Ruxolitinib Treatment in Inclusion Body Myositis (BIGTIM)

May 27, 2026 updated by: Assistance Publique - Hôpitaux de Paris

Blocking Interferon-γ by Ruxolitinib for Treating Inclusion Body Myositis: a Phase IIb Trial

Refer to the "Detailed Description" section.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of Ruxolitinib in the Treatment of Subjects with Inclusion Body Myositis (IBM) IBM is the most frequent idiopathic immune myopathy (IIM) over age 45, pathologically characterized by the combination of intramuscular inflammation and degenerative features. It differs from other IIMs by its chronic evolution and refractoriness to common immunomodulatory drugs leading to marked disability and poor quality of life. Histological and molecular analyses of muscle biopsies from IBM patients showed intense muscular type II interferon (IFNγ) signature, stronger than observed in other IIMs. In vitro and in vivo experimental studies showed that IFNγ exerts myosuppressive effects through JAK/STAT pathway activation mimicking the degenerative features observed in IBM, and that these effects can be prevented by JAK-inhibitor ruxolitinib.

Hypothesis/Objective : Ruxolitinib could be an effective therapy for IBM. Objective is to evaluate its therapeutic effects in IBM.

Method : Comparative, multicenter, randomized, parallel-group, superiority, placebo-controlled, double-blind, phase 2 trial. 60 IBM patients able to walk during at least 6mn will be randomized in two groups (30/group) and received either ruxolitinib 15mgx2/d or placebo during 1 yr. Evaluation includes 6MWT, muscle strength quantification, functional scales, respiratory functional test and muscle MRI.

Study Type

Interventional

Enrollment (Estimated)

80

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • France
    • France
      • Angers, France, France, 49100
        • Enrolling by invitation
        • CHU d'Angers
      • Bayonne, France, France, 64109
        • Enrolling by invitation
        • Centre Hospitalier de la Cote Basque
      • Besançon, France, France, 25030
        • Enrolling by invitation
        • Hôpital CHU Jean Minjoz, CHU Besançon
      • Bordeaux, France, France, 33076
        • Active, not recruiting
        • Hôpital Pellegrin - Tripode, CHU de Bordeaux
      • Brest, France, France, 29200
        • Enrolling by invitation
        • Hôpital de la Cavale Blanche, CHU de Brest
      • Bron, France, France, 69500
        • Active, not recruiting
        • Hôpital Pierre Wertheimer, CHU de Lyon
      • Caen, France, France, 14000
        • Recruiting
        • CHU Caen Normandie
        • Contact:
      • Créteil, France, France, 94010
      • Garches, France, France, 92380
        • Active, not recruiting
        • Hôpital Raymond Poincaré, APHP
      • Lille, France, France, 59037
        • Recruiting
        • Hôpital Roger Salengro, CHU de Lille
        • Contact:
      • Limoges, France, France, 87000
        • Recruiting
        • Hôpital Dupuytren, CHU de Limoges
        • Contact:
      • Marseille, France, France, 13005
        • Not yet recruiting
        • Hôpital de la Timone, APHM
        • Contact:
      • Montpellier, France, France, 34000
        • Enrolling by invitation
        • Hôpital Gui de Chauliac, CHU de Montpellier
      • Nancy, France, France, 54035
        • Recruiting
        • CHU Nancy
        • Contact:
      • Nantes, France, France, 44093
        • Active, not recruiting
        • Hôtel-Dieu, CHU Nantes
      • Nice, France, France, 06001
        • Recruiting
        • Hôpital Pasteur, CHU de Nice
        • Contact:
      • Paris, France, France, 75013
        • Recruiting
        • Hôpital Pitié-Salpêtrière, APHP
        • Contact:
      • Reims, France, France, 51100
        • Recruiting
        • Hôpital Christian Cabrol, CHU Reims
        • Contact:
      • Saint-Etienne, France, France, 42270
      • Strasbourg, France, France, 67089
        • Active, not recruiting
        • Hôpital de Hautepierre, Hôpitaux Universitaires de Strasbourg
      • Toulouse, France, France, 31059
        • Recruiting
        • Hôpital Pierre-Paul Riquet, CHU de Toulouse
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Age ≥ 45 years
  • Effective contraception for the duration of the clinical trial for fertile women of childbearing age
  • Defined diagnosis of IBM according to data-derived criteria (Llyod et al, 2014): Patient must fulfill the three following criteria for being diagnosed as IBM: (1) finger flexor or quadriceps weakness; and (2) muscle biopsy showing endomysial inflammation; and (3) muscle biopsy showing invasion of nonnecrotic muscle fibers or rimmed vacuoles
  • To be able to walk 6 min without assistance from another person (external assist devices permitted [e.g., canes, walkers, or rollators])
  • Patient informed and having signed the consent for participation, possibly assisted by a trusted person

Exclusion Criteria:

