Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02481453
• Date of registration: 13/05/2015
• Prospective Registration: Yes
• Primary sponsor: Institut National de la Santé Et de la Recherche Médicale, France
• Public title: Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis RAPAMI
• Scientific 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"
• Date of first enrolment: July 15, 2015
• Target sample size: 44
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02481453
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Double (Participant, Outcomes Assessor).
• Phase: Phase 2/Phase 3

Countries of recruitment

France

Key inclusion & exclusion 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



Age minimum: 45 Years
Age maximum: 85 Years
Gender: All

Health Condition(s) or Problem(s) studied

Inclusion Body Myositis (IBM)

Intervention(s)

Drug: Placebo

Drug: Rapamycin

Primary Outcome(s)

stabilization of quadiceps strength measured by myometry [Time Frame: 52 weeks]

Secondary Outcome(s)

comparison of 6 minutes walking test [Time Frame: 52 weeks]

stabilization of hand grip strength measured by myometry [Time Frame: 52 weeks]

Measure of the tolerance [Time Frame: 52 weeks]

Quality of life by different scales [Time Frame: 52 weeks]

measures of muscle fatty replacement by MRI [Time Frame: 52 weeks]

composite measure of the handicap [Time Frame: 52 weeks]

measure of inflammation by MRI [Time Frame: 52 weeks]

Secondary ID(s)

C12-66

2013-003485-14

Source(s) of Monetary Support

Please refer to primary and secondary sponsors