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Juvenile DermatomyositisPhase 2MYOCITNCT05524311

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

A Phase 2 study testing Baricitinib, pharmacokinetics study and dosage of cytokines in people with Juvenile Dermatomyositis, run by Assistance Publique - Hôpitaux de Paris at 13 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 3 to 18 years
Conditions the study is for
Juvenile Dermatomyositis
What is being tested?
Baricitinib, pharmacokinetics study and dosage of cytokines
Could I receive a placebo?
No, everyone receives an active treatment
How many people
16 participants
Where
13 sitesFrance
Study dates
10 Nov 2022to 2 Jan 2026
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 3 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 18Show fewer
  • 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

Trial locations and contacts

13 sites in 1 country.

13 sites in 1 country

France13 sites

  • Hôpital Pellegrin

    Bordeaux

  • Hôpital Femme Mère Enfant

    Bron

  • Hôpital Jeanne de Flandre

    Lille

  • Hôpital La Timone

    Marseille

  • Hôpital Villeneuce

    Montpellier

  • Hôpital Brabois

    Nancy

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

    Paris

  • Hôpital du Kremlin-Bicêtre

    Paris

  • Hôpital Necker - Enfants malades : service de dermatologie

    Paris

  • Hôpital Robert Debré

    Paris

  • Hôpital Trousseau

    Paris

  • Hôpital de Hautepierre

    Strasbourg

  • Hôpital Purpan

    Toulouse

Other trials in France

About this study

Official title: Baricitinib in the Treatment of New-onset Juvenile Dermatomyositis

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.

Read the 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.

Being tested
Baricitinib, pharmacokinetics study, dosage of cytokines, transcriptomic analysis, Parent version of the Child Health Questionnaire (CHQ)
Keywords
juvenile dermatomyositis, baricitinib
Medical subject headings
Dermatomyositis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BaricitinibDrug

    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

  • pharmacokinetics studyBiological

    additionnal blood sampling at week 4, 8, 12, and 24

  • dosage of cytokinesBiological

    additionnal blood sampling at weeks 0, 4 and 24

  • transcriptomic analysisBiological

    additionnal blood sampling at weeks 0, 4 and 24

  • Parent version of the Child Health Questionnaire (CHQ)Behavioural

    Evaluate by parents at each visits

  • Childhood Health Assessment QuestionnaireBehavioural

    Evaluate by parents at each visits

  • Pregnancy testBiological

    Urine pregnancy test at V4 A dosage of bHCG with current biological analysis is done at each visit (except V4)

Study groups in NCT05524311
Study groupRoleWhat they receive
BaricitinibExperimentalNot described
Receives: Drug: Baricitinib; Biological: pharmacokinetics study; Biological: dosage of cytokines; Biological: transcriptomic analysis; Behavioral: Parent version of the Child Health Questionnaire (CHQ); Behavioral: Childhood Health Assessment Questionnaire; Biological: Pregnancy test

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. PRINTO 20 (Paediatric Rheumatology INternational Trials Organisation scale)

    Achievement of the validated juvenile dermatomyositis PRINTO 20 level of improvement. PRINTO 20 level of improvement is defined as a 20% or greater improvement in three or more of the six variables of the juvenile dermatomyositis core set, with one or no variable worsening by more than 30% (muscle strength can not be the variable worsening) :

    1. muscle strength, assessed with the Childhood Myositis Assessment Scale (CMAS),
    2. physician's global assessment of the patient's disease activity (Physician's VAS)
    3. global disease activity assessment through the Disease Activity Score (DAS)
    4. functional ability through the Childhood Health Assessment Questionnaire (C-HAQ)
    5. parent's global assessment of the child's overall wellbeing (Parent's VAS)
    6. health-related quality of life, through the parent version of the Child Health Questionnaire (CHQ-Phs) A higher score is 100% and means a better outcome, a lower score is 0% and means a worse result.

