Baricitinib in Patients With Relapsing or naïve Dermatomyositis
ConditionDermatomyositis
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age18-64
SponsorAssistance Publique - Hôpitaux de Paris
About this trial
Dermatomyositis (DM) is a rare and disabling condition with an important impairment of quality of life and possible life-threatening complications.
Treatment is based on high doses of corticosteroids but this exposes patients to adverse events (cardiovascular mortality, glucocorticoids-induced muscle and skin damages). Corticosteroids taper is associated with disease relapses. Although there is no evidence from the literature, clinical practice guidelines recommends the use of DMARDs such as methotrexate. However, response is not complete and these DMARDS take time to act.
The interferon type I (IFN-I) pathway is involved in the pathophysiology of DM. Janus kinase 1 and 2 transduces IFN-I signals. In addition, JAK2 inhibition enhances muscle repair and force generation.
JAK 1/2 inhibitors permitted to dramatically and rapidly improve relapsing DM patients (n=4, case series).
Our hypothesis is that Janus kinase 1 and 2 (JAK1/2) inhibitors (baricitinib) will permit to obtain dermatomyositis (DM) improvement with a steroid sparing effect as compared to usual care.
Our primary objective is to evaluate the efficacy of baricitinib (JAK1/2 inhibitor) to obtain prednisone-free moderate improvement (ACR/EULAR ≥ 40) of DM as compared to placebo in addition to usual care.
BIRD is a multicenter phase III double blind randomized placebo-controlled trial with two parallel arms (1:1). This is an add-on trial to usual care with rapid corticoid taper.
This is a multicenter trial in different medical departments in hospitals across France in different regions.
Out- and in patients will be recruited in hospital departments involved in management and diagnosis of DM: departments of dermatology, rheumatology and internal medicine.
Eligibility criteria
Qualifiers
Adult subjects (≥ 18 years old) < 65 years old
Dermatomyositis defined according to the 239th ENMC criteria either naïve or non-naïve DM
Manual Muscle Testing (MMT-8) <145/150 and at least two additional abnormal corset measurements (CSM): >3/10 cm on Visual Analogue Scale (VAS) of patient global, physician global and extra-muscular disease activity, Health Assessment Questionnaire Disability Index >0.25, or elevated muscle enzymes.
Or cutaneous CDASI > 20 and at least two additional abnormal corset measurements (CSM): >3/10 cm on Visual Analogue Scale (VAS) of patient global, physician global and extra-muscular disease activity, Health Assessment Questionnaire Disability Index >0.25, or elevated muscle enzymes
Disqualifiers
Severe swallowing troubles defined as: food swallowed the wrong way and/or time to drink a glass of 200 ml water above 30 seconds related to DM.
Interstitial lung disease related to the DM with one among the following complications (complications must be related to the ILD): dyspnea NYHA III, hypoxemia with PaO2≤65 mmHg, and/or DLCOc/Alveolar Volume ≤70% (pulmonary function test)
Symptomatic myocarditis o Loss of walking ability
Patient with deep vein thrombosis/pulmonary embolism or antecedent
Trial design
Treatments tested in this trial
- Baricitinib
- Placebo
Treatment groups
62 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov