Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis (RAPAMI)
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|ClinicalTrials.gov Identifier: NCT02481453|
Recruitment Status : Completed
First Posted : June 25, 2015
Last Update Posted : January 9, 2019
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|
|Inclusion Body Myositis (IBM)||Drug: Rapamycin Drug: Placebo||Phase 2 Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||44 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Double (Participant, Outcomes Assessor)|
|Official Title:||Étude de l'Effet de la Rapamycine Sur la Force Musculaire et la réponse Immunitaire au Cours de la Myosite à Inclusions: étude RAPAMI"|
|Actual Study Start Date :||July 15, 2015|
|Actual Primary Completion Date :||January 22, 2018|
|Actual Study Completion Date :||January 22, 2018|
rapamycin 1 mg/ml oral solution, 2 mg/day (2 ml/day), once a day, during one year
Experimental: rapamycin oral solution, 2 mg/day during one year Comparator: placebo
Placebo Comparator: Placebo
Placebo oral solution, 2 ml/day, once a day, during one year
Other Name: Phosal
- 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 [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 52 weeks ]
- Measure of the tolerance [ Time Frame: 52 weeks ]
Efficacy will be measured by:
check list of the known side effect of rapamycin.
- measure of inflammation by MRI [ Time Frame: 52 weeks ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02481453
|CIC Paris Est _Hôpital Pitié Salpêtrière|
|Paris, France, 75651|