Using baricitinib to treat new-onset juvenile dermatomyositis
Baricitinib in the Treatment of New-onset Juvenile Dermatomyositis
PHASE2 · Assistance Publique - Hôpitaux de Paris · NCT05524311
This study is testing if a new medication called baricitinib, used with steroids, can help children with newly diagnosed juvenile dermatomyositis feel better compared to the usual treatment.
Quick facts
| Phase | PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 16 (estimated) |
| Ages | 3 Years to 18 Years |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris (other) |
| Drugs / interventions | Baricitinib, prednisone, methotrexate, tofacitinib |
| Locations | 13 sites (Bordeaux and 12 other locations) |
| Trial ID | NCT05524311 on ClinicalTrials.gov |
What this trial studies
The MYOCIT study evaluates the efficacy and safety of baricitinib combined with corticosteroids in children aged 3-18 with new-onset juvenile dermatomyositis (JDM). This phase II trial aims to demonstrate that baricitinib can provide better outcomes than the conventional treatment of methotrexate and corticosteroids over a 24-week period. The study is motivated by the need for a more effective first-line treatment, especially for severe cases of JDM. Participants will undergo assessments including pharmacokinetics, cytokine dosage, and transcriptomic analysis.
Who should consider this trial
Good fit: Ideal candidates are children aged 3-18 years with newly diagnosed juvenile dermatomyositis exhibiting muscle weakness.
Not a fit: Patients with amyopathic dermatomyositis or those who have previously received JAK inhibitors or other immunosuppressive treatments may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could offer a more effective first-line option for children with juvenile dermatomyositis, potentially improving their health outcomes.
How similar studies have performed: While there have been reports of JAK inhibitors showing efficacy in refractory dermatomyositis cases, this specific approach in new-onset JDM is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / 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
Where this trial is running
Bordeaux and 12 other locations
- Hôpital Pellegrin — Bordeaux, France (RECRUITING)
- Hôpital Femme Mère Enfant — Bron, France (RECRUITING)
- Hôpital Jeanne de Flandre — Lille, France (RECRUITING)
- Hôpital La Timone — Marseille, France (RECRUITING)
- Hôpital Villeneuce — Montpellier, France (NOT_YET_RECRUITING)
- Hôpital Brabois — Nancy, France (RECRUITING)
- Hopital Necker - Enfants malades : unité d'immuno-hématologie et rhumatologie — Paris, France (RECRUITING)
- Hôpital du Kremlin-Bicêtre — Paris, France (RECRUITING)
- Hôpital Necker - Enfants malades : service de dermatologie — Paris, France (RECRUITING)
- Hôpital Robert Debré — Paris, France (RECRUITING)
- Hôpital Trousseau — Paris, France (RECRUITING)
- Hôpital de Hautepierre — Strasbourg, France (NOT_YET_RECRUITING)
- Hôpital Purpan — Toulouse, France (RECRUITING)
Study contacts
- Study coordinator: Brigitte BADER-MEUNIER, Doctor
- Email: brigitte.bader-meunier@aphp.fr
- Phone: +33 1 44 49 43 32
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Juvenile Dermatomyositis, juvenile dermatomyositis, baricitinib