Baricitinib in Patients With Relapsing or naïve Dermatomyositis

NCT04972760 | Recruiting Phase 3 DMC

• 2 other identifiers: APHP1806122020-004987-24
• Study Type: interventional
• Target: 62
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Dermatomyositis

Keywords

Dermatomyositis; baricitinib; steroid sparing

Outcome Measures

Primary Outcomes (1)

• Moderate improvement at 24 weeks without prednisone: prednisone-free moderate improvement.

  The rate of patients with a moderate improvement at 24 weeks, defined as a total improvement score superior or equal to 40 following ACR/EULAR definition. The investigator will consider that patients following the planned prednisone tapering scheme (corticosteroids 0 mg/d at W24) will be prednisone-free.

  24 weeks

Secondary Outcomes (9)

• Dermatomyositis minimal improvement

  5, 12 and 24 weeks

• Dermatomyositis moderate improvement

  5, 12 and 24 weeks

• Dermatomyositis major improvement

  5, 12 and 24 weeks

• Primary endpoint prednisone-free moderate improvement at Weeks 24 in subgroups

  24 weeks

• Cutaneous disease activity and damage

  5, 12 and 24 weeks

Study Arms (2)

baricitinib arm

EXPERIMENTAL

Patients receive baricitinib plus prednisone taper plus one immunosuppressive drug (either methotrexate or azathioprine) for a duration of 24 weeks. Corticosteroids are tapered following a predefined protocol.

Drug: Baricitinib

placebo arm

PLACEBO COMPARATOR

Patients receive placebo plus prednisone taper plus one immunosuppressive drug (either methotrexate or azathioprine) for a duration of 24 weeks. Corticosteroids are tapered following a predefined protocol.

Drug: Placebo

Interventions

Baricitinib DRUG

Baricitinib, 4 mg/d, oral route for 24 weeks

baricitinib arm

Placebo DRUG

Placebo, 4 mg/d, oral route for 24 weeks

placebo arm

Eligibility Criteria

• Age: 18 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Adult subjects (≥ 18 years old) \< 65 years old
• Dermatomyositis defined according to the 239th ENMC criteria either naïve or non-naïve DM
• Active disease (ACR/EULAR criteria) defined as :
• 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
• for relapsing/non naïve DM patients :
• in case of corticosteroid exposure patient must receive a stable dose \< 30 mg/d prednisone with or without additional immunosuppressive therapy for at least 4 weeks before the baseline visit.
• Stable dose of immunosuppressive therapy for at least 3 months before
• Affiliation to a social security regime
• Written informed consent

You may not qualify if:

• Life-threatening complications :
• 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
• Patient with antecedent of cardiovascular event (myocardial infarction or ischemic stroke)
• Patient who is current or past long-time smoker
• Pregnant or lactating, or women planning to become pregnant or initiating breastfeeding
• No effective contraception during the study and one week after for women of childbearing age
• Renal impairment defined as clearance \< 60 ml
• Strong Organic Anion Transporter 3 (OAT3) inhibitors
• Active cancer or history of malignancy
• Active severe infection including active hepatitis
• Evidence of latent tuberculosis (as documented by a positive QuantiFERON-TB Gold plus test)
• Absolute Neutrophil Count \< 1x109 cells/L

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Pitie-Salpêtrière hospital APHP

Paris, 75013, France

RECRUITING

MeSH Terms

Conditions

Dermatomyositis

Interventions

baricitinib

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Study Officials

• YVES ALLENBACH, MD, PhD

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Central Study Contacts

YVES ALLENBACH, MD, PhD

CONTACT

00 33 1 42 16 10 68; yves.allenbach@aphp.fr

SARRA POCHON

CONTACT

00 33 1 42 16 75 74; sarra.pochon@aphp.fr

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 14, 2021
• First Posted: July 22, 2021
• Study Start: August 31, 2022
• Primary Completion: February 28, 2026
• Study Completion: February 28, 2026
• Last Updated: August 11, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF
• Time Frame: Beginning 3 months and ending 3 years following article publication. Requests out of these time frame can also be submitted to the sponsor
• Access Criteria: Researchers who provide a methodological sound proposal.

Locations

FR (1)