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

• ClinicalTrials.gov Identifier: NCT02481453
• Recruitment Status: Completed
• First Posted: June 25, 2015
• Last Update Posted: January 9, 2019
• Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
• Information provided by (Responsible Party): Institut National de la Santé Et de la Recherche Médicale, France

Tracking Information

• First Submitted Date: May 13, 2015
• First Posted Date: June 25, 2015
• Last Update Posted Date: January 9, 2019
• Actual Study Start Date: July 15, 2015
• Actual Primary Completion Date: January 22, 2018 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: June 23, 2015): stabilization of quadiceps strength measured by myometry [ Time Frame: 52 weeks ]
• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (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: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis
• 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 Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2; Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double (Participant, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Inclusion Body Myositis (IBM)

Intervention

• 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

• 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 *

• Benveniste O, Hilton-Jones D. International Workshop on Inclusion Body Myositis held at the Institute of Myology, Paris, on 29 May 2009. Neuromuscul Disord. 2010 Jun;20(6):414-21. doi: 10.1016/j.nmd.2010.03.014. Epub 2010 Apr 21. No abstract available.
• Lloyd TE, Mammen AL, Amato AA, Weiss MD, Needham M, Greenberg SA. Evaluation and construction of diagnostic criteria for inclusion body myositis. Neurology. 2014 Jul 29;83(5):426-33. doi: 10.1212/WNL.0000000000000642. Epub 2014 Jun 27.

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: June 23, 2015): 44
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: January 22, 2018
• Actual Primary Completion Date: January 22, 2018 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 45 Years to 85 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: France

Administrative Information

• NCT Number: NCT02481453
• Other Study ID Numbers: C12-66; 2013-003485-14 ( EudraCT Number )
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: Institut National de la Santé Et de la Recherche Médicale, France
• Original Responsible Party: Same as current
• Current Study Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Institut National de la Santé Et de la Recherche Médicale, France
• Verification Date: January 2017