Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis

Inclusion Body Myositis (IBM) Clinical Trial

— RAPAMI  

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"

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02481453
• Other study ID #: C12-66
• Secondary ID: 2013-003485-14
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: July 15, 2015
• Est. completion date: January 22, 2018

Study information

• Verified date: January 2017
• Source: Institut National de la Santé Et de la Recherche Médicale, France
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 44
• Est. completion date: January 22, 2018
• Est. primary completion date: January 22, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 45 Years to 85 Years

Eligibility

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

Related Conditions & MeSH terms

• Inclusion Body Myositis (IBM)
• Myositis
• Myositis, Inclusion Body

Intervention

Drug:
• Rapamycin
Experimental: rapamycin oral solution, 2 mg/day during one year Comparator: placebo
• Placebo
Comparator: placebo

Locations

Country	Name	City	State
France	CIC Paris Est _Hôpital Pitié Salpêtrière	Paris

Sponsors (1)

Lead Sponsor	Collaborator
Institut National de la Santé Et de la Recherche Médicale, France

Country where clinical trial is conducted

France, 

References & Publications (2)

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. — View 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. Review. — View Citation

Outcome

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