    Measured: At week 24

Secondary outcomes (17)

  1. PRINTO 20 Paediatric Rheumatology INternational Trials Organisation scale - level 20

    achievement of the validated juvenile dermatomyositis PRINTO 20 level ; reaching a minimum of 20 % a higher score is 100% and mean a better outcome a lower score is 0% and means a worse result

    Measured: At week 4, 8, 12 and 16

  2. PRINTO 50 Paediatric Rheumatology INternational Trials Organisation scale - level 50

    achievement of the validated juvenile dermatomyositis PRINTO 50 level ; reaching a minimum of 50 % a higher score is 100% and mean a better outcome a lower score is 0% and means a worse result

    Measured: At week 4, 8, 12 and 16

  3. PRINTO 70 Paediatric Rheumatology INternational Trials Organisation scale - level 70

    achievement of the validated juvenile dermatomyositis PRINTO 70 level ; reaching a minimum of 70 % a higher score is 100% and mean a better outcome a lower score is 0% and means a worse result

    Measured: At week 4, 8, 12 and 16

  4. PRINTO 90 Paediatric Rheumatology INternational Trials Organisation scale - level 90

    achievement of the validated juvenile dermatomyositis PRINTO 90 level ; reaching a minimum of 90 % a higher score is 100% and mean a better outcome a lower score is 0% and means a worse result

    Measured: At week 4, 8, 12 and 16

  5. Total Improvement Score (TIS)

    Relative and absolute variations of TIS. The TIS is the sum of the improvement in each of the six core set measures of disease activits The minimum score is 0 (worse) and maximum score is 100 (better) A major response is defined by a score > 70 A moderate response is defined by a score > 45 A minimal response is defined by a score > 30

    Measured: At inclusion, weeks 4, 8, 12, 16 and 24

  6. Clinically inactive disease

    according to the PRINTO criteria

    Measured: At weeks 4, 8, 12 and 24

  7. Cutaneous Dermatomyositis Disease Area and Severity Index (CDSAI)

    assess skin activity and damage across multiple body regions in patients with dermatomyositis

    Measured: At inclusion, weeks 4, 8, 12, 16 and 24

  8. Myositis Disease Activity Assessment VAS (MYOACT)

    Assess Relative and absolute variations of extramuscular activity

    Measured: At inclusion, weeks 4, 8, 12, 16 and 24

  9. interstitial lung disease

    Assessed by improvement of at least 10% of FCV, PTC, and DLCO and/or improvement of Lung tomodensitomery according to a specific scale

    Measured: At inclusion and at week 24

  10. Dose of corticosteroid

    Dose tapering at 6 months

    Measured: At week 24

  11. Pharmacokinetics study

    Non-compartmental analysis of baricitinib

    Measured: At weeks 4, 8, 12, and 24

  12. Pharmacokinetics (PK) study with area under the curve

    Correlation between area under the curve AUC in ng.h/ml (PK parameter of baricitinib) and disease activity 's scores

    Measured: At weeks 4, 8, 12, and 24

  13. Pharmacokinetics (PK) study with maximal concentration

    Correlation between maximal concentration Cmax in ng/mL (PK parameter of baricitinib) and disease activity 's scores

    Measured: At weeks 4, 8, 12, and 24

  14. Pharmacokinetics (PK) study with through concentration

    Correlation between through concentration Ctrough, in ng/mL (PK parameter of baricitinib) and disease activity 's scores

    Measured: At weeks 4, 8, 12, and 24

  15. dosage of cytokines

    Measurement of serum IFN -, IFN-γ, IL-1β, IL-4, Il-5, IL-6, IL-8, IL-10, IL-12p70, IL-22, TNF α

    Measured: At inclusion, weeks 4 and 24

  16. transcriptomic analysis

    Study of genes expression within 800 genes related to immunity

    Measured: At inclusion, weeks 4 and 24

  17. Biopsy

    Assessment of muscle biopsies according to the internationally validated score system

