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New drug combo shows promise for rare childhood muscle disease

NCT ID NCT05524311

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

16 people

The number who actually took part.

Started

Nov 2022

Finished

Jan 2026

Lead sponsor

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

3 to 18 years

Sex

Anyone

Healthy volunteers

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

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

  • Hopital Necker - Enfants malades : unité d'immuno-hématologie et rhumatologie

    Paris, France

  • Hôpital Brabois

    Nancy, France

  • Hôpital Femme Mère Enfant

    Bron, France

  • Hôpital Jeanne de Flandre

    Lille, France

  • Hôpital La Timone

    Marseille, France

  • Hôpital Necker - Enfants malades : service de dermatologie

    Paris, France

  • Hôpital Pellegrin

    Bordeaux, France

  • Hôpital Purpan

    Toulouse, France

  • Hôpital Robert Debré

    Paris, France

  • Hôpital Trousseau

    Paris, France

  • Hôpital Villeneuce

    Montpellier, France

  • Hôpital de Hautepierre

    Strasbourg, France

  • Hôpital du Kremlin-Bicêtre

    Paris, France

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