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NCT Number: NCT05437263

A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis

This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of treatment with brepocitinib (TYK2/JAK1 inhibitor) in adults with dermatomyositis (DM). The primary objective of this study is to assess the efficacy of two dose levels of brepocitinib in comparison to placebo, as measured by differences in the Total Improvement Score (TIS). After 52 weeks of double-blind treatment, participants have the option to continue therapy in a 52 week open-label extension phase where all participants will receive brepocitinib.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

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

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Clinical Trial Site, Quilmes, Buenos Aires, Argentina

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • A diagnosis of dermatomyositis according to 2017 EULAR/ACR Classification Criteria for Idiopathic Inflammatory Myopathies
  • Adult subjects (18-75 years old)
  • Active muscle and skin disease at screening and baseline
  • Prior therapy OR current therapy with corticosteroids, hydroxychloroquine, and/or one non-steroid immunosuppressant
  • Weight > 40 kg to < 130 kg, and with a body mass index (BMI) < 40 kg/m2.

Exclusion criteria

  • Dermatomyositis with end-stage organ involvement
  • Dermatomyositis with irreversible muscle involvement
  • History of:
  • Any lymphoproliferative disorder
  • Active malignancy;
  • History of cancer within 5 years prior to randomization (exceptions for basal cell carcinoma, squamous cell carcinoma, ductal carcinoma in situ of the breast, carcinoma in situ of the uterine cervix, or thyroid carcinoma.)
  • Cancer-associated dermatomyositis
  • Overlap myositis/connective tissue disease (except for overlap with Sjögren's syndrome)
  • Participants at a risk of thrombosis or cardiovascular disease
  • Participants with a high risk for herpes zoster reactivation
  • Participants with active or recent infections

Treatment and study plan

Brepocitinib

Drug

Oral Brepocitinib

Placebo

Drug

Oral Placebo

Primary outcomes

  1. Total Improvement Score (TIS) at Week 52

    Time frame: 52 weeks

    TIS is a composite endpoint based on improvement in the 6 Disease Activity Core Set Measure (CSM) scores and ranges from 0 to 100 (2016 American College of Rheumatology [ACR] Myositis Response Criteria/European League Against Rheumatism [EULAR]) where a higher score indicates more improvement

Secondary outcomes

  1. Change from baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 52

    Time frame: 52 weeks

    CDASI Activity Score 0 to 100 with higher scores indicating a worse outcome.

  2. Dermatomyositis Outcomes for Muscle and Skin (DMOMS) at Week 52

    Time frame: 52 weeks

    DMOMS is a composite endpoint based on 4 component measures and ranges from 0 to 100 (Pandya, 2024) where a higher score indicates more improvement.

  3. The proportion of participants achieving TIS ≥ 40 points (moderate improvement) at Week 52

    Time frame: 52 weeks

  4. Time to achievement of consecutive (≥ 2 visits) TIS ≥ 40 points (moderate improvement) by Week 52

    Time frame: 52 weeks

  5. The proportion of participants, regardless of baseline corticosteroid use, achieving TIS ≥ 40 points (moderate improvement) at Week 52 with 0 to ≤ 2.5 mg/day of oral prednisone (or equivalent) at both Week 48 and Week 52

    Time frame: 52 weeks

  6. The proportion of participants achieving ≥ 40% improvement with a ≥ 4-point improvement from baseline in CDASI Activity Score at Week 52

    Time frame: 52 weeks

  7. The proportion of participants achieving TIS ≥ 60 points (major improvement) at Week 52

    Time frame: 52 weeks

  8. Change from baseline in HAQ Disability Index score at Week 52

    Time frame: 52 weeks

    Change from baseline in HAQ Disability Index score at Week 52. Health Assessment Questionnaire (HAQ) Disability Index: Score for function and disability from 0 [without any difficulty] to 3 [unable to do]. Higher score associated with worse outcome.

  9. Change from baseline in CDASI Activity Score at Week 4

    Time frame: 4 weeks

Sponsors and collaborators

Lead sponsor

Priovant Therapeutics, Inc.

Industry

Registry information

Official study title

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Oral Brepocitinib in Adults With Dermatomyositis

Acronym: VALOR

Important dates

Study start
2022
Primary completion
2025
Study completion
2026
First posted
Jun 29, 2022
Registry last updated
Jul 17, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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