A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults with Dermatomyositis
- Registration Number
- NCT05437263
- Lead Sponsor
- Priovant Therapeutics, Inc.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 241
- 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.
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Dermatomyositis with end-stage organ involvement
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Dermatomyositis with irreversible muscle involvement
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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
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Overlap myositis/connective tissue disease (except for overlap with Sjögren's syndrome)
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Participants at a risk of thrombosis or cardiovascular disease
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Participants with a high risk for herpes zoster reactivation
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Participants with active or recent infections
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Brepocitinib Dose Level 2 PO QD Brepocitinib - Placebo PO QD Placebo - Brepocitinib Dose Level 1 PO QD Brepocitinib -
- Primary Outcome Measures
Name Time Method Total Improvement Score (TIS) at Week 52 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 Outcome Measures
Name Time Method TIS responder rate after 52 weeks of brepocitinib administration QD, in comparison to placebo 52 weeks Proportion of responders, defined as achieving TIS ≥ 40 points (moderate improvement) at Week 52
Change from baseline in HAQ Disability Index score at Week 52 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.
Cutaneous Dermatomyositis Area and Severity Index (CDASI) Activity Score after 52 weeks of brepocitinib administration QD, in comparison to placebo 52 weeks Change from baseline in Cutaneous Dermatomyositis Area and Severity Index (CDASI) Activity score at Week 52. Cutaneous Dermatomyositis Disease Area and Severity Index Activity Score 0 to 100 with higher scores indicating a worse outcome.
Average daily prednisone-equivalent dose from Week 36 to Week 52 minus baseline 52 weeks
Trial Locations
- Locations (1)
Clinical Trial Site
🇬🇧Wolverhampton, United Kingdom