RecruitingPhase 3ACTRN12620001226998

Sirolimus in Inclusion Body Myositis (IBM)

Optimism in IBM: A Double-Blind Randomised Controlled Trial (dbRCT) Phase III trial of Sirolimus in patients with Inclusion Body Myositis (IBM), to slow or stabilise disease progression, as measured by the IBM Functional Rating Scale (IBM-FRS)

Sponsor

The Perron Institute for Neurological and Translational Science

Enrollment

140 participants

Start Date

Jun 15, 2022

Study Type

Interventional

Conditions

Inclusion Body Myositis

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.

Eligibility

Sex: Both males and femalesMin Age: 45 Yearss

Plain Language Summary

Simplified for easier understanding

This international Phase 3 clinical trial tests whether a medication called sirolimus (also known as rapamycin) can slow the progression of Inclusion Body Myositis (IBM) — a rare and currently incurable muscle disease. IBM causes progressive weakness, particularly in the hands and legs, making everyday tasks like gripping, climbing stairs, and walking increasingly difficult. There are no approved treatments that modify the disease course. Sirolimus has been identified as a strong candidate based on how IBM develops at the cellular level, and a small pilot study in France showed promising signs of disease stabilisation. In this larger, double-blind, randomised trial, half of participants will receive sirolimus and half will receive a placebo over 52 weeks, with the main outcome being changes in brain and muscle biomarkers. You may be eligible if you are 45 years of age or older, have been diagnosed with IBM according to established criteria, can walk at least 200 metres but no more than 500 metres within 6 minutes (with walking aids allowed), and are not currently on immunosuppressive medications. Women must use effective contraception and must not be pregnant.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

Interested in this trial?

Get notified about updates and connect with the research team.

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

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.