Challenges in international investigator-led rare disease clinical trials and the case for optimism in inclusion body myositis
Authors' conclusion
Instrumental to the progress of the trial has been the collaboration between site teams, patient partnership and adaptability.
Study context
- Population
- International, multicentre, randomised, controlled trial of repurposed sirolimus in Inclusion Body Myositis (IBM), a rare inflammatory myopathy
Methodological detailsexperience report on an investigator-led RCT, international, multicentre
- Experience report on an investigator-led RCT
- International
- Multicentre
Primary result
As reported in the abstract
- Key challenges: securing adequate funding, coordinating placebo manufacture, negotiating international contracts, managing limited study budgets and COVID-19 pandemic delays
- Opportunities: flexibility of building study databases, optimising digital data capture and harnessing patient involvement
Original publication
Challenges in international investigator-led rare disease clinical trials and the case for optimism in inclusion body myositis. Clin Exp Rheumatol. Published 26 February 2025. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →