2023 January: Community Webinar about Natural History Study and Clinical Trials in the UK
In this community webinar, Theodora Markati and Laurent Servais from the University of Oxford provide updates on the Natural History Study for Angelman syndrome in the UK. The study aims to understand the natural progression of the syndrome and identify objective tools for use in clinical trials. They discuss the study’s components, eligibility criteria, and recruitment process. They also mention ongoing clinical trials for potential treatments, including those by Ultragenyx, Roche, and Ionis. The trials have different eligibility criteria and are at different stages. The speakers emphasize the burden and limitations of these trials and the need for careful patient selection. Additionally, Katie Cunnea from Angelman UK discusses the organization’s involvement in the Angelman Syndrome Alliance and their funding of research projects. She also mentions the Community Advisory Board, which provides expert guidance to pharmaceutical companies conducting clinical trials. The webinar concludes with a Q&A session.
Thank you all for joining tonight’s webinar. We are here to provide updates on the Natural History Study and clinical trials for Angelman syndrome in the UK. In this webinar, Dora will discuss the Natural History Study, while Laurent will provide updates on ongoing and upcoming clinical trials.
The Natural History Study
Dora Markati, from the University of Oxford, shared information about the Natural History Study. This two-year study, funded by FAST UK, aims to understand the natural progression of Angelman syndrome and identify objective tools for use in clinical trials. The study collects data on different domains affected by Angelman syndrome, such as communication, motor skills, behavior, and cognition. The study also aims to connect with the community and understand their current needs. Recruitment for the study is ongoing, and participants of any age or genotype can be eligible. Dora provided details on the study components and encouraged interested individuals to contact her or Professor Servais for more information.
Clinical Trials in the UK
Laurent Servais discussed the ongoing clinical trials for Angelman syndrome in the UK. He mentioned three companies – Ultragenyx, Roche, and Ionis – that are developing antisense oligonucleotide treatments to re-express the paternal UBE3A gene in the brain. Laurent emphasized that the trials are currently recruiting or planning to recruit participants. He explained the eligibility criteria for each trial and highlighted the importance of the natural history study in bridging the gap between research and clinical trials. Laurent also mentioned the challenges and limitations in terms of capacity and the need for anesthesiologists in conducting the trials.
Updates from FAST UK and Angelman UK
Tom Keogh provided updates from FAST UK, including their focus on funding the Natural History Study and organizing social events for the community. He mentioned their recent donation to the International Angelman Day event and their plans for future events. Louise Prince introduced Louise’s Cafe, an initiative aimed at providing support and a safe space for parents to share and vent. Katie Cunnea discussed the Angelman Syndrome Alliance, a partnership of organizations funding scientific research. She highlighted recent research grants and the community advisory board’s role in providing expert guidance to pharmaceutical companies.
The webinar concluded with a Q&A session, where participants had the opportunity to ask questions to the presenters. Questions ranged from eligibility criteria for clinical trials to the role of placebos and the availability of research institutions in the UK. The presenters provided detailed answers and encouraged participants to reach out via email for further inquiries.
The webinar provided valuable updates on the Natural History Study and clinical trials for Angelman syndrome in the UK. Participants gained insights into ongoing research, eligibility criteria, and the importance of community involvement. The presenters emphasized the need for collaboration and the role of the natural history study in advancing research and clinical trials.
- Title: 2023 January: Community Webinar about Natural History Study and Clinical Trials in the UK
- Author(s): Theodora Markati, Laurent Servais, Tom Keogh, Louise Prince, Katie Cunnea
- Author(s)’ affiliation: University of Oxford; FAST UK; AngelmanUK
- Publication date: 2023-01-31
- Collection: FAST UK Webinars