Refer to the "Detailed Description" section.
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
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
80
IBM patients treated by ruxolitinib (JAKAVI®), 15mg per os, twice a day, during 12 months.
IBM patients treated by placebo, twice a day, during 12 months.
Hôpital Pellegrin - Tripode, CHU de Bordeaux
Bordeaux, France, France
ACTIVE_NOT_RECRUITINGHôpital Pierre Wertheimer, CHU de Lyon
6 minutes-walk distance (6MWT): A distance walked in 6 min. superior in treated patients compared to placebo group
The 6MWT is performed in a corridor, between two cones separated by a distance of 25 m.
Time frame: 12 months
Adverse events (safety and tolerability) of ruxolitinib in IBM patients
Adverse events collected according to the MedDRA classification
Time frame: Through study completion that is to say 15 months
Therapeutic muscular efficacy of ruxolitinib on muscle strength
Quantification of muscle strength using dynamometer
Time frame: Until last consultation that is to say 12 months
Therapeutic muscular efficacy of ruxolitinib on overall muscle status
Measurement of overall muscle status using scales and serum creatine kinase (CK) levels
Time frame: Until last consultation that is to say 12 months
Respiratory ability
Forced vital capacity (FVC) measurement
Time frame: 12 months
Evaluate swallowing using Swallowing Disturbance Questionnaire (SDQ)
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.
Time frame: 12 months
Lower limb quantification of fat replacement of muscle tissue, residual muscle tissue and markers of disease activity using MRI
Measurement of the differences in fat fraction value calculated on thigh Dixon MRI pictures
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Bron, France, France
CHU Caen Normandie
Caen, France, France
ACTIVE_NOT_RECRUITINGHôpital Henri-Mondor, APHP
Créteil, France, France
RECRUITINGHôpital Raymond Poincaré, APHP
Garches, France, France
ACTIVE_NOT_RECRUITINGHôpital Roger Salengro, CHU de Lille
Lille, France, France
NOT_YET_RECRUITINGHôpital Dupuytren, CHU de Limoges
Limoges, France, France
ACTIVE_NOT_RECRUITINGHôpital de la Timone, APHM
Marseille, France, France
NOT_YET_RECRUITINGCHU Nancy
Nancy, France, France
ACTIVE_NOT_RECRUITINGHôtel-Dieu, CHU Nantes
Nantes, France, France
NOT_YET_RECRUITING...and 6 more locations
Time frame: 12 months
Evaluate quality of life using Health Assessment Questionnaire without Disability Index (HAQ-DI)
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.
Time frame: 12 months
Evaluate quality of life using Duke health profile
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.
Time frame: 12 months