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Completed

NCT Number: NCT02481453

Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis

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.

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Key information

Conditions

Inclusion Body Myositis (IBM) Muscular Diseases Musculoskeletal Diseases Myositis Myositis, Inclusion Body Nervous System Diseases Neuromuscular Diseases

Age range

45 year–85 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

CIC Paris Est _Hôpital Pitié Salpêtrière

Paris, 75651, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Rapamycin

Drug

Experimental: rapamycin oral solution, 2 mg/day during one year Comparator: placebo

Other names: Sirolimus, Rapamune

Placebo

Drug

Comparator: placebo

Other names: Phosal

Primary outcomes

  1. stabilization of quadiceps strength measured by myometry

    Time frame: 52 weeks

Secondary outcomes

  1. stabilization of hand grip strength measured by myometry

    Time frame: 52 weeks

  2. comparison of 6 minutes walking test

    Time frame: 52 weeks

  3. 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)

  4. 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)

  5. measures of muscle fatty replacement by MRI

    Time frame: 52 weeks

  6. Measure of the tolerance

    Time frame: 52 weeks

    Efficacy will be measured by:

    check list of the known side effect of rapamycin.

  7. measure of inflammation by MRI

    Time frame: 52 weeks

Sponsors and collaborators

Lead sponsor

Institut National de la Santé Et de la Recherche Médicale, France

Other Gov

Registry information

Official study 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"

Acronym: RAPAMI

Important dates

Study start
2015
Primary completion
2018
Study completion
2018
First posted
Jun 25, 2015
Registry last updated
Sep 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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