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Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis (RAPAMI)

Primary Purpose

Inclusion Body Myositis (IBM)

Status

Completed

Phase

Phase 2

Locations

France

Study Type

Interventional

Intervention

Rapamycin

Placebo

About this trial

This is an interventional treatment trial for Inclusion Body Myositis (IBM)

Eligibility Criteria

45 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

CIC Paris Est _Hôpital Pitié Salpêtrière

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Rapamycin

Placebo

Arm Description

rapamycin 1 mg/ml oral solution, 2 mg/day (2 ml/day), once a day, during one year

Placebo oral solution, 2 ml/day, once a day, during one year

Outcomes

Primary Outcome Measures

stabilization of quadiceps strength measured by myometry

Secondary Outcome Measures

stabilization of hand grip strength measured by myometry

comparison of 6 minutes walking test

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)

Quality of life by different scales

Health Assessment Questionnaire (HAQ), Instrumental activities of daily living (IADL),Individualized Neuromuscular Quality of Life Questionnaire (INQol)

measures of muscle fatty replacement by MRI

Measure of the tolerance

Efficacy will be measured by: check list of the known side effect of rapamycin.

measure of inflammation by MRI

Full Information

NCT ID

NCT02481453

First Posted

May 13, 2015

Last Updated

January 8, 2019

Sponsor

Institut National de la Santé Et de la Recherche Médicale, France

1. Study Identification

Unique Protocol Identification Number

NCT02481453

Brief Title

Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis

Acronym

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"

Study Type

Interventional

2. Study Status

Record Verification Date

January 2017

Overall Recruitment Status

Completed

Study Start Date

July 15, 2015 (Actual)

Primary Completion Date

January 22, 2018 (Actual)

Study Completion Date

January 22, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Institut National de la Santé Et de la Recherche Médicale, France

4. Oversight

Data Monitoring Committee

Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Inclusion Body Myositis (IBM)

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Parallel Assignment

Masking

ParticipantOutcomes Assessor

Allocation

Randomized

Enrollment

44 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Rapamycin

Arm Type

Experimental

Arm Description

rapamycin 1 mg/ml oral solution, 2 mg/day (2 ml/day), once a day, during one year

Arm Title

Placebo

Arm Type

Placebo Comparator

Arm Description

Placebo oral solution, 2 ml/day, once a day, during one year

Intervention Type

Drug

Intervention Name(s)

Rapamycin

Other Intervention Name(s)

Sirolimus, Rapamune

Intervention Description

Experimental: rapamycin oral solution, 2 mg/day during one year Comparator: placebo

Intervention Type

Drug

Intervention Name(s)

Placebo

Other Intervention Name(s)

Phosal

Intervention Description

Comparator: placebo

Primary Outcome Measure Information:

Title

stabilization of quadiceps strength measured by myometry

Time Frame

52 weeks

Secondary Outcome Measure Information:

Title

stabilization of hand grip strength measured by myometry

Time Frame

52 weeks

Title

comparison of 6 minutes walking test

Time Frame

52 weeks

Title

composite measure of the handicap

Description

Rivermead Mobility Index (RMI), scale Walton, Inclusion Body Myositis Weakness Composite Index (IBMWCI), Inclusion Body Myositis Functional rating scale (IBMFRS)

Time Frame

52 weeks

Title

Quality of life by different scales

Description

Health Assessment Questionnaire (HAQ), Instrumental activities of daily living (IADL),Individualized Neuromuscular Quality of Life Questionnaire (INQol)

Time Frame

52 weeks

Title

measures of muscle fatty replacement by MRI

Time Frame

52 weeks

Title

Measure of the tolerance

Description

Efficacy will be measured by: check list of the known side effect of rapamycin.

Time Frame

52 weeks

Title

measure of inflammation by MRI

Time Frame

52 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

45 Years

Maximum Age & Unit of Time

85 Years

Accepts Healthy Volunteers

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

Facility Information:

Facility Name

CIC Paris Est _Hôpital Pitié Salpêtrière

City

Paris

ZIP/Postal Code

75651

Country

France

12. IPD Sharing Statement

Citations:

PubMed Identifier

20413309

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.

Results Reference

background

PubMed Identifier

24975859

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.

Results Reference

background

Learn more about this trial

Rapamycine vs Placebo for the Treatment of Inclusion Body Myositis

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