NCT05524311

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

Baricitinib in the Treatment of New-onset Juvenile Dermatomyositis

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 16 (actual)
• Sponsor: Assistance Publique - Hôpitaux de Paris · Academic / Other
• Sex: All
• Age: 3 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed 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.

Conditions

• Juvenile Dermatomyositis

Interventions

Type	Name	Description
DRUG	Baricitinib	Oral tablets (2 mg) will be used For children \> or = 6 years: 4 mg once a day (2 x 2 mg) during the 24 weeks-period study For children \< 6 years: 2 mg once a day during the 24 weeks -period study
BIOLOGICAL	pharmacokinetics study	additionnal blood sampling at week 4, 8, 12, and 24
BIOLOGICAL	dosage of cytokines	additionnal blood sampling at weeks 0, 4 and 24
BIOLOGICAL	transcriptomic analysis	additionnal blood sampling at weeks 0, 4 and 24
BEHAVIORAL	Parent version of the Child Health Questionnaire (CHQ)	Evaluate by parents at each visits
BEHAVIORAL	Childhood Health Assessment Questionnaire	Evaluate by parents at each visits
BIOLOGICAL	Pregnancy test	Urine pregnancy test at V4 A dosage of bHCG with current biological analysis is done at each visit (except V4)

Timeline

• Start date: 2022-11-10
• Primary completion: 2025-05-13
• Completion: 2026-01-02
• First posted: 2022-09-01
• Last updated: 2026-03-23

Locations

13 sites across 1 country: France