impact factor, citescore
logo
In press
In Press
Recent issue
Recent issue
Recent Supplement
Recent supplement
archives
Archives
articles free
Free to view
Search
Search
 

Full Papers

 

Challenges in international investigator-led rare disease clinical trials and the case for optimism in inclusion body myositis

1, 2, 3, 4, 5, 6, 7, 8

 

  1. Department of Neurology, Fiona Stanley Hospital, Western Australia; Centre for Molecular Medicine & Innovative Therapeutics, Murdoch University, Western Australia; School of Medicine, Notre Dame University, Western Australia; and Perron Institute for Neurological and Translational Science, Western Australia, Australia.
  2. Leiden University Medical Center, Leiden, The Netherlands.
  3. Centre for Molecular Medicine & Innovative Therapeutics, Murdoch University, Western Australia; and Perron Institute for Neurological and Translational Science, Western Australia, Australia.
  4. University of Kansas Medical Center, Kansas City, KS, USA. aheim2@kumc.edu
  5. Centre for Molecular Medicine & Innovative Therapeutics, Murdoch University, Western Australia; and Perron Institute for Neurological and Translational Science, Western Australia, Australia.
  6. Perron Institute for Neurological and Translational Science, Western Australia, Australia.
  7. Sorbonne Université, Hôpital de la Pitié-Salpêtrière, Paris, France.
  8. University of Kansas Medical Center, Kansas City, KS, USA.

CER18317
2025 Vol.43, N°2
PI 0309, PF 0315
Full Papers

Free to view
(click on article PDF icon to read the article)

PMID: 40018747 [PubMed]

Received: 08/11/2024
Accepted : 03/02/2025
In Press: 26/02/2025
Published: 26/02/2025

Abstract

OBJECTIVES:
This paper aims to provide insight into the challenges and opportunities of conducting an investigator-led, international, multicentre clinical trial for Inclusion Body Myositis (IBM), a rare inflammatory myopathy.
METHODS:
An international, multicentre, randomised, controlled trial of a repurposed drug (sirolimus) was initiated based on promising results from a mono-centric pilot study. The progress of the trial was analysed to identify key challenges encountered and solutions developed.
RESULTS:
This large, collaborative study has presented a mosaic of challenges and opportunities, many ubiquitous with investigator-led trials. Key challenges have included securing adequate funding, coordinating manufacture of placebo, negotiating international contracts, managing limited study budgets and delays linked to the COVID-19 pandemic. Alongside these challenges, the study team have found opportunities for creative and effective solutions, including the flexibility of building study databases, optimising digital data capture and harnessing patient involvement.
CONCLUSIONS:
Instrumental to the progress of the trial has been the collaboration between site teams, patient partnership and adaptability.

DOI: https://doi.org/10.55563/clinexprheumatol/dyjcsn

Rheumatology Article