Could a transplant drug help patients with rare muscle disease?
NCT ID NCT04789070
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 3 trial tests whether sirolimus, a drug used in organ transplants, can slow disease progression in people with inclusion body myositis (IBM), a rare muscle-weakening condition. The study involves 140 adults aged 45 and older who can walk at least 200 meters. Over 84 weeks, researchers will measure changes in muscle function using a rating scale and a walking test. The goal is to see if sirolimus helps patients stay stronger and more independent.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
sirolimus (also known as rapamycin)
What this could lead to
If successful, sirolimus could slow or stabilize muscle decline in inclusion body myositis, helping patients maintain function and independence longer.
What could go wrong
This is a phase 3 trial, but results are not yet available. Sirolimus is an immunosuppressant and may cause side effects like infections or mouth sores. It may not work for all patients.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Austin Health
Melbourne, Victoria, Australia
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Concord Repatriation Hospital
Sydney, New South Wales, Australia
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Johns Hopkins University
Baltimore, Maryland, 21218, United States
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Leiden University Medical Center
Leiden, Netherlands
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Perron Institute
Perth, Washington, Australia
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Royal Adelaide Hospital
Adelaide, South Australia, Australia
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Royal Brisbane and Women's Hospital
Brisbane, Queensland, Australia
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Royal Northshore Hospital
Sydney, New South Wales, Australia
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St Vincent's Hospital
Melbourne, Victoria, Australia
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University of Kansas Medical Center
Kansas City, Kansas, 66160, United States