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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
44
CIC Paris Est _Hôpital Pitié Salpêtrière
Paris, France
stabilization of quadiceps strength measured by myometry
Time frame: 52 weeks
stabilization of hand grip strength measured by myometry
Time frame: 52 weeks
comparison of 6 minutes walking test
Time frame: 52 weeks
composite measure of the handicap
Rivermead Mobility Index (RMI), scale Walton, Inclusion Body Myositis Weakness Composite Index (IBMWCI), Inclusion Body Myositis Functional rating scale (IBMFRS)
Time frame: 52 weeks
Quality of life by different scales
Health Assessment Questionnaire (HAQ), Instrumental activities of daily living (IADL),Individualized Neuromuscular Quality of Life Questionnaire (INQol)
Time frame: 52 weeks
measures of muscle fatty replacement by MRI
Time frame: 52 weeks
Measure of the tolerance
Efficacy will be measured by: check list of the known side effect of rapamycin.
Time frame: 52 weeks
measure of inflammation by MRI
Time frame: 52 weeks
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