Baricitinib in the Treatment of New-onset Juvenile Dermatomyositis (MYOCIT)

Assistance Publique - Hôpitaux de Paris

Status and phase

Enrolling

Phase 2

Conditions

Juvenile Dermatomyositis

Treatments

Behavioral: Childhood Health Assessment Questionnaire

Behavioral: Parent version of the Child Health Questionnaire (CHQ)

Biological: pharmacokinetics study

Biological: dosage of cytokines

Biological: Pregnancy test

Biological: transcriptomic analysis

Drug: Baricitinib

Study type

Interventional

Funder types

Other

Identifiers

NCT05524311

2022-000506-10 (EudraCT Number)

APHP211036

Details and patient eligibility

About

The MYOCIT study aims to evaluate the efficacy and safety of baricitinib in association with corticosteroids in new-onset patients with juvenile dermatomyositis (JDM) in a phase II trial with the objective to obtain a better efficacy than the conventional combination methotrexate (MTX) and corticosteroids over the 24 week study period. Thus, the investigators hypothesize that baricitinib could be used as a first line treatment in all forms of DMJ, including the most severe one, with a good safety profile.

Full description

Juvenile dermatomyositis (JDM) is a rare and severe paediatric-onset idiopathic inflammatory myopathy, associated with significant morbidity and mortality. The combination of corticosteroids and methotrexate (MTX) is recommended in new-onset JDM according to one randomized trial. However, in this trial, treatment failures were reported in 13/46 (28%) patients and severe JDM, (cutaneous or gastrointestinal ulceration, interstitial pulmonary disease, cardiomyopathy) were not taken into account. These data emphasize the need for a more efficient first-line treatment. Considering: 1) the strong implication of type IFN-I in the pathophysiology of JDM 2) the report of the efficacy and safety of JAK inhibitors (JAKis) (baricitinb, tofacitinib) in about 50 refractory DM patients, and 9 JDM, a trial which evaluates the efficacy and safety of baricitinib in combination with corticosteroids in new-onset JDM is warranted.

Enrollment

16 estimated patients

Sex

All

Ages

3 to 18 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria:

Patient aged 3-18 years with new-onset juvenile dermatomyositis, according to the ENMC 2018 dermatomyositis classification criteria
Muscle weakness at MMT and/or CMAS (MMT < 74 and/or CMAS < 45)
Seropositivity or vaccination for chickenpox
For patients of childbearing age (following menarche) : Negative βHCG and effective method of contraception (sexual abstinence, hormonal contraception, intrauterine device or hormone-releasing system, cap, diaphragm or sponge with spermicide, condom) until the 7 days after administration of the last dose of Baricitinib
Informed consent form signed by the patient or child' s parents Patient affiliated to a social security regime

Exclusion Criteria

Amyopathic dermatomyositis (without muscle weakness)
Inability to be treated by oral way or to take pills
Previous treatment with JAK inhibitor
Previous treatment of JDM with immunosuppressive drugs or biologics other than corticosteroids. Previous treatment with prednisone was allowed for no more than 1 month.
Previous history of cancer
Live vaccine within the 4 weeks before starting baricitinib therapy
Current, or recent (< 4 weeks prior to baseline) of active infections according to investigator appreciation, but necessarily, including HBV, HCV, HIV, tuberculosis.
Positive blood CMV PCR
Creatinine clearance < 40 ml/min
Lymphocytes < 0,5x109 cell/L and Neutrophils < 1x109 cell/L
Hemoglobin < 8 g/dL
Symptomatic herpes herpes simplex infection within 12 weeks prior to inclusion
History of thrombosis or considered at high risk of venous thrombosis by the investigator
Presence of severe JDM-related involvements: cardiovascular (requiring vasopressive drug and/or intensive care unit), respiratory (requiring oxygen and/or intensive care unit), gastrointestinal (requiring abdominal surgery).
History of severe non-related JDM involvement: cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, hematological, neurological or neuropsychiatric disorders or any other serious and/or instable illness that, in the opinion of the investigator, could constitute an unacceptable risk, when taking baricitinib.
Actual or in project of pregrancy and breast-feeding until the 7 days after administration of the last dose of Baricitinib
Patient on AME (state medical aid)
Participation in another interventional study involving human participants or being in the exclusion period at the end of a previous study involving human participants