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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05437263
Recruitment Status : Recruiting
First Posted : June 29, 2022
Last Update Posted : May 3, 2023
Sponsor:
Information provided by (Responsible Party):
Priovant Therapeutics, Inc.

Tracking Information
First Submitted Date  ICMJE June 24, 2022
First Posted Date  ICMJE June 29, 2022
Last Update Posted Date May 3, 2023
Actual Study Start Date  ICMJE October 31, 2022
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: December 30, 2022)		 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
Original Primary Outcome Measures  ICMJE
 (submitted: June 24, 2022)		 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 European League Against Rheumatism [EULAR]/American College of Rheumatology [ACR] Myositis Response Criteria)
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 30, 2022)
  • 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
  • Time to initiation of rescue medication through 52 weeks of brepocitinib administration QD, in comparison to placebo [ Time Frame: 52 weeks ]
    Time to initiation of rescue medication through Week 52
  • Manual Muscle Testing (MMT-8) after 52 weeks of brepocitinib administration QD, in comparison to placebo [ Time Frame: 52 weeks ]
    Change from baseline in MMT-8 score at Week 52. The MMT-8 evaluation includes 7 muscles tested bilaterally plus neck flexors, each scored from 0 to 10 for a potential score ranging from 0 to 150 where a higher score indicates more improvement.
Original Secondary Outcome Measures  ICMJE
 (submitted: June 24, 2022)
  • 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
  • Time to use of rescue medication through 52 weeks of brepocitinib administration QD, in comparison to placebo [ Time Frame: 52 weeks ]
    Time to use of rescue medication through Week 52
  • MMT-8 after 52 weeks of brepocitinib administration QD, in comparison to placebo [ Time Frame: 52 weeks ]
    Change from baseline in MMT-8 score at Week 52
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis
Official Title  ICMJE A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Oral Brepocitinib in Adults With Dermatomyositis
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 Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Dermatomyositis
Intervention  ICMJE
  • Drug: Brepocitinib
    Oral Brepocitinib
  • Drug: Placebo
    Oral Placebo
Study Arms  ICMJE
  • Experimental: Brepocitinib Dose Level 1 PO QD
    Intervention: Drug: Brepocitinib
  • Experimental: Brepocitinib Dose Level 2 PO QD
    Intervention: Drug: Brepocitinib
  • Placebo Comparator: Placebo PO QD
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: June 24, 2022)		 225
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2024
Estimated Primary Completion Date December 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 screening (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 and cardiovascular disease
  • Participants with a high risk for herpes zoster reactivation
  • Participants with active or recent infections
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Clinical Trial Administrator (212) 634-9743 ValorStudyManager@PriovantTx.com
Listed Location Countries  ICMJE Belgium,   Bulgaria,   Chile,   Czechia,   Germany,   Hungary,   Israel,   Italy,   Korea, Republic of,   Mexico,   Poland,   Romania,   Slovakia,   Spain,   Turkey,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05437263
Other Study ID Numbers  ICMJE PVT-2201-301
2022-500367-12-00 ( Registry Identifier: EU CT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
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
Supporting Materials: Statistical Analysis Plan (SAP)
Supporting Materials: 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.
Current Responsible Party Priovant Therapeutics, Inc.
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Priovant Therapeutics, Inc.
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Noriko Iikuni, MD VP, Clinical Development
PRS Account Priovant Therapeutics, Inc.
Verification Date April 2023

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP