NCT05437263

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Strong global presence with extensive site network
Enrollment
241

participants targeted

Target at P50-P75 for phase_3

Timeline
2mo left

Started Oct 2022

Typical duration for phase_3

Geographic Reach
22 countries

109 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress96%
Oct 2022Jul 2026

First Submitted

Initial submission to the registry

June 24, 2022

Completed
5 days until next milestone

First Posted

Study publicly available on registry

June 29, 2022

Completed
4 months until next milestone

Study Start

First participant enrolled

October 31, 2022

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 17, 2025

Completed
12 months until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2026

Expected
Last Updated

September 29, 2025

Status Verified

September 1, 2025

Enrollment Period

2.7 years

First QC Date

June 24, 2022

Last Update Submit

September 23, 2025

Conditions

Keywords

brepocitinibdermatomyositisTYK2/JAK1 inhibitorPF-06700841PVT-2201

Outcome Measures

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

    52 weeks

Secondary Outcomes (9)

  • Change from baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 52

    52 weeks

  • Dermatomyositis Outcomes for Muscle and Skin (DMOMS) at Week 52

    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

  • +4 more secondary outcomes

Study Arms (3)

Brepocitinib Dose Level 1 PO QD

EXPERIMENTAL
Drug: Brepocitinib

Brepocitinib Dose Level 2 PO QD

EXPERIMENTAL
Drug: Brepocitinib

Placebo PO QD

PLACEBO COMPARATOR
Drug: Placebo

Interventions

Oral Brepocitinib

Brepocitinib Dose Level 1 PO QDBrepocitinib Dose Level 2 PO QD

Oral Placebo

Placebo PO QD

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (109)

Clinical Trial Site

Phoenix, Arizona, 85028, United States

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Scottsdale, Arizona, 85258, United States

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Scottsdale, Arizona, 85259, United States

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Irvine, California, 92617, United States

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Los Angeles, California, 90095, United States

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San Francisco, California, 94115, United States

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Aurora, Colorado, 80045, United States

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Denver, Colorado, 80230, United States

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Boynton Beach, Florida, 33472, United States

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Gainesville, Florida, 32606, United States

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Jacksonville, Florida, 32224, United States

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Plantation, Florida, 33324, United States

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Tampa, Florida, 33613, United States

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Atlanta, Georgia, 30322, United States

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Augusta, Georgia, 30912, United States

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Marietta, Georgia, 30060, United States

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Chicago, Illinois, 60637, United States

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Iowa City, Iowa, 52242, United States

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Kansas City, Kansas, 66160, United States

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New Orleans, Louisiana, 70112, United States

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New Orleans, Louisiana, 70433, United States

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Baltimore, Maryland, 21224, United States

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Boston, Massachusetts, 02115, United States

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Ann Arbor, Michigan, 48103, United States

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Minneapolis, Minnesota, 55455, United States

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Rochester, Minnesota, 55905, United States

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Manhasset, New York, 11030, United States

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New York, New York, 10017, United States

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New York, New York, 10021, United States

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Cincinnati, Ohio, 45267, United States

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Cleveland, Ohio, 44195, United States

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Oklahoma City, Oklahoma, 73116, United States

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Portland, Oregon, 97239, United States

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Philadelphia, Pennsylvania, 19104, United States

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Pittsburgh, Pennsylvania, 15213, United States

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Jackson, Tennessee, 38305, United States

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Austin, Texas, 78756, United States

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Houston, Texas, 77030, United States

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Irving, Texas, 75039, United States

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Quilmes, Buenos Aires, B1878DVB, Argentina

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Caba, Buenos Aires F.D., 1425, Argentina

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Mendoza, 5519, Argentina

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Leuven, 3000, Belgium

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Plovdiv, 4000, Bulgaria

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Plovdiv, 4001, Bulgaria

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Plovdiv, 4004, Bulgaria

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Sofia, 1407, Bulgaria

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Vancouver, British Colombia, V5Y1K2, Canada

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Newmarket, Ontario, L3Y 5G8, Canada

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Concepción, Región del Biobío, 4070280, Chile

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Recoleta, 8420383, Chile

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Santiago, 7640881, Chile

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Santiago, 8331150, Chile

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Temuco, 4800827, Chile

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Prague, 128 00, Czechia

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Mainz, Rhineland-Palatinate, 55131, Germany

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Berlin, 10117, Germany

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Berlin, 15562, Germany

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Dresden, 01307, Germany

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Essen, 45147, Germany

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Freiburg im Breisgau, 79106, Germany

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Debrecen, 4032, Hungary

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Pécs, 7632, Hungary

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Szeged, 6720, Hungary

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Ashkelon, 7830604, Israel

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Haifa, 3109601, Israel

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Poria – Neve Oved, 1528001, Israel

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Tel Aviv, 6423906, Israel

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Tel Litwinsky, 52621, Israel

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Bari, 70126, Italy

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Pavia, 27100, Italy

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Roma, 00168, Italy

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Torino, 10126, Italy

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Monterrey, Nuevo León, 64718, Mexico

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Mérida, Yucatán, 97070, Mexico

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Guadalajara, 44690, Mexico

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Mexico City, 06700, Mexico

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San Luis Potosí City, 78290, Mexico

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Nijmegen, Gelderland, 6500 HB, Netherlands

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Amsterdam, 1105 AZ, Netherlands

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Bialystok, Podlaskie Voivodeship, 15-704, Poland

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Krakow, 30-363, Poland

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Lublin, 20-400, Poland

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Lublin, 20-607, Poland

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Nowa Sól, 67-100, Poland

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Poznan, 61-293, Poland

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Warsaw, 02-637, Poland

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Vila Nova de Gaia, Porto District, 4434-502, Portugal

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Guimarães, 4835-044, Portugal

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Lisbon, 1649-028, Portugal

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Porto, 4099-001, Portugal

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Bucharest, 011172, Romania

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Cluj-Napoca, 400000, Romania

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Belgrade, 11000, Serbia

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Seoul, 03080, South Korea

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Suwon, 16499, South Korea

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Madrid, 28007, Spain

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Kaohsiung City, 833401, Taiwan

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Taichung, 40447, Taiwan

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Tainan, 710, Taiwan

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Taipei, 10002, Taiwan

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Ankara, 06560, Turkey (Türkiye)

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Antalya, 07070, Turkey (Türkiye)

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Istanbul, 34096, Turkey (Türkiye)

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Izmir, 35210, Turkey (Türkiye)

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İzmit, 41380, Turkey (Türkiye)

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Bath, BA1 3NG, United Kingdom

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Manchester, M13 9PT, United Kingdom

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Wolverhampton, WV10 0QP, United Kingdom

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Related Links

MeSH Terms

Conditions

Dermatomyositis

Condition Hierarchy (Ancestors)

PolymyositisMyositisMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin Diseases

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 24, 2022

First Posted

June 29, 2022

Study Start

October 31, 2022

Primary Completion

July 17, 2025

Study Completion (Estimated)

July 1, 2026

Last Updated

September 29, 2025

Record last verified: 2025-09

Data Sharing

IPD Sharing
Will share

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.

Shared Documents
STUDY PROTOCOL, SAP, 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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