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Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis (RAPAMI)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
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

Study Description
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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.


Condition or disease Intervention/treatment Phase
Inclusion Body Myositis (IBM) Drug: Rapamycin Drug: Placebo Phase 2 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 44 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Myositis

Arms and Interventions
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Arm Intervention/treatment
Experimental: Rapamycin
rapamycin 1 mg/ml oral solution, 2 mg/day (2 ml/day), once a day, during one year
 Drug: Rapamycin
Experimental: rapamycin oral solution, 2 mg/day during one year Comparator: placebo
Other Names:
  • Sirolimus
  • Rapamune
Placebo Comparator: Placebo
Placebo oral solution, 2 ml/day, once a day, during one year
 Drug: Placebo
Comparator: placebo
Other Name: Phosal


Outcome Measures
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Primary Outcome Measures :
  1. stabilization of quadiceps strength measured by myometry [ Time Frame: 52 weeks ]

Secondary Outcome Measures :
  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 ]

Eligibility Criteria
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Ages Eligible for Study:   45 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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France
CIC Paris Est _Hôpital Pitié Salpêtrière
Paris, France, 75651
Sponsors and Collaborators
Institut National de la Santé Et de la Recherche Médicale, France
More Information
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Publications:

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Responsible Party: Institut National de la Santé Et de la Recherche Médicale, France
ClinicalTrials.gov Identifier: NCT02481453    
Other Study ID Numbers: C12-66
2013-003485-14 ( EudraCT Number )
First Posted: June 25, 2015    Key Record Dates
Last Update Posted: January 9, 2019
Last Verified: January 2017
Additional relevant MeSH terms:
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Myositis
Myositis, Inclusion Body
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Sirolimus
Anti-Bacterial Agents
 Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs