Clinical trial NCT05437263: A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults with Dermatomyositis
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Oral Brepocitinib in Adults with Dermatomyositis
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Dermatomyositis
Keywords: brepocitinib
dermatomyositis
TYK2/JAK1 inhibitor
PF-06700841
PVT-2201
Lead sponsor: Priovant Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2022-06-29
Enrollment: 241
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility 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 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

Primary outcomes:
measure: Total Improvement Score (TIS) at Week 52
timeFrame: 52 weeks
Secondary outcomes:
measure: TIS responder rate after 52 weeks of brepocitinib administration QD, in comparison to placebo
timeFrame: 52 weeks
measure: Change from baseline in HAQ Disability Index score at Week 52
timeFrame: 52 weeks
measure: Cutaneous Dermatomyositis Area and Severity Index (CDASI) Activity Score after 52 weeks of brepocitinib administration QD, in comparison to placebo
timeFrame: 52 weeks
measure: Average daily prednisone-equivalent dose from Week 36 to Week 52 minus baseline
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Clinical Trial Site
state: Arizona
city: Scottsdale
country: United States
facility: Clinical Trial Site
state: Arizona
city: Scottsdale
country: United States
facility: Clinical Trial Site
state: Arizona
city: Irvine
country: United States
facility: Clinical Trial Site
state: California
city: Los Angeles
country: United States
facility: Clinical Trial Site
state: California
city: San Francisco
country: United States
facility: Clinical Trial Site
state: California
city: Aurora
country: United States
facility: Clinical Trial Site
state: Colorado
city: Denver
country: United States
facility: Clinical Trial Site
state: Colorado
city: Boynton Beach
country: United States
facility: Clinical Trial Site
state: Florida
city: Gainesville
country: United States
facility: Clinical Trial Site
state: Florida
city: Jacksonville
country: United States
facility: Clinical Trial Site
state: Florida
city: Plantation
country: United States
facility: Clinical Trial Site
state: Florida
city: Tampa
country: United States
facility: Clinical Trial Site
state: Florida
city: Atlanta
country: United States
facility: Clinical Trial Site
state: Georgia
city: Augusta
country: United States
facility: Clinical Trial Site
state: Georgia
city: Marietta
country: United States
facility: Clinical Trial Site
state: Georgia
city: Chicago
country: United States
facility: Clinical Trial Site
state: Illinois
city: Iowa City
country: United States
facility: Clinical Trial Site
state: Iowa
city: Kansas City
country: United States
facility: Clinical Trial Site
state: Kansas
city: New Orleans
country: United States
facility: Clinical Trial Site
state: Louisiana
city: New Orleans
country: United States
facility: Clinical Trial Site
state: Louisiana
city: Baltimore
country: United States
facility: Clinical Trial Site
state: Maryland
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: Ann Arbor
country: United States
facility: Clinical Trial Site
state: Michigan
city: Minneapolis
country: United States
facility: Clinical Trial Site
state: Minnesota
city: Rochester
country: United States
facility: Clinical Trial Site
state: Minnesota
city: Manhasset
country: United States
facility: Clinical Trial Site
state: New York
city: New York
country: United States
facility: Clinical Trial Site
state: New York
city: New York
country: United States
facility: Clinical Trial Site
state: New York
city: Cincinnati
country: United States
facility: Clinical Trial Site
state: Ohio
city: Cleveland
country: United States
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state: Ohio
city: Oklahoma City
country: United States
facility: Clinical Trial Site
state: Oklahoma
city: Portland
country: United States
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state: Oregon
city: Philadelphia
country: United States
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state: Pennsylvania
city: Pittsburgh
country: United States
facility: Clinical Trial Site
state: Pennsylvania
city: Jackson
country: United States
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state: Tennessee
city: Austin
country: United States
facility: Clinical Trial Site
state: Texas
city: Houston
country: United States
facility: Clinical Trial Site
state: Texas
city: Irving
country: United States
facility: Clinical Trial Site
state: Texas
