluate safety, tolerability, and preliminary efficacy in Stargardt disease.
The study specifically targets people with a genetic form of Stargardt disease, because the source requires confirmed bi-allelic ABCA4 gene mutations. This makes the trial focused on a clearly defined patient group rather than all people with vision loss.
Key patient terms
- Interventional study: a study where participants receive a treatment and the researchers measure the results.
- Preliminary efficacy: early signs that a treatment may help, but not final proof.
- Adverse events: unwanted medical problems that happen during a study.
- Week 96: the study time point used for the main safety and tolerability assessment.