Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis (NCT02481453) | Clinical Trial Compass
CompletedPhase 2/3
Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis
France44 participantsStarted 2015-07-15
Plain-language summary
Sporadic Inclusion Body Myositis (IBM) is the most frequent inflammatory myopathy in patients over 50. It is a slowly progressive, but today untreatable (notably by classical immunosuppressants) disease.
Rapamycin used in organ transplantation blocks the activity of T effector cells, preserves T regulatory cells and induces autophagy (protein degradation), all parameters impaired during IBM.
RAPAMI is a prospective, randomised, controlled, double blind, monocentric, phase IIb trial evaluating rapamycine against placebo.
Who can participate
Age range
45 Years – 85 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
stabilization of quadiceps strength measured by myometry
Timeframe: 52 weeks
Trial details
NCT IDNCT02481453
SponsorInstitut National de la Santé Et de la Recherche Médicale, France