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

NCT ID: NCT05524311

Last Updated: 2025-11-20

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

16 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-11-10

Study Completion Date

2026-09-30

Brief Summary

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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

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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

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Juvenile Dermatomyositis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Baricitinib

Group Type EXPERIMENTAL

Baricitinib

Intervention Type DRUG

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 study

Intervention Type BIOLOGICAL

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

dosage of cytokines

Intervention Type BIOLOGICAL

additionnal blood sampling at weeks 0, 4 and 24

transcriptomic analysis

Intervention Type BIOLOGICAL

additionnal blood sampling at weeks 0, 4 and 24

Parent version of the Child Health Questionnaire (CHQ)

Intervention Type BEHAVIORAL

Evaluate by parents at each visits

Childhood Health Assessment Questionnaire

Intervention Type BEHAVIORAL

Evaluate by parents at each visits

Pregnancy test

Intervention Type BIOLOGICAL

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

Interventions

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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

Intervention Type DRUG

pharmacokinetics study

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

Intervention Type BIOLOGICAL

dosage of cytokines

additionnal blood sampling at weeks 0, 4 and 24

Intervention Type BIOLOGICAL

transcriptomic analysis

additionnal blood sampling at weeks 0, 4 and 24

Intervention Type BIOLOGICAL

Parent version of the Child Health Questionnaire (CHQ)

Evaluate by parents at each visits

Intervention Type BEHAVIORAL

Childhood Health Assessment Questionnaire

Evaluate by parents at each visits

Intervention Type BEHAVIORAL

Pregnancy test

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

Intervention Type BIOLOGICAL

Eligibility Criteria

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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
Minimum Eligible Age

3 Years

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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URC-CIC Paris Descartes Necker Cochin

OTHER

Sponsor Role collaborator

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Cyril GITIAUX, Doctor

Role: STUDY_DIRECTOR

Assistance Publique - Hôpitaux de Paris

Locations

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Hôpital Pellegrin

Bordeaux, , France

Site Status RECRUITING

Hôpital Femme Mère Enfant

Bron, , France

Site Status RECRUITING

Hôpital Jeanne de Flandre

Lille, , France

Site Status RECRUITING

Hôpital La Timone

Marseille, , France

Site Status RECRUITING

Hôpital Villeneuce

Montpellier, , France

Site Status NOT_YET_RECRUITING

Hôpital Brabois

Nancy, , France

Site Status RECRUITING

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

Paris, , France

Site Status RECRUITING

Hôpital du Kremlin-Bicêtre

Paris, , France

Site Status RECRUITING

Hôpital Necker - Enfants malades : service de dermatologie

Paris, , France

Site Status RECRUITING

Hôpital Robert Debré

Paris, , France

Site Status RECRUITING

Hôpital Trousseau

Paris, , France

Site Status RECRUITING

Hôpital de Hautepierre

Strasbourg, , France

Site Status NOT_YET_RECRUITING

Hôpital Purpan

Toulouse, , France

Site Status RECRUITING

Countries

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France

Central Contacts

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Brigitte BADER-MEUNIER, Doctor

Role: CONTACT

+33 1 44 49 43 32

Laure CHOUPEAUX, MSc

Role: CONTACT

+33 1 44 38 17 11

Facility Contacts

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Pascal PILLET, Docteur

Role: primary

Alexandre BELOT, Professeur

Role: primary

Héloïse REUMAUX, Docteur

Role: primary

Frédérique AUDIC, Docteur

Role: primary

Aurélia CARBASSE, Docteur

Role: primary

Irène LEMELLE, Docteur

Role: primary

Brigitte BADER-MEUNIER, Docteur

Role: primary

Isabelle KONE-PAUT, Professeur

Role: primary

Anne WELFRINGER, Professeur

Role: primary

Isabelle MELKI, Docteur

Role: primary

Arnaud ISAPOF, Docteur

Role: primary

Alain MEYER, Docteur

Role: primary

Karine BROCHARD, Docteur

Role: primary

References

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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.

Reference Type BACKGROUND
PMID: 18163404 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 22736096 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 12579588 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 31118099 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 31791867 (View on PubMed)

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.

Reference Type BACKGROUND
PMID: 7986222 (View on PubMed)

Other Identifiers

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2022-000506-10

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

APHP211036

Identifier Type: -

Identifier Source: org_study_id

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