Clinical Trials Forum

The WA Clinical Trials Forum is a new initiative of the WAHTN Clinical Trials Enabling Platform-WA (CTEP-WA) to help support a thriving clinical trials ecosystem in WA by bringing together clinicians and researchers to strengthen the design of clinical trials.

The forum aims to build a community of practice with an emphasis on trial science that cuts across disease areas and disciplines, giving researchers and clinicians a platform to showcase their proposed clinical trials to an expert panel and the wider clinical trials community. This provides an opportunity for researchers and clinicians to receive early critical feedback from an expert panel, helping shape and strengthen proposed trials before significant time and resources are committed. The forum is planned to run regularly thorghout the year, with each session featuring one or two presentations from investigators.

Being part of a supportive community critically engaging with trial design is a powerful way to build experience and expertise that will ultimately strengthen the quality of proposals and funding success amongst the WA clinical trials community. Audience members are encouraged to attend and participate in the discussion, making the forum a valuable learning opportunity for anyone involved in clinical trials in WA. At the inaugural forum, Dr Arazu Sharif and Dr Bradley MacDonald will present their proposed trial on Probucol for Childhood Dementia.

 

Repurposing Probucol for Childhood Dementia: A Phase II Feasibility Study

Presenters: Dr Arazu Sharif and Dr Bradley MacDonald

Childhood dementia encompasses a group of rare, progressive neurodegenerative disorders that collectively affect more than 100 genetic conditions, with no approved disease-modifying therapies for most children. We propose a 52-week, investigator-initiated, open-label Phase II feasibility study evaluating oral probucol in children with genetically confirmed Batten disease, Niemann-Pick disease type C, Sanfilippo syndrome (MPS III) or Leigh syndrome. The primary objectives are to establish the feasibility of recruitment and retention, treatment adherence and the safety and tolerability of long-term probucol administration in this population, while collecting preliminary clinical, functional and biomarker data to inform future controlled studies.

Doors will open at 4:15pm, with the presentation and panel starting at 4:30, ending with a networking session to 6pm. Light refreshments provided.

Venue: McCusker Auditorium, at the Harry Perkins Institute of Medical Research, 6 Verdun St Nedlands WA (QEII Campus)

Online: An online livestream option is available for those who cannot attend in person. Select the 'Online' ticket type.

This forum is suited to investigators who have a trial in development and would benefit from methodological peer review prior to funding application. Investigators are encouraged to submit an expression of interest to present via email to ctep-wa@curtin.edu.au