    Measured: At inclusion, weeks 4 and 24

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Sep 2022
  2. Study start10 Nov 2022
  3. Primary completion13 May 2025
  4. Study completion2 Jan 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05524311
Sponsor's protocol ID
APHP211036
Other IDs
2022-000506-10
Acronym
MYOCIT
Status last verified
Mar 2026
First submitted
5 Jul 2022
Last updated
23 Apr 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Ruperto N, Ravelli A, Pistorio A, Ferriani V, Calvo I, Ganser G, Brunner J, Dannecker G, Silva CA, Stanevicha V, Cate RT, van Suijlekom-Smit LW, Voygioyka O, Fischbach M, Foeldvari I, Hilario O, Modesto C, Saurenmann RK, Sauvain MJ, Scheibel I, Sommelet D, Tambic-Bukovac L, Barcellona R, Brik R, Ehl S, Jovanovic M, Rovensky J, Bagnasco F, Lovell DJ, Martini A; Paediatric Rheumatology International Trials Organisation (PRINTO); Pediatric Rheumatology Collaborative Study Group (PRCSG). The provisional Paediatric Rheumatology International Trials Organisation/American College of Rheumatology/European League Against Rheumatism Disease activity core set for the evaluation of response to therapy in juvenile dermatomyositis: a prospective validation study. Arthritis Rheum. 2008 Jan 15;59(1):4-13. doi: 10.1002/art.23248.

    PubMed 18163404

  • Lazarevic D, Pistorio A, Palmisani E, Miettunen P, Ravelli A, Pilkington C, Wulffraat NM, Malattia C, Garay SM, Hofer M, Quartier P, Dolezalova P, Penades IC, Ferriani VP, Ganser G, Kasapcopur O, Melo-Gomes JA, Reed AM, Wierzbowska M, Rider LG, Martini A, Ruperto N; Paediatric Rheumatology International Trials Organisation (PRINTO). The PRINTO criteria for clinically inactive disease in juvenile dermatomyositis. Ann Rheum Dis. 2013 May;72(5):686-93. doi: 10.1136/annrheumdis-2012-201483. Epub 2012 Jun 26.

    PubMed 22736096

  • Bode RK, Klein-Gitelman MS, Miller ML, Lechman TS, Pachman LM. Disease activity score for children with juvenile dermatomyositis: reliability and validity evidence. Arthritis Rheum. 2003 Feb 15;49(1):7-15. doi: 10.1002/art.10924.

    PubMed 12579588

  • Giancane G, Lavarello C, Pistorio A, Oliveira SK, Zulian F, Cuttica R, Fischbach M, Magnusson B, Pastore S, Marini R, Martino S, Pagnier A, Soler C, Stanevicha V, Ten Cate R, Uziel Y, Vojinovic J, Fueri E, Ravelli A, Martini A, Ruperto N; Paediatric Rheumatology International Trials Organisation (PRINTO). The PRINTO evidence-based proposal for glucocorticoids tapering/discontinuation in new onset juvenile dermatomyositis patients. Pediatr Rheumatol Online J. 2019 May 22;17(1):24. doi: 10.1186/s12969-019-0326-5.

    PubMed 31118099

  • Mammen AL, Allenbach Y, Stenzel W, Benveniste O; ENMC 239th Workshop Study Group. 239th ENMC International Workshop: Classification of dermatomyositis, Amsterdam, the Netherlands, 14-16 December 2018. Neuromuscul Disord. 2020 Jan;30(1):70-92. doi: 10.1016/j.nmd.2019.10.005. Epub 2019 Oct 25. No abstract available.

    PubMed 31791867

  • Singh G, Athreya BH, Fries JF, Goldsmith DP. Measurement of health status in children with juvenile rheumatoid arthritis. Arthritis Rheum. 1994 Dec;37(12):1761-9. doi: 10.1002/art.1780371209.

    PubMed 7986222

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Baricitinib, pharmacokinetics study and dosage of cytokines in people with Juvenile Dermatomyositis, run by Assistance Publique - Hôpitaux de Paris at 13 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 23 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 13 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

PRINTO 20 (Paediatric Rheumatology INternational Trials Organisation scale) (time frame: At week 24). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05524311, which also lists 6 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Apr 2026.

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