FAQs

Answering questions such as this one is the reason we need clinical trials. As these gene therapies have not been trialed in humans before, the answer is not yet known. It is expected that administration of these gene therapies will lead to a stopping or a slowing down of disease progression in participants who receive them.

Participation varies depending on the trial however many of the visit procedures are similar to the assessments carried out during a regular visit to your ophthalmologist. In addition, there may be other tests which are not part of your routine visit, including blood tests, answering questions related to your quality of life and more. A full list of the tests which will be carried out during the trial is shared with interested participants in the informed consent form prior to them being included in the study.

Information relating to the visit schedule will be shared in the informed consent form. Participants can still visit their ophthalmologist for regular eye clinic appointments if they wish to do so.

There is no cost to trial participants for participating in the trial. All costs incurred arising from trial participation such as transport, accommodation and meals (within fair market value) will be reimbursed by the sponsor of the trial for trial participants and their caregiver (where applicable) 

The duration of each visit varies; in some cases, the visits may take place over a full working day or may be run over several days depending on the number of assessments during that particular visit. Information relating to the visit schedule will be shared in the informed consent form.

While on the trial a participant should not participate in any other clinical trials. Participants should also not start any newly prescribed or over-the-counter medication or take health supplements without first consulting with their trial doctor. Depending on the trial, there may be other trial specific restrictions and your trial doctor will share this information before and during the trial.

A potential participant can be referred to the trial doctor by their own doctor or can, in most cases, contact the trial site themselves to make an appointment and ask about the potential to participate in the trial.

There is a risk that a participant’s vision may worsen. The reasons why this might happen are included in the informed consent form that every participant needs to sign to ensure that everyone participating in the trial is aware of any associated risks. It is important that any potential trial participant discuss their individual circumstances with their primary care physician and the study doctor.

Yes, a participant can withdraw from the trial at any stage; they only need to let their trial doctor know that they wish to withdraw. The trial doctor may ask the participant to return to the clinic for some follow-up assessments to ensure their safety as part of their withdrawal from the trial but there is no obligation on the participant to return if they do not wish to do so.

A participant's potential to be included in another clinical trial, including another gene therapy trial, at a later date can be affected by participation in this clinical trial. It is important that any potential trial participant discuss their individual circumstances and any potential impact of participation in future trials with their primary care physician and the study doctor. 

Where a clinical trial demonstrates efficacy, then widening of the age criteria may be possible but, usually, only following other clinical trials where that age group is included and where the trial has been shown to be safe and effective. 

As the investigational gene therapy is a one-time injection, participants will be followed for up to 5 years. 

AAVantgarde’s research focus is on Stargardt disease and Usher Syndrome Type 1B. It is possible, however, that our research may generate insights that spark new lines of research which could, in turn, benefit the wider IRD community.

New treatments are typically approved once regulatory agencies are satisfied that robust clinical trial data prove that a new medication is safe to use and demonstrates a clear benefit compared to current standards of care. The timelines depend on the results from ongoing studies, so while progress is being made, exact dates are difficult to predict.

Each country’s regulatory agency has its own criteria for licensing new treatments and deciding how and when those treatments are made available. This means that approval in country does not automatically guarantee access in another country. Advocacy from patient groups supports the evaluation process.

Patients can play an important role in advancing research, including helping to raise funds for research, supporting patient advocacy organisations in their work, advocating for patients with the rare condition with regulatory agencies and payers, participating in clinical trials including in natural history studies (where eligible), engaging with local representatives to ensure their community’s voice and needs are heard and understood.