Clinical Trial: Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
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"
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.
Detailed Summary:
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
Current Primary Outcome: stabilization of quadiceps strength measured by myometry [ Time Frame: 52 weeks ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- 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 ]
Original Secondary Outcome: Same as current
Information By: Institut National de la Santé Et de la Recherche Médicale, France
Dates:
Date Received: May 13, 2015
Date Started: July 2015
Date Completion: April 2018
Last Updated: January 17, 2017
Last Verified: January 2017