The Study is the most important project for FAST UK and it is invaluable for the development, trials, regulatory approval and NHS adoption of any future disease-modifying therapeutics for Angelman Syndrome.
The researchers want to obtain a better understanding of the syndrome and help the healthcare system in UK prepare for the stream of upcoming therapies. The research team wants to contribute in the development of effective diagnostic tools, treatments and the design of successful clinical trials. This initiative is supported and funded by FAST UK.
With this study we aim to:
Ideally, all assessments will be completed in one day. Participants affected by Angelman syndrome and primary carers should expect to spend a full working day on site (approximately 8 hours). If assessments last longer than 8 hours during the first (baseline) on-site visit, then every follow-up on-site visit will be extended to two days. For non-Oxford residents, appropriate reimbursement, and arrangements for overnight stays in Oxford will be organised by the research team.
Unfortunately, the duration of the assessments cannot be accurately predicted prior to the baseline visit, as it largely depends on each participant’s individual progress with the assessments. However, we consider it unlikely that assessments will be extended to two days.
The baseline visit will require an overnight stay in Oxford if you are travelling from outside of Oxford for the study. This is due to the 24-hour EEG recording which must be returned to the research team the following day.
As this study is non-interventional, it does not include the administration of any drug.
Therefore, we do not anticipate any serious risks associated with this study. However, there are times when the participant might experience some discomfort. Blood sampling can lead to dizziness and bruising. However, appropriate measures to minimise pain will be taken. EEG can cause itchiness of the scalp particularly in the beginning of the recording. Additionally, the duration of the assessments might cause distress to the participant. However, these procedures will only be performed by qualified and experienced members of the research team or other specialists involved in the study. Similarly, if these events are causing too much distress, then they can be paused, and breaks can be taken.
Ionis has shared an important update with the Angelman syndrome community about the design of CHAMPION, its Phase 3 clinical trial evaluating ION582 (obudanersen). The
“Guided by input from the Angelman community, REVEAL was intentionally designed to evaluate obudanersen across a broad range of people living with Angelman syndrome, reflecting
Oak Hill Bio has shared a community letter to the Angelman syndrome community with an important update on the next steps of the development of
We found solace and purpose through FAST. Their relentless pursuit of a cure resonated with us, and we started our fundraising journey with them.