NCT02481453

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
44

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Jul 2015

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 13, 2015

Completed
1 month until next milestone

First Posted

Study publicly available on registry

June 25, 2015

Completed
20 days until next milestone

Study Start

First participant enrolled

July 15, 2015

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 22, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 22, 2018

Completed
Last Updated

September 2, 2025

Status Verified

January 1, 2017

Enrollment Period

2.5 years

First QC Date

May 13, 2015

Last Update Submit

August 25, 2025

Conditions

Inclusion Body Myositis (IBM)

Outcome Measures

Primary Outcomes (1)

  • stabilization of quadiceps strength measured by myometry

    52 weeks

Secondary Outcomes (7)

  • stabilization of hand grip strength measured by myometry

    52 weeks

  • comparison of 6 minutes walking test

    52 weeks

  • composite measure of the handicap

    52 weeks

  • Quality of life by different scales

    52 weeks

  • measures of muscle fatty replacement by MRI

    52 weeks

  • +2 more secondary outcomes

Study Arms (2)

Rapamycin

EXPERIMENTAL

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

Drug: Rapamycin

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

RapamycinDRUG

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

Also known as: Sirolimus, Rapamune
Rapamycin
PlaceboDRUG

Comparator: placebo

Also known as: Phosal
Placebo

Eligibility Criteria

Age45 Years - 85 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • IBM defined by the Benveniste \& Hilton-Jones ( Neuromuscul Disord. 2010;20: 414-21) or Llyod criteria (Neurology 2014; 83: 426-433)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

CIC Paris Est _Hôpital Pitié Salpêtrière

Paris, 75651, France

Location

Related Publications (3)

  • Benveniste O, Hogrel JY, Belin L, Annoussamy M, Bachasson D, Rigolet A, Laforet P, Dzangue-Tchoupou G, Salem JE, Nguyen LS, Stojkovic T, Zahr N, Hervier B, Landon-Cardinal O, Behin A, Guilloux E, Reyngoudt H, Amelin D, Uruha A, Mariampillai K, Marty B, Eymard B, Hulot JS, Greenberg SA, Carlier PG, Allenbach Y. Sirolimus for treatment of patients with inclusion body myositis: a randomised, double-blind, placebo-controlled, proof-of-concept, phase 2b trial. Lancet Rheumatol. 2021 Jan;3(1):e40-e48. doi: 10.1016/S2665-9913(20)30280-0. Epub 2020 Oct 12.

    PMID: 38273639BACKGROUND

  • 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.

    PMID: 20413309BACKGROUND

  • 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.

    PMID: 24975859BACKGROUND

MeSH Terms

Conditions

Myositis, Inclusion Body

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

MyositisMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System Diseases

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER GOV
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 13, 2015

First Posted

June 25, 2015

Study Start

July 15, 2015

Primary Completion

January 22, 2018

Study Completion

January 22, 2018

Last Updated

September 2, 2025

Record last verified: 2017-01

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)