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Ruxolitinib for people with inclusion body myositis

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

PHASE2 · Assistance Publique - Hôpitaux de Paris · NCT06536166

This trial will test whether ruxolitinib can help improve walking distance, muscle strength, and breathing in adults 45 and older with inclusion body myositis.

Quick facts

PhasePHASE2
Study typeInterventional
Enrollment80 (estimated)
Ages45 Years and up
SexAll
SponsorAssistance Publique - Hôpitaux de Paris (other)
Drugs / interventionsruxolitinib
Locations16 sites (Bordeaux, France and 15 other locations)
Trial IDNCT06536166 on ClinicalTrials.gov

What this trial studies

This is a randomized, double-blind, placebo-controlled Phase 2 trial enrolling 60 participants with biopsy-proven inclusion body myositis who can walk for at least six minutes. Participants are randomized 1:1 to receive ruxolitinib 15 mg twice daily or placebo for 12 months, with regular clinic visits. Outcomes include the 6-minute walk test, quantitative muscle strength, functional scales, respiratory function tests, and muscle MRI. The trial is based on preclinical data showing a strong IFNγ/JAK-STAT signature in IBM muscle and that JAK inhibition can prevent IFNγ-related muscle suppression in models.

Who should consider this trial

Good fit: Adults aged 45 or older with biopsy-confirmed inclusion body myositis who can walk for at least six minutes (with or without assistive devices) and can give informed consent are ideal candidates.

Not a fit: People who are non-ambulatory, pregnant or breastfeeding, have significant cognitive impairment, or do not meet the biopsy-based diagnostic criteria are unlikely to benefit from participation.

Why it matters

Potential benefit: If successful, ruxolitinib could slow muscle decline and improve mobility, strength, and quality of life for people with IBM.

How similar studies have performed: Preclinical studies and some early reports support JAK inhibition in IBM, but large randomized controlled evidence in IBM is currently limited, making this approach relatively novel in this disease.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Bordeaux, France and 15 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.

View on ClinicalTrials.gov →

Conditions: Inclusion Body Myositis, Sporadic, Inclusion body myositis, Idiopathic immune myopathy, JAK-inhibitor, Ruxolitinib

Last reviewed 2026-05-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.