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DermatomyositisPhase 3VALORNCT05437263

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

A Phase 3 study testing Brepocitinib in people with Dermatomyositis, run by Priovant Therapeutics, Inc. at 109 sites in 22 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
Search all trials

Trial 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… Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Dermatomyositis
What is being tested?
Brepocitinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
241 participants
Where
109 sitesin 22 countries
Study dates
31 Oct 2022to 9 Sep 2026
Phase
Phase 3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.)
Show all 11Show fewer
  • 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

Trial locations and contacts

109 sites in 22 countries.

109 sites in 22 countries

United States39 sites
  • Clinical Trial Site

    Ann Arbor, Michigan, 48103

  • Clinical Trial Site

    Atlanta, Georgia, 30322

  • Clinical Trial Site

    Augusta, Georgia, 30912

  • Clinical Trial Site

    Aurora, Colorado, 80045

  • Clinical Trial Site

    Austin, Texas, 78756

  • Clinical Trial Site

    Baltimore, Maryland, 21224

  • Clinical Trial Site

    Boston, Massachusetts, 02115

  • Clinical Trial Site

    Boynton Beach, Florida, 33472

  • Clinical Trial Site

    Chicago, Illinois, 60637

  • Clinical Trial Site

    Cincinnati, Ohio, 45267

  • Clinical Trial Site

    Cleveland, Ohio, 44195

  • Clinical Trial Site

    Denver, Colorado, 80230

  • Clinical Trial Site

    Gainesville, Florida, 32606

  • Clinical Trial Site

    Houston, Texas, 77030

  • Clinical Trial Site

    Iowa City, Iowa, 52242

  • Clinical Trial Site

    Irvine, California, 92617

  • Clinical Trial Site

    Irving, Texas, 75039

  • Clinical Trial Site

    Jackson, Tennessee, 38305

  • Clinical Trial Site

    Jacksonville, Florida, 32224

  • Clinical Trial Site

    Kansas City, Kansas, 66160

  • Clinical Trial Site

    Los Angeles, California, 90095

  • Clinical Trial Site

    Manhasset, New York, 11030

  • Clinical Trial Site

    Marietta, Georgia, 30060

  • Clinical Trial Site

    Minneapolis, Minnesota, 55455

  • Clinical Trial Site

    New Orleans, Louisiana, 70112

  • Clinical Trial Site

    New Orleans, Louisiana, 70433

  • Clinical Trial Site

    New York, New York, 10017

  • Clinical Trial Site

    New York, New York, 10021

  • Clinical Trial Site

    Oklahoma City, Oklahoma, 73116

  • Clinical Trial Site

    Philadelphia, Pennsylvania, 19104

  • Clinical Trial Site

    Phoenix, Arizona, 85028

  • Clinical Trial Site

    Pittsburgh, Pennsylvania, 15213

  • Clinical Trial Site

    Plantation, Florida, 33324

  • Clinical Trial Site

    Portland, Oregon, 97239

  • Clinical Trial Site

    Rochester, Minnesota, 55905

  • Clinical Trial Site

    San Francisco, California, 94115

  • Clinical Trial Site

    Scottsdale, Arizona, 85258

  • Clinical Trial Site

    Scottsdale, Arizona, 85259

  • Clinical Trial Site

    Tampa, Florida, 33613

Other trials in United States

Poland7 sites
  • Clinical Trial Site

    Bialystok, Podlaskie Voivodeship, 15-704

  • Clinical Trial Site

    Krakow, 30-363

  • Clinical Trial Site

    Lublin, 20-400

  • Clinical Trial Site

    Lublin, 20-607

  • Clinical Trial Site

    Nowa Sól, 67-100

  • Clinical Trial Site

    Poznan, 61-293

  • Clinical Trial Site

    Warsaw, 02-637

Other trials in Poland

Germany6 sites
  • Clinical Trial Site

    Berlin, 10117

  • Clinical Trial Site

    Berlin, 15562

  • Clinical Trial Site

    Dresden, 01307

  • Clinical Trial Site

    Essen, 45147

  • Clinical Trial Site

    Freiburg im Breisgau, 79106

  • Clinical Trial Site

    Mainz, Rhineland-Palatinate, 55131

Other trials in Germany

Chile5 sites
  • Clinical Trial Site

    Concepción, Biobio, 4070280

  • Clinical Trial Site

    Recoleta, 8420383

  • Clinical Trial Site

    Santiago, 7640881

  • Clinical Trial Site

    Santiago, 8331150

  • Clinical Trial Site

    Temuco, 4800827

Other trials in Chile

Israel5 sites
  • Clinical Trial Site

    Ashkelon, 7830604

  • Clinical Trial Site

    Haifa, 3109601

  • Clinical Trial Site

    Poria – Neve Oved, 1528001

  • Clinical Trial Site

    Tel Aviv, 6423906

  • Clinical Trial Site

    Tel HaShomer, 52621

Other trials in Israel

Mexico5 sites
  • Clinical Trial Site

    Guadalajara, 44690

  • Clinical Trial Site

    Mérida, Yucatán, 97070

  • Clinical Trial Site

    Mexico City, 06700

  • Clinical Trial Site

    Monterrey, Nuevo León, 64718

  • Clinical Trial Site

    San Luis Potosí City, 78290

Other trials in Mexico

Turkey (Türkiye)5 sites
  • Clinical Trial Site

    Ankara, 06560

  • Clinical Trial Site

    Antalya, 07070

  • Clinical Trial Site

    Istanbul, 34096

  • Clinical Trial Site

    Izmir, 35210

  • Clinical Trial Site

    İzmit, 41380

Other trials in Turkey (Türkiye)

