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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
80

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

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 Côte 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Assistance Publique - Hôpitaux de Paris

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: François Jérôme AUTHIER, Pr, Assistance Publique - Hopitaux 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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
May 27, 2025

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 10, 2028

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 10, 2028

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 26, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 31, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
August 2, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 29, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 27, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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