New drug combo shows promise for rare childhood muscle disease
NCT ID NCT05524311
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested the drug baricitinib, given with steroids, in 16 children aged 3-18 with newly diagnosed juvenile dermatomyositis, a rare disease causing muscle weakness and skin rashes. The goal was to see if baricitinib works better than the standard treatment (methotrexate plus steroids) at improving muscle strength and daily activities over 24 weeks. The approach aims to control the disease with a good safety profile, but ongoing management is still needed.
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Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
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16 people
The number who actually took part.
- Started
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Nov 2022
- Finished
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Jan 2026
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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3 to 18 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Hopital Necker - Enfants malades : unité d'immuno-hématologie et rhumatologie
Paris, France
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Hôpital Brabois
Nancy, France
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Hôpital Femme Mère Enfant
Bron, France
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Hôpital Jeanne de Flandre
Lille, France
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Hôpital La Timone
Marseille, France
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Hôpital Necker - Enfants malades : service de dermatologie
Paris, France
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Hôpital Pellegrin
Bordeaux, France
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Hôpital Purpan
Toulouse, France
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Hôpital Robert Debré
Paris, France
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Hôpital Trousseau
Paris, France
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Hôpital Villeneuce
Montpellier, France
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Hôpital de Hautepierre
Strasbourg, France
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Hôpital du Kremlin-Bicêtre
Paris, France
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