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CompletedNCT05437263VALORUpdated Sep 15, 2026

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

A Phase 3 interventional study of Brepocitinib and Placebo in Dermatomyositis, sponsored by Priovant Therapeutics, Inc.. Completed at 109 sites in 22 countries. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-09-15.

Sponsored by Priovant Therapeutics, Inc. · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
241
Allocation
Randomized
Ages
18 Years to 75 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Dermatomyositis

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Keywords

  • brepocitinib
  • dermatomyositis
  • TYK2/JAK1 inhibitor
  • PF-06700841
  • PVT-2201
03

Who can participate

Ages eligible
18 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

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

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
04

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Triple (Participant, Investigator, Outcomes assessor)
Enrollment
241 participants (actual)

Study arms

  • Experimental
    Brepocitinib Dose Level 1 PO QD

    Drug: Brepocitinib

  • Experimental
    Brepocitinib Dose Level 2 PO QD

    Drug: Brepocitinib

  • Placebo comparator
    Placebo PO QD

    Drug: Placebo

Interventions

  • DrugBrepocitinib

    Oral Brepocitinib

  • DrugPlacebo

    Oral Placebo

05

What researchers measure

Primary outcomes

  1. 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

    Time frame: 52 weeks

Secondary outcomes

  1. 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.

    Time frame: 52 weeks

  2. 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.

    Time frame: 52 weeks

  3. The proportion of participants achieving TIS ≥ 40 points (moderate improvement) at Week 52

    Time frame: 52 weeks

  4. Time to achievement of consecutive (≥ 2 visits) TIS ≥ 40 points (moderate improvement) by Week 52

    Time frame: 52 weeks

  5. 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

    Time frame: 52 weeks

  6. The proportion of participants achieving ≥ 40% improvement with a ≥ 4-point improvement from baseline in CDASI Activity Score at Week 52

    Time frame: 52 weeks

  7. The proportion of participants achieving TIS ≥ 60 points (major improvement) at Week 52

    Time frame: 52 weeks

  8. 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.

    Time frame: 52 weeks

  9. Change from baseline in CDASI Activity Score at Week 4

    Time frame: 4 weeks

06

Study locations

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

Showing the first 100 of 109 sites across 22 countries.

07

References and documents

Publications

  • Mangold AR, Haemel A, Shahriari N, Chong B, Fernandez AP, Fiorentino D, Pearson D, Sluzevich J, Onajin O, Rambhatla PV, Rashighi M, Hurabielle C, Fivenson D, Boh E, Paravar T, Crew A, Flowers RH, Taylor D, Subhardashani S, Culton D, Schadt C, Callen J, Lam C, Graham L, Hashemi K, Castillo R, Maderal A, Elman S, Shaw KS, Cascino MD, LaChance A, Min MS, Femia A, Vleugels RA, Werth VP. Skin-Specific Outcomes of Brepocitinib in Patients With Dermatomyositis: Secondary Analysis of a Phase 3 Randomized Clinical Trial. JAMA Dermatol. 2026 Aug 26. doi: 10.1001/jamadermatol.2026.3199. Online ahead of print. PubMed 42646746 ↗
  • Vleugels RA, Paik JJ, Bauer Ventura I, Mangold AR, Gandiga PC, Haemel A, Chinoy H, Hussain YM, Sivakumar K, Griger Z, Lee EB, Bozan F, Hsu CY, Femia A, Dimachkie MM, Min MS, Mozaffar T, Charles-Schoeman C, Fernandez DR, Onajin O, Campanilho-Marques R, Marder G, Ernste F, Schiopu E, Sluzevich J, Pearson D, Lindsey S, Luggen M, Bubb MR, Boh E, Maganti R, Heinlen L, Shaw KS, Cascino MD, Mudd PN Jr, Vencovsky J, Fernandez AP, Fiorentino D, Christopher-Stine L, Werth VP, Aggarwal R; VALOR Investigators. A Phase 3 Trial of Brepocitinib in Dermatomyositis. N Engl J Med. 2026 May 14;394(19):1883-1893. doi: 10.1056/NEJMoa2503531. Epub 2026 Mar 28. PubMed 41910335 ↗

Related links

Individual participant data

Plan to share: Yes — 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 information: Study protocol, Sap, Csr

08

Registry details

Key details

Study ID
NCT05437263
Lead sponsor
Priovant Therapeutics, Inc.
Responsible party
Sponsor
First posted
Jun 29, 2022
Start date
Oct 31, 2022
Primary completion
Jul 17, 2025
Completion
Sep 9, 2026
Last update
Sep 15, 2026

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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