WAHTN CTEP-WA 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.

Wednesday 7 October 2026 4:00 PM – 6:00 PM

Repurposing Probucol for Childhood Dementia: A Phase II Feasibility Study

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

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

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

Online: An online livestream option is available for those who cannot attend in person. Select the ‘Online’ ticket type using the registration link above.

You can find out more about the event and register here: https://www.trybooking.com/DNAYR