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

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Oral 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.

Study Overview

• Status: Active, not recruiting
• Conditions: Dermatomyositis
• Intervention / Treatment: Drug: Brepocitinib; Drug: Placebo

• Study Type: Interventional
• Enrollment (Actual): 241
• Phase: Phase 3

Contacts and Locations

Study Locations

• Argentina
  • Mendoza, Argentina, 5519
    • Clinical Trial Site
  • Buenos Aires
    • Quilmes, Buenos Aires, Argentina, B1878DVB
      • Clinical Trial Site
  • Buenos Aires F.D.
    • Caba, Buenos Aires F.D., Argentina, 1425
      • Clinical Trial Site
• Belgium
  • Leuven, Belgium, 3000
    • Clinical Trial Site
• Bulgaria
  • Plovdiv, Bulgaria, 4000
    • Clinical Trial Site
  • Plovdiv, Bulgaria, 4001
    • Clinical Trial Site
  • Plovdiv, Bulgaria, 4004
    • Clinical Trial Site
  • Sofia, Bulgaria, 1407
    • Clinical Trial Site
• Canada
  • British Colombia
    • Vancouver, British Colombia, Canada, V5Y1K2
      • Clinical Trial Site
  • Ontario
    • Newmarket, Ontario, Canada, L3Y 5G8
      • Clinical Trial Site
• Chile
  • Recoleta, Chile, 8420383
    • Clinical Trial Site
  • Santiago, Chile, 7640881
    • Clinical Trial Site
  • Santiago, Chile, 8331150
    • Clinical Trial Site
  • Temuco, Chile, 4800827
    • Clinical Trial Site
  • Región del Biobío
    • Concepción, Región del Biobío, Chile, 4070280
      • Clinical Trial Site
• Czechia
  • Prague, Czechia, 128 00
    • Clinical Trial Site
• Germany
  • Berlin, Germany, 10117
    • Clinical Trial Site
  • Berlin, Germany, 15562
    • Clinical Trial Site
  • Dresden, Germany, 01307
    • Clinical Trial Site
  • Essen, Germany, 45147
    • Clinical Trial Site
  • Freiburg im Breisgau, Germany, 79106
    • Clinical Trial Site
  • Rhineland-Palatinate
    • Mainz, Rhineland-Palatinate, Germany, 55131
      • Clinical Trial Site
• Hungary
  • Debrecen, Hungary, 4032
    • Clinical Trial Site
  • Pécs, Hungary, 7632
    • Clinical Trial Site
  • Szeged, Hungary, 6720
    • Clinical Trial Site
• Israel
  • Ashkelon, Israel, 7830604
    • Clinical Trial Site
  • Haifa, Israel, 3109601
    • Clinical Trial Site
  • Poria – Neve Oved, Israel, 1528001
    • Clinical Trial Site
  • Tel Aviv, Israel, 6423906
    • Clinical Trial Site
  • Tel Litwinsky, Israel, 52621
    • Clinical Trial Site
• Italy
  • Bari, Italy, 70126
    • Clinical Trial Site
  • Pavia, Italy, 27100
    • Clinical Trial Site
  • Roma, Italy, 00168
    • Clinical Trial Site
  • Torino, Italy, 10126
    • Clinical Trial Site
• Mexico
  • Guadalajara, Mexico, 44690
    • Clinical Trial Site
  • Mexico City, Mexico, 06700
    • Clinical Trial Site
  • San Luis Potosí City, Mexico, 78290
    • Clinical Trial Site
  • Nuevo León
    • Monterrey, Nuevo León, Mexico, 64718
      • Clinical Trial Site
  • Yucatán
    • Mérida, Yucatán, Mexico, 97070
      • Clinical Trial Site
• Netherlands
  • Amsterdam, Netherlands, 1105 AZ
    • Clinical Trial Site
  • Gelderland
    • Nijmegen, Gelderland, Netherlands, 6500 HB
      • Clinical Trial Site
• Poland
  • Krakow, Poland, 30-363
    • Clinical Trial Site
  • Lublin, Poland, 20-400
    • Clinical Trial Site
  • Lublin, Poland, 20-607
    • Clinical Trial Site
  • Nowa Sól, Poland, 67-100
    • Clinical Trial Site
  • Poznan, Poland, 61-293
    • Clinical Trial Site
  • Warsaw, Poland, 02-637
    • Clinical Trial Site
  • Podlaskie Voivodeship
    • Bialystok, Podlaskie Voivodeship, Poland, 15-704
      • Clinical Trial Site
• Portugal
  • Guimarães, Portugal, 4835-044
    • Clinical Trial Site
  • Lisbon, Portugal, 1649-028
    • Clinical Trial Site
  • Porto, Portugal, 4099-001
    • Clinical Trial Site
  • Porto District
    • Vila Nova de Gaia, Porto District, Portugal, 4434-502
      • Clinical Trial Site
• Romania
  • Bucharest, Romania, 011172
    • Clinical Trial Site
  • Cluj-Napoca, Romania, 400000
    • Clinical Trial Site
• Serbia
  • Belgrade, Serbia, 11000
    • Clinical Trial Site
• South Korea
  • Seoul, South Korea, 03080
    • Clinical Trial Site
  • Suwon, South Korea, 16499
    • Clinical Trial Site
• Spain
  • Madrid, Spain, 28007
    • Clinical Trial Site
• Taiwan
  • Kaohsiung City, Taiwan, 833401
    • Clinical Trial Site
  • Taichung, Taiwan, 40447
    • Clinical Trial Site
  • Tainan City, Taiwan, 710
    • Clinical Trial Site
  • Taipei, Taiwan, 10002
    • Clinical Trial Site
• Turkey (Türkiye)
  • Ankara, Turkey (Türkiye), 06560
    • Clinical Trial Site
  • Antalya, Turkey (Türkiye), 07070
    • Clinical Trial Site
  • Istanbul, Turkey (Türkiye), 34096
    • Clinical Trial Site
  • Izmir, Turkey (Türkiye), 35210
    • Clinical Trial Site
  • İzmit, Turkey (Türkiye), 41380
    • Clinical Trial Site
• United Kingdom
  • Bath, United Kingdom, BA1 3NG
    • Clinical Trial Site
  • Manchester, United Kingdom, M13 9PT
    • Clinical Trial Site
  • Wolverhampton, United Kingdom, WV10 0QP
    • Clinical Trial Site
• United States
  • Arizona
    • Phoenix, Arizona, United States, 85028
      • Clinical Trial Site
    • Scottsdale, Arizona, United States, 85259
      • Clinical Trial Site
    • Scottsdale, Arizona, United States, 85258
      • Clinical Trial Site
  • California
    • Irvine, California, United States, 92617
      • Clinical Trial Site
    • Los Angeles, California, United States, 90095
      • Clinical Trial Site
    • San Francisco, California, United States, 94115
      • Clinical Trial Site
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Clinical Trial Site
    • Denver, Colorado, United States, 80230
      • Clinical Trial Site
  • Florida
    • Boynton Beach, Florida, United States, 33472
      • Clinical Trial Site
    • Gainesville, Florida, United States, 32606
      • Clinical Trial Site
    • Jacksonville, Florida, United States, 32224
      • Clinical Trial Site
    • Plantation, Florida, United States, 33324
      • Clinical Trial Site
    • Tampa, Florida, United States, 33613
      • Clinical Trial Site
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Clinical Trial Site
    • Augusta, Georgia, United States, 30912
      • Clinical Trial Site
    • Marietta, Georgia, United States, 30060
      • Clinical Trial Site
  • Illinois
    • Chicago, Illinois, United States, 60637
      • Clinical Trial Site
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • Clinical Trial Site
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • Clinical Trial Site
  • Louisiana
    • New Orleans, Louisiana, United States, 70112
      • Clinical Trial Site
    • New Orleans, Louisiana, United States, 70433
      • Clinical Trial Site
  • Maryland
    • Baltimore, Maryland, United States, 21224
      • Clinical Trial Site
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Clinical Trial Site
  • Michigan
    • Ann Arbor, Michigan, United States, 48103
      • Clinical Trial Site
  • Minnesota
    • Minneapolis, Minnesota, United States, 55455
      • Clinical Trial Site
    • Rochester, Minnesota, United States, 55905
      • Clinical Trial Site
  • New York
    • Manhasset, New York, United States, 11030
      • Clinical Trial Site
    • New York, New York, United States, 10021
      • Clinical Trial Site
    • New York, New York, United States, 10017
      • Clinical Trial Site
  • Ohio
    • Cincinnati, Ohio, United States, 45267
      • Clinical Trial Site
    • Cleveland, Ohio, United States, 44195
      • Clinical Trial Site
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73116
      • Clinical Trial Site
  • Oregon
    • Portland, Oregon, United States, 97239
      • Clinical Trial Site
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • Clinical Trial Site
    • Pittsburgh, Pennsylvania, United States, 15213
      • Clinical Trial Site
  • Tennessee
    • Jackson, Tennessee, United States, 38305
      • Clinical Trial Site
  • Texas
    • Austin, Texas, United States, 78756
      • Clinical Trial Site
    • Houston, Texas, United States, 77030
      • Clinical Trial Site
    • Irving, Texas, United States, 75039
      • Clinical Trial Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 75 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

