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

NCT05524311 | Completed Phase 2 DMC

• 2 other identifiers: APHP2110362022-000506-10
• Study Type: interventional
• Target: 16
• Locations: 1 country
• Sites: 13

Brief 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 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.

Conditions

Juvenile Dermatomyositis

Keywords

juvenile dermatomyositis; baricitinib

Outcome Measures

Primary Outcomes (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.

  At week 24

Secondary Outcomes (17)

• PRINTO 20 Paediatric Rheumatology INternational Trials Organisation scale - level 20

  At week 4, 8, 12 and 16

• PRINTO 50 Paediatric Rheumatology INternational Trials Organisation scale - level 50

  At week 4, 8, 12 and 16

• PRINTO 70 Paediatric Rheumatology INternational Trials Organisation scale - level 70

  At week 4, 8, 12 and 16

• PRINTO 90 Paediatric Rheumatology INternational Trials Organisation scale - level 90

  At week 4, 8, 12 and 16

• Total Improvement Score (TIS)

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

Study Arms (1)

Baricitinib

EXPERIMENTAL

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

Interventions

Baricitinib 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

Baricitinib

Pregnancy test BIOLOGICAL

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

Baricitinib

pharmacokinetics study BIOLOGICAL

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

Baricitinib

dosage of cytokines BIOLOGICAL

additionnal blood sampling at weeks 0, 4 and 24

Baricitinib

transcriptomic analysis BIOLOGICAL

additionnal blood sampling at weeks 0, 4 and 24

Baricitinib

Parent version of the Child Health Questionnaire (CHQ) BEHAVIORAL

Evaluate by parents at each visits

Baricitinib

Childhood Health Assessment Questionnaire BEHAVIORAL

Evaluate by parents at each visits

Baricitinib

Eligibility Criteria

• Age: 3 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead
• URC-CIC Paris Descartes Necker Cochin: collaborator

Study Sites (13)

Hôpital Pellegrin

Bordeaux, France

Location

Hôpital Femme Mère Enfant

Bron, France

Location

Hôpital Jeanne de Flandre

Lille, France

Location

Hôpital La Timone

Marseille, France

Location

Hôpital Villeneuce

Montpellier, France

Location

Hôpital Brabois

Nancy, France

Location

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

Paris, France

Location

Hôpital du Kremlin-Bicêtre

Paris, France

Location

Hôpital Necker - Enfants malades : service de dermatologie

Paris, France

Location

Hôpital Robert Debré

Paris, France

Location

Hôpital Trousseau

Paris, France

Location

Hôpital de Hautepierre

Strasbourg, France

Location

Hôpital Purpan

Toulouse, France

Location

Related Publications (6)

• 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.

  PMID: 18163404 BACKGROUND

• 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.

  PMID: 22736096 BACKGROUND

• 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.

  PMID: 12579588 BACKGROUND

• 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.

  PMID: 31118099 BACKGROUND

• 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.

  PMID: 31791867 BACKGROUND

• 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.

  PMID: 7986222 BACKGROUND

MeSH Terms

Conditions

Dermatomyositis

Interventions

baricitinib; Pregnancy Tests

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Clinical Laboratory Techniques; Diagnostic Techniques and Procedures; Diagnosis; Diagnostic Techniques, Obstetrical and Gynecological; Investigative Techniques

Study Officials

• Cyril GITIAUX, Doctor

  Assistance Publique - Hôpitaux de Paris

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 5, 2022
• First Posted: September 1, 2022
• Study Start: November 10, 2022
• Primary Completion: May 13, 2025
• Study Completion: January 2, 2026
• Last Updated: April 23, 2026

Record last verified: 2026-03

Locations

FR (13)