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