city: Quilmes
country: Argentina
facility: Clinical Trial Site
state: Buenos Aires
city: Caba
country: Argentina
facility: Clinical Trial Site
city: Mendoza
country: Argentina
facility: Clinical Trial Site
city: Leuven
country: Belgium
facility: Clinical Trial Site
city: Plovdiv
country: Bulgaria
facility: Clinical Trial Site
city: Plovdiv
country: Bulgaria
facility: Clinical Trial Site
city: Plovdiv
country: Bulgaria
facility: Clinical Trial Site
city: Sofia
country: Bulgaria
facility: Clinical Trial Site
city: Vancouver
country: Canada
facility: Clinical Trial Site
state: British Colombia
city: Newmarket
country: Canada
facility: Clinical Trial Site
state: Ontario
city: Concepción
country: Chile
facility: Clinical Trial Site
state: Region del Bio Bio
city: Recoleta
country: Chile
facility: Clinical Trial Site
city: Santiago
country: Chile
facility: Clinical Trial Site
city: Santiago
country: Chile
facility: Clinical Trial Site
city: Temuco
country: Chile
facility: Clinical Trial Site
city: Prague
country: Czechia
facility: Clinical Trial Site
city: Mainz
country: Germany
facility: Clinical Trial Site
state: Rheinland-Pfalz
city: Berlin
country: Germany
facility: Clinical Trial Site
city: Berlin
country: Germany
facility: Clinical Trial Site
city: Dresden
country: Germany
facility: Clinical Trial Site
city: Essen
country: Germany
facility: Clinical Trial Site
city: Freiburg
country: Germany
facility: Clinical Trial Site
city: Debrecen
country: Hungary
facility: Clinical Trial Site
city: Pécs
country: Hungary
facility: Clinical Trial Site
city: Szeged
country: Hungary
facility: Clinical Trial Site
city: Ashkelon
country: Israel
facility: Clinical Trial Site
city: Haifa
country: Israel
facility: Clinical Trial Site
city: Poriyya
country: Israel
facility: Clinical Trial Site
city: Tel Aviv
country: Israel
facility: Clinical Trial Site
city: Tel HaShomer
country: Israel
facility: Clinical Trial Site
city: Bari
country: Italy
facility: Clinical Trial Site
city: Pavia
country: Italy
facility: Clinical Trial Site
city: Roma
country: Italy
facility: Clinical Trial Site
city: Torino
country: Italy
facility: Clinical Trial Site
city: Seoul
country: Korea, Republic of
facility: Clinical Trial Site
city: Suwon
country: Korea, Republic of
facility: Clinical Trial Site
city: Monterrey
country: Mexico
facility: Clinical Trial Site
state: Nuevo Leon
city: Mérida
country: Mexico
facility: Clinical Trial Site
state: Yucatan
city: Guadalajara
country: Mexico
facility: Clinical Trial Site
city: Mexico City
country: Mexico
facility: Clinical Trial Site
city: San Luis Potosí
country: Mexico
facility: Clinical Trial Site
city: Nijmegen
country: Netherlands
facility: Clinical Trial Site
state: Gelderland
city: Amsterdam
country: Netherlands
facility: Clinical Trial Site
city: Białystok
country: Poland
facility: Clinical Trial Site
state: Podlaskie
city: Kraków
country: Poland
facility: Clinical Trial Site
city: Lublin
country: Poland
facility: Clinical Trial Site
city: Lublin
country: Poland
facility: Clinical Trial Site
city: Nowa Sól
country: Poland
facility: Clinical Trial Site
city: Poznań
country: Poland
facility: Clinical Trial Site
city: Warsaw
country: Poland
facility: Clinical Trial Site
city: Vila Nova De Gaia
country: Portugal
facility: Clinical Trial Site
state: Porto
city: Guimarães
country: Portugal
facility: Clinical Trial Site
city: Lisboa
country: Portugal
facility: Clinical Trial Site
city: Porto
country: Portugal
facility: Clinical Trial Site
city: Bucharest
country: Romania
facility: Clinical Trial Site
city: Cluj-Napoca
country: Romania
facility: Clinical Trial Site
city: Belgrade
country: Serbia
facility: Clinical Trial Site
city: Belgrad
country: Serbia
facility: Clinical Trial Site
city: Madrid
country: Spain
facility: Clinical Trial Site
city: Kaohsiung
country: Taiwan
facility: Clinical Trial Site
city: Taichung
country: Taiwan
facility: Clinical Trial Site
city: Tainan
country: Taiwan
facility: Clinical Trial Site
city: Taipei
country: Taiwan
facility: Clinical Trial Site
city: Ankara
country: Turkey
facility: Clinical Trial Site
city: Antalya
country: Turkey
facility: Clinical Trial Site
city: Istanbul
country: Turkey
facility: Clinical Trial Site
city: İzmir
country: Turkey
facility: Clinical Trial Site
city: İzmit
country: Turkey
facility: Clinical Trial Site
city: Bath
country: United Kingdom
facility: Clinical Trial Site
city: Manchester
country: United Kingdom
facility: Clinical Trial Site
city: Wolverhampton
country: United Kingdom
facility: Clinical Trial Site
Study officials:
References: