RAPAMI: Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis
Study Details
Study Description
Brief 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.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2/Phase 3 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Rapamycin rapamycin 1 mg/ml oral solution, 2 mg/day (2 ml/day), once a day, during one year |
Drug: Rapamycin
Experimental: rapamycin oral solution, 2 mg/day during one year Comparator: placebo
Other Names:
|
| Placebo Comparator: Placebo Placebo oral solution, 2 ml/day, once a day, during one year |
Drug: Placebo
Comparator: placebo
Other Names:
|
Outcome Measures
Primary Outcome Measures
- stabilization of quadiceps strength measured by myometry [52 weeks]
Secondary Outcome Measures
- stabilization of hand grip strength measured by myometry [52 weeks]
- comparison of 6 minutes walking test [52 weeks]
- composite measure of the handicap [52 weeks]
Rivermead Mobility Index (RMI), scale Walton, Inclusion Body Myositis Weakness Composite Index (IBMWCI), Inclusion Body Myositis Functional rating scale (IBMFRS)
- Quality of life by different scales [52 weeks]
Health Assessment Questionnaire (HAQ), Instrumental activities of daily living (IADL),Individualized Neuromuscular Quality of Life Questionnaire (INQol)
- measures of muscle fatty replacement by MRI [52 weeks]
- Measure of the tolerance [52 weeks]
Efficacy will be measured by: check list of the known side effect of rapamycin.
- measure of inflammation by MRI [52 weeks]
Eligibility Criteria
Criteria
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
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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
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | CIC Paris Est _Hôpital Pitié Salpêtrière | Paris | France | 75651 |
Sponsors and Collaborators
- Institut National de la Santé Et de la Recherche Médicale, France
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
- Benveniste O, Hilton-Jones D. International Workshop on Inclusion Body Myositis held at the Institute of Myology, Paris, on 29 May 2009. Neuromuscul Disord. 2010 Jun;20(6):414-21. doi: 10.1016/j.nmd.2010.03.014. Epub 2010 Apr 21.
- Lloyd TE, Mammen AL, Amato AA, Weiss MD, Needham M, Greenberg SA. Evaluation and construction of diagnostic criteria for inclusion body myositis. Neurology. 2014 Jul 29;83(5):426-33. doi: 10.1212/WNL.0000000000000642. Epub 2014 Jun 27. Review.
- C12-66
- 2013-003485-14