Could a transplant drug slow muscle decline in rare disease?
NCT ID NCT02481453
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This study tested whether rapamycin, a drug used in organ transplants, can help people with Inclusion Body Myositis (IBM), a rare muscle disease that causes progressive weakness and has no approved treatment. Forty-four adults with IBM received either rapamycin or a placebo for one year. The main goal was to see if the drug could stabilize thigh muscle strength.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Rapamycin (also called sirolimus or Rapamune)
- What this could lead to
- If it works, this could point toward a treatment that slows muscle weakness in Inclusion Body Myositis, a disease with no current therapy.
- What could go wrong
- This is a small, early-phase trial with only 44 participants. The results may not apply to everyone, and rapamycin can have side effects like infections or mouth sores.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2/3
Runs two stages together: whether the treatment works, then large-scale confirmation.
- Participants
-
44 people
The number who actually took part.
- Started
-
Jul 2015
- Finished
-
Jan 2018
- Lead sponsor
-
A government agency
The lead sponsor is a government body.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
45 to 85 years
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
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Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * IBM defined by the Benveniste \& Hilton-Jones ( Neuromuscul Disord. 2010;20: 414-21) or Llyod criteria (Neurology 2014; 83: 426-433) Exclusion Criteria: * Impossiblility to walk 10 meters * Hypersensitivity to rapamycin or one compound of the oral solution * Severe respiratory insufficiency (FVC \< 50% and/or FEV1 \< 50%) * Severe chronic kidney disease (Estimated Glomerular Filtration Rate \< 15 ml/min and/or proteinuria \> 0.3 g/24h) * Chronic liver disease (cirrhosis and/or ALT/AST \> 2.5 normal values) * Cancer non in remission (necessitating specific treatment) during the past 12 months * Connective Tissue Disease non in remission (necessitating specific treatment) during the past 12 months * Pregnancy * Seropositivity for HIV, HCV or HBV * Total cholesterolemia \> 8 mmol/l * Triglyceridemia \> 5 mmol/l * Hemoglobinemia \< 11 g/dL * Thrombopenia \< 100 000/mm3 * Neutropenia \< 1500/ mm3 * Lymphopenia \< 1000/ mm3
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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
Locations
-
CIC Paris Est _Hôpital Pitié Salpêtrière
Paris, 75651, France
More trials for these conditions
Other studies related to the condition(s) this trial covers.