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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 : March 1, 2023
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. 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

  3. 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.


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 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
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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United States, Arizona
Clinical Trial Site Recruiting
Phoenix, Arizona, United States, 85028
Clinical Trial Site Recruiting
Scottsdale, Arizona, United States, 85258
United States, California
Clinical Trial Site Recruiting
Irvine, California, United States, 92617
Clinical Trial Site Recruiting
Los Angeles, California, United States, 90095
United States, Colorado
Clinical Trial Site Recruiting
Denver, Colorado, United States, 80230
United States, Florida
Clinical Trial Site Recruiting
Gainesville, Florida, United States, 32606
Clinical Trial Site Recruiting
Jacksonville, Florida, United States, 32224
Clinical Trial Site Recruiting
Orlando, Florida, United States, 32819
Clinical Trial Site Recruiting
Plantation, Florida, United States, 33324
United States, Kansas
Clinical Trial Site Recruiting
Kansas City, Kansas, United States, 66160
United States, Louisiana
Clinical Trial Site Recruiting
New Orleans, Louisiana, United States, 70433
United States, Maryland
Clinical Trial Site Recruiting
Baltimore, Maryland, United States, 21224
United States, Massachusetts
Clinical Trial Site Recruiting
Boston, Massachusetts, United States, 02115
United States, Minnesota
Clinical Trial Site Recruiting
Minneapolis, Minnesota, United States, 55455
Clinical Trial Site Recruiting
Rochester, Minnesota, United States, 55905
United States, New York
Clinical Trial Site Recruiting
New York, New York, United States, 10017
Clinical Trial Site Recruiting
New York, New York, United States, 10021
Clinical Trial Site Recruiting
New York, New York, United States, 10029
United States, Ohio
Clinical Trial Site Recruiting
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
Clinical Trial Site Recruiting
Pittsburgh, Pennsylvania, United States, 15213
United States, Texas
Clinical Trial Site Recruiting
Austin, Texas, United States, 78756
Clinical Trial Site Recruiting
Dallas, Texas, United States, 75231
Bulgaria
Clinical Trial Site Recruiting
Plovdiv, Bulgaria, 4001
Clinical Trial Site Recruiting
Plovdiv, Bulgaria, 4004
Clinical Trial Site Recruiting
Sofia, Bulgaria, 1407
Czechia
Clinical Trial Site Recruiting
Prague, Czechia, 128 00
Germany
Clinical Trial Site Recruiting
Mainz, Rheinland-Pfalz, Germany, 55131
Hungary
Clinical Trial Site Recruiting
Szeged, Hungary, 6720
Israel
Clinical Trial Site Recruiting
Haifa, Israel, 3109601
Clinical Trial Site Recruiting
Tel HaShomer, Israel, 52621
Italy
Clinical Trial Site Recruiting
Pavia, Italy, 27100
Clinical Trial Site Recruiting
Torino, Italy, 10126
Poland
Clinical Trial Site Recruiting
Kraków, Poland, 30-363
Clinical Trial Site Recruiting
Lublin, Poland, 20-607
Clinical Trial Site Recruiting
Nowa Sól, Poland, 67-100
Clinical Trial Site Recruiting
Poznań, Poland, 61-293
Clinical Trial Site Recruiting
Warsaw, Poland, 02-637
Romania
Clinical Trial Site Recruiting
Bucharest, Romania, 011172
Clinical Trial Site Recruiting
Cluj-Napoca, Romania, 400000
Clinical Trial Site Recruiting
Timişoara, Romania, 300373
Slovakia
Clinical Trial Site Recruiting
Nové Mesto Nad Váhom, Slovakia, 91501
Spain
Clinical Trial Site Recruiting
Mataró, Spain, 8302
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: March 1, 2023
Last Verified: February 2023
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