Inclusion Body Myositis (IBM)
Conditions
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.
Interventions
DRUGRapamycin
Experimental: rapamycin oral solution, 2 mg/day during one year Comparator: placebo
DRUGPlacebo
Comparator: placebo
Sponsors
Institut National de la Santé Et de la Recherche Médicale, France
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
45 Years to 85 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| stabilization of quadiceps strength measured by myometry | 52 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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) |
| stabilization of hand grip strength measured by myometry | 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 | — |
| measures of muscle fatty replacement by MRI | 52 weeks | — |
Countries
France
Outcome results
None listed