3

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Total Improvement Score (TIS) at Week 52	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	52 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in HAQ Disability Index score at Week 52	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.	52 weeks
Change from baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 52	CDASI Activity Score 0 to 100 with higher scores indicating a worse outcome.	52 weeks
Dermatomyositis Outcomes for Muscle and Skin (DMOMS) at Week 52	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.	52 weeks
The proportion of participants achieving TIS ≥ 40 points (moderate improvement) at Week 52		52 weeks
Time to achievement of consecutive (≥ 2 visits) TIS ≥ 40 points (moderate improvement) by Week 52		52 weeks
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		52 weeks
The proportion of participants achieving ≥ 40% improvement with a ≥ 4-point improvement from baseline in CDASI Activity Score at Week 52		52 weeks
The proportion of participants achieving TIS ≥ 60 points (major improvement) at Week 52		52 weeks
Change from baseline in CDASI Activity Score at Week 4		4 weeks

Collaborators and Investigators

• Sponsor: Priovant Therapeutics, Inc.
• Investigators: Study Director: Noriko Iikuni, MD, VP, Clinical Development

Publications and helpful links

Helpful Links

• Study Website

Study record dates

Study Major Dates

• Study Start (Actual): October 31, 2022
• Primary Completion (Actual): July 17, 2025
• Study Completion (Estimated): July 1, 2026

Study Registration Dates

• First Submitted: June 24, 2022
• First Submitted That Met QC Criteria: June 24, 2022
• First Posted (Actual): June 29, 2022

Study Record Updates

• Last Update Posted (Estimated): September 29, 2025
• Last Update Submitted That Met QC Criteria: September 23, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Keywords: PF-06700841; dermatomyositis; brepocitinib; TYK2/JAK1 inhibitor; PVT-2201
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Diseases; Connective Tissue Diseases; Skin Diseases; Myositis; Polymyositis; Skin and Connective Tissue Diseases; Dermatomyositis
• Other Study ID Numbers: PVT-2201-301; 2022-500367-12-00 (Registry Identifier: EU CT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD 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.
• IPD Sharing 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.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No