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

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ClinicalTrials.gov Identifier: NCT05437263
Recruitment Status : Recruiting
First Posted : June 29, 2022
Last Update Posted : April 8, 2024
Sponsor:
Information provided by (Responsible Party):
Priovant Therapeutics, Inc.

Study Description
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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.

Condition or disease Intervention/treatment Phase
Dermatomyositis Drug: Brepocitinib Drug: Placebo Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 225 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Oral Brepocitinib in Adults With Dermatomyositis
Actual Study Start Date : October 31, 2022
Estimated Primary Completion Date : December 2024
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Brepocitinib Dose Level 1 PO QD Drug: Brepocitinib
Oral Brepocitinib
Experimental: Brepocitinib Dose Level 2 PO QD Drug: Brepocitinib
Oral Brepocitinib
Placebo Comparator: Placebo PO QD Drug: Placebo
Oral Placebo


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  1. TIS responder rate after 52 weeks of brepocitinib administration QD, in comparison to placebo [ Time Frame: 52 weeks ]
    Proportion of responders, defined as achieving TIS ≥ 40 points (moderate improvement) at Week 52

  2. 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.

  3. Cutaneous Dermatomyositis Area and Severity Index (CDASI) Activity Score after 52 weeks of brepocitinib administration QD, in comparison to placebo [ Time Frame: 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.

  4. Average daily prednisone-equivalent dose from Week 36 to Week 52 minus baseline [ Time Frame: 52 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05437263


Contacts
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Contact: Clinical Trial Administrator (212) 634-9743 ValorStudyManager@PriovantTx.com

Locations
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Sponsors and Collaborators
Priovant Therapeutics, Inc.
Investigators
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Study Director: Noriko Iikuni, MD VP, Clinical Development
More Information
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Additional Information:

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Responsible Party: Priovant Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05437263    
Other Study ID Numbers: PVT-2201-301
2022-500367-12-00 ( Registry Identifier: EU CT Number )
First Posted: June 29, 2022    Key Record Dates
Last Update Posted: April 8, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Priovant Therapeutics will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) from eligible studies upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Data requests will be reviewed and approved on the basis of scientific merit.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Clinical Study Report (CSR)
Time Frame: The data will be made available within 24 months after study completion and will be accessible for a time frame appropriate for the approved proposal.
Access Criteria: Access will be provided following review and approval of a research proposal and execution of a Data Sharing Agreement (DSA). Further details on Priovant Therapeutics' data sharing criteria and process for requesting access can be obtained by emailing info@priovanttx.com.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Priovant Therapeutics, Inc.:
brepocitinib
dermatomyositis
TYK2/JAK1 inhibitor
PF-06700841
PVT-2201
Additional relevant MeSH terms:
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Dermatomyositis
Polymyositis
Myositis
Muscular Diseases
Musculoskeletal Diseases
 Neuromuscular Diseases
Nervous System Diseases
Connective Tissue Diseases
Skin Diseases