Ruxolitinib Treatment in Inclusion Body Myositis

NCT ID: NCT06536166

Last Updated: 2025-10-03

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

80 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-05-27

Study Completion Date

2028-09-10

Brief Summary

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Refer to the "Detailed Description" section.

Detailed Description

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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.

Conditions

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Inclusion Body Myositis, Sporadic

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Experimental group

Randomization in experimental group.

Group Type EXPERIMENTAL

Ruxolitinib

Intervention Type DRUG

IBM patients treated by ruxolitinib (JAKAVI®), 15mg per os, twice a day, during 12 months.

Control group

Randomization in control group.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

IBM patients treated by placebo, twice a day, during 12 months.

Interventions

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Ruxolitinib

IBM patients treated by ruxolitinib (JAKAVI®), 15mg per os, twice a day, during 12 months.

Intervention Type DRUG

Placebo

IBM patients treated by placebo, twice a day, during 12 months.

Intervention Type DRUG

Eligibility Criteria

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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.
Minimum Eligible Age

45 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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François Jérôme AUTHIER, Pr

Role: STUDY_DIRECTOR

Assistance Publique - Hôpitaux de Paris

Locations

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Hôpital Pellegrin - Tripode, CHU de Bordeaux

Bordeaux, France, France

Site Status ACTIVE_NOT_RECRUITING

Hôpital Pierre Wertheimer, CHU de Lyon

Bron, France, France

Site Status ACTIVE_NOT_RECRUITING

CHU Caen Normandie

Caen, France, France

Site Status ACTIVE_NOT_RECRUITING

Hôpital Henri-Mondor, APHP

Créteil, France, France

Site Status RECRUITING

Hôpital Raymond Poincaré, APHP

Garches, France, France

Site Status ACTIVE_NOT_RECRUITING

Hôpital Roger Salengro, CHU de Lille

Lille, France, France

Site Status NOT_YET_RECRUITING

Hôpital Dupuytren, CHU de Limoges

Limoges, France, France

Site Status ACTIVE_NOT_RECRUITING

Hôpital de la Timone, APHM

Marseille, France, France

Site Status NOT_YET_RECRUITING

CHU Nancy

Nancy, France, France

Site Status ACTIVE_NOT_RECRUITING

Hôtel-Dieu, CHU Nantes

Nantes, France, France

Site Status NOT_YET_RECRUITING

Hôpital Pasteur, CHU de Nice

Nice, France, France

Site Status RECRUITING

Hôpital Pitié-Salpêtrière, APHP

Paris, France, France

Site Status ACTIVE_NOT_RECRUITING

Hôpital Christian Cabrol, CHU Reims

Reims, France, France

Site Status ACTIVE_NOT_RECRUITING

Hôpital Bellevue, CHU Saint-Etienne

Saint-Etienne, France, France

Site Status ACTIVE_NOT_RECRUITING

Hôpital de Hautepierre, Hôpitaux Universitaires de Strasbourg

Strasbourg, France, France

Site Status NOT_YET_RECRUITING

Hôpital Pierre-Paul Riquet, CHU de Toulouse

Toulouse, France, France

Site Status NOT_YET_RECRUITING

Countries

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France

Central Contacts

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François Jérôme AUTHIER, Pr

Role: CONTACT

+33 1 4981 2735

Facility Contacts

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François Jérôme AUTHIER, Pr

Role: primary

01.49.81.27.35 ext. +33

Céline TARD, Dr.

Role: primary

03.20.44.58.08 ext. +33

Shahram ATTARIAN, Pr.

Role: primary

04.91.38.65.79 ext. +33

Armelle MAGOT, Dr.

Role: primary

02.40.08.36.17 ext. +33

Sabrina SACCONI, Pr.

Role: primary

04.92.03.90.02 ext. +33

Aleksandra NADAJ PAKLEZA, Dr.

Role: primary

03.88.12.85.56 ext. +33

Pascal CINTAS, Dr.

Role: primary

05.61.77.94.40 ext. +33

Other Identifiers

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APHP220829

Identifier Type: -

Identifier Source: org_study_id

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