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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 : Active, not recruiting
First Posted : June 25, 2015
Last Update Posted : January 18, 2017
Sponsor:
Information provided by (Responsible Party):
Institut National de la Santé Et de la Recherche Médicale, France

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 Type : Interventional  (Clinical Trial)
Estimated 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"
Study Start Date : July 2015
Estimated Primary Completion Date : April 2018
Estimated Study Completion Date : April 2018


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




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 ]


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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

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

Publications:
Responsible Party: Institut National de la Santé Et de la Recherche Médicale, France
ClinicalTrials.gov Identifier: NCT02481453     History of Changes
Other Study ID Numbers: C12-66
2013-003485-14 ( EudraCT Number )
First Posted: June 25, 2015    Key Record Dates
Last Update Posted: January 18, 2017
Last Verified: January 2017

Additional relevant MeSH terms:
Myositis
Myositis, Inclusion Body
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs