Ruxolitinib for people with inclusion body myositis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• CHU Caen Normandie
  Caen, France, France Recruiting
• Hôpital Henri-Mondor, APHP
  Créteil, France, France Recruiting
• Hôpital Roger Salengro, CHU de Lille
  Lille, France, France Recruiting
• Hôpital Dupuytren, CHU de Limoges
  Limoges, France, France Recruiting
• CHU Nancy
  Nancy, France, France Recruiting
• Hôpital Pasteur, CHU de Nice
  Nice, France, France Recruiting

Study contacts

• Study coordinator: François Jérôme AUTHIER, Pr
• Email: francois-jerome.authier@aphp.fr
• Phone: +33 1 4981 2735

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Graft-versus-host disease after stem cell transplant: testing LBP-MARCO with ruxolitinib

  We are testing whether combining LBP-MARCO with ruxolitinib is safe for adults with GVHD after a stem cell transplant.

  Recruiting at 7 sites Phase 1 Memorial Sloan Kettering Cancer Center · Basking Ridge, New Jersey and 6 other locations · NCT07913672

• DMR-001 in CALR-mutant myeloproliferative neoplasms

  This study tests DMR-001 alone for CALR-mutant essential thrombocythemia or myelofibrosis, and in combination with ruxolitinib for CALR-mutant myelofibrosis.

  Recruiting at 3 sites Phase 1 Damora Therapeutics · Birmingham, Alabama and 35 other locations · NCT07849231

• Testing WJ01024 Alone and with Ruxolitinib for Myelofibrosis with Splenomegaly

  This early-phase study tests whether WJ01024, by itself or with ruxolitinib, is safe and tolerable and whether it shows signs of activity in adults with myelofibrosis and enlarged spleen.

  Recruiting Phase 1 Henan Cancer Hospital · Zhengzhou, Henan · NCT07802405

• Belumosudil vs. Ruxolitinib for hepatic cGVHD after HSCT

  This trial tests whether belumosudil or ruxolitinib works better for hepatic chronic GVHD after a stem cell transplant in people 12 and older.

  Recruiting First Affiliated Hospital of Zhejiang University · Hangzhou, Zhejiang · NCT07794514

• Topical ruxolitinib for adults with pyoderma gangrenosum

  This pilot study tests a topical cream of ruxolitinib to see if it helps heal PG ulcers in adults without adding systemic immunosuppressants.

  Recruiting Phase 2 Oregon Health and Science University · Portland, Oregon · NCT07783412

• Ruxolitinib Cream Plus Corticosteroids for Facial Non-Segmental Vitiligo in Real-World Practice

  This real-world study tests whether adding 1.5% ruxolitinib cream to corticosteroids helps people 12 and older with active facial non-segmental vitiligo.

  Recruiting at 6 sites Dermavon Holdings Limited · Shenzhen, Guangdong and 19 other locations · NCT07673640

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.