Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis

• Status: Completed
• Trial end date: 2018-01-22
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
• Treatments: Everolimus; Sirolimus

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