Bulgaria4 sites
  • Clinical Trial Site

    Plovdiv, 4000

  • Clinical Trial Site

    Plovdiv, 4001

  • Clinical Trial Site

    Plovdiv, 4004

  • Clinical Trial Site

    Sofia, 1407

Other trials in Bulgaria

Italy4 sites
  • Clinical Trial Site

    Bari, 70126

  • Clinical Trial Site

    Pavia, 27100

  • Clinical Trial Site

    Roma, 00168

  • Clinical Trial Site

    Torino, 10126

Other trials in Italy

Portugal4 sites
  • Clinical Trial Site

    Guimarães, 4835-044

  • Clinical Trial Site

    Lisbon, 1649-028

  • Clinical Trial Site

    Porto, 4099-001

  • Clinical Trial Site

    Vila Nova de Gaia, Porto District, 4434-502

Other trials in Portugal

Taiwan4 sites
  • Clinical Trial Site

    Kaohsiung City, 833401

  • Clinical Trial Site

    Taichung, 40447

  • Clinical Trial Site

    Tainan, 710

  • Clinical Trial Site

    Taipei, 10002

Other trials in Taiwan

Argentina3 sites
  • Clinical Trial Site

    Caba, Buenos Aires F.D., 1425

  • Clinical Trial Site

    Mendoza, 5519

  • Clinical Trial Site

    Quilmes, Buenos Aires, B1878DVB

Other trials in Argentina

Hungary3 sites
  • Clinical Trial Site

    Debrecen, 4032

  • Clinical Trial Site

    Pécs, 7632

  • Clinical Trial Site

    Szeged, 6720

Other trials in Hungary

United Kingdom3 sites
  • Clinical Trial Site

    Bath, BA1 3NG

  • Clinical Trial Site

    Manchester, M13 9PT

  • Clinical Trial Site

    Wolverhampton, WV10 0QP

Other trials in United Kingdom

Canada2 sites
  • Clinical Trial Site

    Newmarket, Ontario, L3Y 5G8

  • Clinical Trial Site

    Vancouver, British Colombia, V5Y1K2

Other trials in Canada

Netherlands2 sites
  • Clinical Trial Site

    Amsterdam, 1105 AZ

  • Clinical Trial Site

    Nijmegen, Gelderland, 6500 HB

Other trials in Netherlands

Romania2 sites
  • Clinical Trial Site

    Bucharest, 011172

  • Clinical Trial Site

    Cluj-Napoca, 400000

Other trials in Romania

South Korea2 sites
  • Clinical Trial Site

    Seoul, 03080

  • Clinical Trial Site

    Suwon, 16499

Other trials in South Korea

Belgium1 site
  • Clinical Trial Site

    Leuven, 3000

Other trials in Belgium

Czechia1 site
  • Clinical Trial Site

    Prague, 128 00

Other trials in Czechia

Serbia1 site
  • Clinical Trial Site

    Belgrade, 11000

Other trials in Serbia

Spain1 site
  • Clinical Trial Site

    Madrid, 28007

Other trials in Spain

About this study

Official title: 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.

Conditions
Dermatomyositis
Being tested
Brepocitinib, Placebo
Keywords
brepocitinib, dermatomyositis, TYK2/JAK1 inhibitor, PF-06700841, PVT-2201
Medical subject headings
Dermatomyositis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • BrepocitinibDrug

    Oral Brepocitinib

  • PlaceboDrug

    Oral Placebo

Study groups in NCT05437263
Study groupRoleWhat they receive
Brepocitinib Dose Level 1 PO QDExperimentalNot described
Receives: Drug: Brepocitinib
Brepocitinib Dose Level 2 PO QDExperimentalNot described
Receives: Drug: Brepocitinib
Placebo PO QDPlacebo comparatorNot described
Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

  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

    Measured: 52 weeks

Secondary outcomes (9)
  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.

    Measured: 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.

    Measured: 52 weeks

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

    Measured: 52 weeks

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

    Measured: 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

    Measured: 52 weeks

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

    Measured: 52 weeks

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

    Measured: 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.

    Measured: 52 weeks

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

    Measured: 4 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted29 Jun 2022
  2. Study start31 Oct 2022
  3. Primary completion17 Jul 2025
  4. Study completion9 Sep 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Triple blind
Who is masked
Participant, Investigator, Outcomes assessor
Enrolment
241 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05437263
Sponsor's protocol ID
PVT-2201-301
Other IDs
2022-500367-12-00
Acronym
VALOR
Status last verified
Sep 2026
First submitted
24 Jun 2022
Last updated
15 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
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.

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

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Brepocitinib in people with Dermatomyositis, run by Priovant Therapeutics, Inc. at 109 sites in 22 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 15 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 109 sites in United States, Poland, Germany, Chile, Israel and 17 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Total Improvement Score (TIS) at Week 52 (time frame: 52 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05437263, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Sep 2026.

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