Clinical trial NCT02481453: 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"
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Inclusion Body Myositis (IBM)
Lead sponsor: Institut National de la Santé Et de la Recherche Médicale, France
Sponsor class: OTHER_GOV
First posted: 2015-06-25
Enrollment: 44
Sex: ALL
Minimum age (years): 45
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: stabilization of quadiceps strength measured by myometry
timeFrame: 52 weeks
Secondary outcomes:
measure: stabilization of hand grip strength measured by myometry
timeFrame: 52 weeks
measure: comparison of 6 minutes walking test
timeFrame: 52 weeks
measure: composite measure of the handicap
timeFrame: 52 weeks
measure: Quality of life by different scales
timeFrame: 52 weeks
measure: measures of muscle fatty replacement by MRI
timeFrame: 52 weeks
measure: Measure of the tolerance
timeFrame: 52 weeks
measure: measure of inflammation by MRI
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Paris
country: France
facility: CIC Paris Est _Hôpital Pitié Salpêtrière
Study officials:
References:
citation: 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.
citation: 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.