Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis

Phase 2

Completed

• Conditions: Inclusion Body Myositis (IBM)
• Interventions: Drug: Placebo; Drug: Rapamycin

• Registration Number: NCT02481453
• Lead Sponsor: 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2015-05-13
• Study First Submitted QC: 2015-06-23
• Study First Posted: 2015-06-25
• Study Start: 2015-07-15
• Status Verified: 2017-01
• Primary Completion: 2018-01-22
• Study Completion: 2018-01-22
• Last Update Submitted: 2019-01-08
• Last Update Posted: 2019-01-09

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 44

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Placebo oral solution, 2 ml/day, once a day, during one year
Rapamycin	Rapamycin	rapamycin 1 mg/ml oral solution, 2 mg/day (2 ml/day), once a day, during one year

Primary Outcome Measures

Name	Time	Method
stabilization of quadiceps strength measured by myometry	52 weeks

Secondary Outcome Measures

Name	Time	Method
measure of inflammation by MRI	52 weeks
Quality of life by different scales	52 weeks	Health Assessment Questionnaire (HAQ), Instrumental activities of daily living (IADL),Individualized Neuromuscular Quality of Life Questionnaire (INQol)
Measure of the tolerance	52 weeks	Efficacy will be measured by: check list of the known side effect of rapamycin.
comparison of 6 minutes walking test	52 weeks
stabilization of hand grip strength measured by myometry	52 weeks
composite measure of the handicap	52 weeks	Rivermead Mobility Index (RMI), scale Walton, Inclusion Body Myositis Weakness Composite Index (IBMWCI), Inclusion Body Myositis Functional rating scale (IBMFRS)
measures of muscle fatty replacement by MRI	52 weeks

Trial Locations

Locations (1)

CIC Paris Est _Hôpital Pitié Salpêtrière; 🇫🇷 Paris, France