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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
Sponsor:
Information provided by (Responsible Party):
Institut National de la Santé Et de la Recherche Médicale, France

Tracking Information
First Submitted Date  ICMJE May 13, 2015
First Posted Date  ICMJE June 25, 2015
Last Update Posted Date January 9, 2019
Actual Study Start Date  ICMJE July 15, 2015
Actual Primary Completion Date January 22, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: June 23, 2015)
stabilization of quadiceps strength measured by myometry [ Time Frame: 52 weeks ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 23, 2015)
  • 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 Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis
Official Title  ICMJE 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 Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Inclusion Body Myositis (IBM)
Intervention  ICMJE
  • Drug: Rapamycin
    Experimental: rapamycin oral solution, 2 mg/day during one year Comparator: placebo
    Other Names:
    • Sirolimus
    • Rapamune
  • Drug: Placebo
    Comparator: placebo
    Other Name: Phosal
Study Arms  ICMJE
  • Experimental: Rapamycin
    rapamycin 1 mg/ml oral solution, 2 mg/day (2 ml/day), once a day, during one year
    Intervention: Drug: Rapamycin
  • Placebo Comparator: Placebo
    Placebo oral solution, 2 ml/day, once a day, during one year
    Intervention: Drug: Placebo
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 23, 2015)
44
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE January 22, 2018
Actual Primary Completion Date January 22, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 45 Years to 85 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02481453
Other Study ID Numbers  ICMJE C12-66
2013-003485-14 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Institut National de la Santé Et de la Recherche Médicale, France
Study Sponsor  ICMJE Institut National de la Santé Et de la Recherche Médicale, France
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Institut National de la Santé Et de la Recherche Médicale, France
Verification Date January 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP