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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for INCLUSION BODY MYOSITIS (IBM) are added.
By submitting, you agree to our Terms of use
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