Optimism in inclusion body myositis: a double-blind randomised controlled phase III trial investigating the effect of sirolimus on disease progression in patients with IBM as measured by the IBM Functional Rating Scale
This phase IIb/III trial builds on prior findings to assess sirolimus's potential in slowing or halting IBM progression, preserving patient function and independence, and advancing IBM therapeutic strategies and trial design.
Authors
- Ian Cooper (ORCID: https://orcid.org/0000-0002-0110-9070)
- Christine Lowe
- Kelly Beer (ORCID: https://orcid.org/0000-0003-0875-781X)
- Christina Liang (ORCID: https://orcid.org/0000-0002-2503-1917)
- Antonia Carroll (ORCID: https://orcid.org/0000-0002-1738-9580)
- Doris Chan
- Katrina Reardon (ORCID: https://orcid.org/0000-0002-6345-1859)
- Max Bulsara (ORCID: https://orcid.org/0000-0002-8033-6123)
- Lauren Sanders (ORCID: https://orcid.org/0000-0001-6420-0181)
- Anna Brusch
- Thomas E. Lloyd (ORCID: https://orcid.org/0000-0003-4756-3700)
- Roula Ghaoui (ORCID: https://orcid.org/0000-0003-2309-2086)
- Krystyne Hiscock
- Jens Schmidt (ORCID: https://orcid.org/0000-0002-5589-2371)
- Robert D. Henderson (ORCID: https://orcid.org/0000-0002-2820-8183)
- Mazen M. Dimachkie (ORCID: https://orcid.org/0000-0002-7148-989X)
- Merrilee Needham (ORCID: https://orcid.org/0000-0001-6591-1444)
- Stefen Brady (ORCID: https://orcid.org/0000-0001-6812-0920)
- Umesh A. Badrising (ORCID: https://orcid.org/0000-0003-0146-2664)
- Anneke J. van der Kooi (ORCID: https://orcid.org/0000-0002-5261-5512)
- Michael McDermott (ORCID: https://orcid.org/0000-0002-1015-0745)
- Matthew C. Kiernan (ORCID: https://orcid.org/0000-0001-9054-026X)
- Aneesya Panicker (ORCID: https://orcid.org/0000-0001-7902-3472)
- Andrew J. Heim
- Marion Simpson
- Payam Mohassel (ORCID: https://orcid.org/0000-0001-5313-9514)
- Timothy J. Fairchild (ORCID: https://orcid.org/0000-0002-3975-2213)
- Jérôme D. Coudert (ORCID: https://orcid.org/0000-0001-6081-8494)
- Alastair Corbett
- Stephen Reddel (ORCID: https://orcid.org/0000-0002-0169-3350)
- Dag Leonard (ORCID: https://orcid.org/0000-0002-6275-7282)
- Althea Doverty
- Gayatri Jain
- Marjolein van Heur-Neuman
- Chris Weihl
- Olivier Benveniste
- Susan Walters
Institutions
- Uppsala University (SE)
- The University of Sydney (AU)
- Johns Hopkins University (US)
- Royal Prince Alfred Hospital (AU)
- Johns Hopkins Medicine (US)
- Leiden University Medical Center (NL)
- Austin Hospital (AU)
- Sir Charles Gairdner Hospital (AU)
- St Vincent's Hospital Melbourne (AU)
- University of Kansas Medical Center (US)
- Perron Institute for Neurological and Translational Science (AU)
- Austin Health (AU)
- The University of Notre Dame Australia (AU)
Publication Details
- Journal
- Clinical and Experimental Rheumatology
- Published
- 2025-02-26
- DOI
- https://doi.org/10.55563/clinexprheumatol/zvffa0
- Citations
- 3
- Primary Topic
- Inflammatory Myopathies and Dermatomyositis
- Type
- article
- Field-Weighted Citation Impact
- 4.06