  • Pregnancy or breastfeeding
  • Patient under guardianship, curatorship, safeguard of justice or deprived of liberty
  • Patient with cognitive disorders or unable, according to the investigator, to understand the study and/or to give informed consent
  • Quadriceps weakness (manual muscle testing, MRC) below or equal 1
  • Forced vital capacity (FVC) or forced expiratory volume (FEV) < 50% of predicted value
  • Concomitant use of immunomodulatory drugs including previous treatment with JAK inhibitor, or medications acting on muscle anabolism or catabolism
  • Live vaccine within the 4 weeks before starting treatment

  • Comorbidity or active chronic disease which contraindicate ruxolitinib:

    • Lipid parameters abnormalities/elevations
    • Severe renal impairment (stage 4) and end-stage renal disease (stage 5)
    • Hepatic impairment
    • Cytopenia
    • Recent history (<6 months) of cardiovascular or thromboembolic disease (documented coronaropathy or hospitalization for acute arterial thrombosis or stroke or deep venous thrombosis or pulmonary embolism)
    • Active smoking more than 20 pack-years or history of respiratory or skin cancer or recent history (<6 months) of other neoplastic disease
    • Very high cardiovascular risk (red color) at SCORE 2 in case of recent history (<6 months) of cardiovascular or thromboembolic disease and non-controlled cardiovascular risk factors
    • History of Stevens-Johnson's syndrome or Lyell's syndrome
  • Active SARS-CoV-2 infection (patient can be included once infection resolved)
  • Any medical condition which limits the ability of participant to participate in study
  • Necessity to use a drug incompatible with ruxolitinib
  • Hypersensitivity to the IMP's active substance (ruxolitinib) or to any of the excipients
  • Non-affiliation to a social security scheme or to another social protection scheme, patient on state medical aid
  • Foreseeable inability, according to the investigator, to participate in all the visits, treatments and measures provided for in the protocol
  • Concomitant participation in another clinical trial on medical product for human use, to a clinical investigation on a medical device, to interventional study involving human participants or in the exclusion period at the end of a previous clinical trial on medical product for human use, a clinical investigation on a medical device, or study involving human participants.

Participation in non-interventional research is permitted.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Experimental group
Randomization in experimental group.
Drug: Ruxolitinib
IBM patients treated by ruxolitinib (JAKAVI®), 15mg per os, twice a day, during 12 months.
Placebo Comparator: Control group
Randomization in control group.
Drug: Placebo
IBM patients treated by placebo, twice a day, during 12 months.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
6 minutes-walk distance (6MWT): A distance walked in 6 min. superior in treated patients compared to placebo group
Time Frame: 12 months
The 6MWT is performed in a corridor, between two cones separated by a distance of 25 m.
12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Adverse events (safety and tolerability) of ruxolitinib in IBM patients
Time Frame: Through study completion that is to say 15 months
Adverse events collected according to the MedDRA classification
Through study completion that is to say 15 months
Therapeutic muscular efficacy of ruxolitinib on muscle strength
Time Frame: Until last consultation that is to say 12 months
Quantification of muscle strength using dynamometer
Until last consultation that is to say 12 months
Therapeutic muscular efficacy of ruxolitinib on overall muscle status
Time Frame: Until last consultation that is to say 12 months
Measurement of overall muscle status using scales and serum creatine kinase (CK) levels
Until last consultation that is to say 12 months
Respiratory ability
Time Frame: 12 months
Forced vital capacity (FVC) measurement
12 months
Evaluate swallowing using Swallowing Disturbance Questionnaire (SDQ)
Time Frame: 12 months
Measurement of the swallowing disorders via Swallowing Disturbance Questionnaire (SDQ). 15 items. Overall score from 0,5 to 44,5. The score increase with the swallowing disorders.
12 months
Evaluate quality of life using Health Assessment Questionnaire without Disability Index (HAQ-DI)
Time Frame: 12 months
Measurement of the difference in the quality of life measured by Health Assessment Questionnaire without Disability Index (HAQ-DI). 8 dimensions rated from 0 (without any difficulty) to 3 (unable to do). Overall score from 0 to 3. The higher the score, the lower the quality of life.
12 months
Evaluate quality of life using Duke health profile
Time Frame: 12 months
Measurement of the difference in the quality of life measured by Duke health profile - The Duke. 10 dimensions. Overall score from 0 to 100. The score increase with the quality of life.
12 months
Lower limb quantification of fat replacement of muscle tissue, residual muscle tissue and markers of disease activity using MRI
Time Frame: 12 months
Measurement of the differences in fat fraction value calculated on thigh Dixon MRI pictures
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Assistance Publique - Hôpitaux de Paris

Investigators

  • Study Director: François Jérôme AUTHIER, Pr, Assistance Publique - Hôpitaux de Paris

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 27, 2025

Primary Completion (Estimated)

June 10, 2028

Study Completion (Estimated)

September 10, 2028

Study Registration Dates

First Submitted

July 26, 2024

First Submitted That Met QC Criteria

July 31, 2024

First Posted (Actual)

August 2, 2024

Study Record Updates

Last Update Posted (Actual)

May 29, 2026

Last Update Submitted That Met QC Criteria

May 27, 2026

Last Verified

September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • APHP220829

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Datas are own by assistance publique - hopitaux de paris, please contact sponsor for further information.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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