Clinical Trial Opportunity for Adults with Stargardt Disease
The clinical trial team at The Retina Clinic London have asked us to share an opportunity to participate in a Phase 1 clinical trial for AAVB 039 for Stargardt Disease.
The clinical trial team at The Retina Clinic London have asked us to share an opportunity to participate in a Phase 1 clinical trial for AAVB 039 for Stargardt Disease. This trial is open to those aged 18-55 years old with a genetically confirmed mutation in the ABCA4 gene.
What is it?
AAVB-039 is a type of adeno-associated viral vector (AAV) gene therapy. This means that a small virus, that doesn’t cause illness in humans, is used as a delivery system to introduce or alter genetic material within cells. Most gene therapies for inherited retinal diseases work on the premise of delivering an entire new gene to replace the mutated one, however, the whole ABCA4 gene is too large to deliver in one viral vector. AAVantgarde (the Sponsor) is looking at delivering the ABCA4 gene in 2 halves, using dual viral vectors, through a one-time injection into the eye. Once in the cells, each half of the gene will make half of the desired protein. AAVB-039 then uses a process known as protein splicing to bond each half of the protein together to make the whole gene. To read more about AAVB-039 and AAVantgarde please visit this link here.
Please find some key inclusion criteria for the trial below and do be aware that the potential participants will need to meet a variety of further requirements for enrolment within the trial and may need to undergo further screening tests and there is no guarantee you will be eligible. Eligibility will be confirmed by the study team and participants may withdraw at any time.
Criteria
- 18-55 years at the time of enrolment
- Confirmed mutation ABCA4 gene
- Any condition that would preclude subretinal surgery
- Complicating ocular and/or systemic diseases
- No history of vitreoretinal or retinal macular surgery
- No current history of cancer for 5 years
- No history of myocardial infarct, stroke, or other heart conditions < 6 months
If you are unsure about whether you meet these criteria, please do feel free to contact The Retina Clinic London on 020 4538 5310. The staff there will be very happy to have a chat to clarify whether you might be eligible.
The trial:
This Phase 1/2 clinical trial, known as CELESTE, is aimed at testing the safety, tolerability and efficacy of a single administration of AAVB-039 via subretinal injection in participants with Stargardt disease. Subretinal injection involves delivering the gene therapy directly under the retina into the subretinal space, where the therapeutic agent can make direct contact with photoreceptors. As this is an early-phase clinical trial, participants may not experience any improvement in their vision.
CELESTE will involve 75 participants across sites in Europe and USA, with all participants receiving the gene therapy through a one-time injection. The study involves initial Screening, then 6 visits over 10 days around the gene therapy injection day, follow up then continues over a 5 year period.
Please note that there is no cost to participate in this trial and that the sponsor will be reimbursing travel, hotel and meal expenses for each visit.
Contact:
Contact details for the researchers can be found below, so do get in touch with them directly if you have any questions or would like to take part. When making contact, please provide your full name, date of birth, location in the UK, email, mobile and/or landline number.
Address: The Retina Clinic London, 24 Queen Anne Street, London W1G 9AX, UK.
Telephone: 020 4538 5310
Email: [email protected]
If you are interested in information on other participating sites, please contact the sponsor company AAVantgarde via [email protected]