None listed
Conditions
Brief summary
Currently an estimated 1,250 Australians are living with Inclusion Body Myositis (IBM), a rare, chronic and incurable neuromuscular disease. IBM causes progressive muscle weakness and disability, compelling major life changes for patients and their families. There are no current disease-modifying treatments available for IBM. We are planning a clinical trial of a re-purposed drug (Sirolimus), to stabilise or slow progression of IBM, allowing patients to retain mobility, independence and quality of life for longer, as well as reducing healthcare costs. Sirolimus (Rapamycin) has been identified in pre-clinical studies as a strong treatment candidate based on its known mechanisms of action and our understanding of the pathogenesis of IBM. A small monocentric pilot study in France of Sirolimus in IBM demonstrated disease stabilisation in a cohort of 44 patients. This is an international, investigator-led Phase III trial of Sirolimus in 140 IBM patients. The trial will be led from Australia, and conducted as a double-blind, randomised, controlled Phase III trial (dbRCT). The study team includes leading IBM specialists across the globe, facilitating rapid translation into clinical care worldwide.
Interventions
Participants will be randomised 1:1 to either Sirolimus or Placebo.
Adherence will be monitored through compliance checks via drug packaging/container checks at study visits, weekly study diaries to monitor missed dosages, and measurement of serum Sirolimus levels at 3 month intervals.
Sirolimus arm: Sirolimus 2mg daily (2 x 1mg oral tablet), for 84 weeks.
Sponsors
The Perron Institute for Neurological and Translational Science
Study design
Allocation
Randomised controlled trial
Intervention model
Parallel
Primary purpose
Treatment
Masking
Blinded (masking used) (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
All
Age
45 Years to No maximum
Healthy volunteers
No
Inclusion criteria
• Adults able to read and understand the Participant Information Sheet, and who freely provide Informed Consent for the study; • Males or females aged 45 years or older; • Diagnosis of IBM according to the criteria proposed by the European Neuromuscular Centre (ENMC) criteria 2011. • Able to walk a minimum distance of 200m and maximum of 500m within 6 minutes (walking aids, including frames, may be used)
Exclusion criteria
• Inability to complete a 6MWT with a minimum distance of 200m and maximum of 500m achieved; • Inability to complete a mTUG or any other study procedure; • Unwillingness or inability to comply with study interventions or study schedule, including inability to swallow the study medication; • Hypersensitivity to Sirolimus, Everolimus or any compound of the oral solution • Any prior exposure to Sirolimus within the last 6 months; • Current use or exposure to Everolimus within the last 6 months; • Presence of any other clinically significant disease that might interfere with patients ability to comply with study procedures, or places the patient at greater risk for SAEs; • Patient taking any other immunosuppressive or immunomodulatory medication; • Other medications or products that may significantly affect the metabolism of Sirolimus at screening; • Pregnancy or planning a pregnancy.
Outcome results
None listed