Dermatomyositis Clinical Trial
— VALOROfficial 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.
| Status | Recruiting |
| Enrollment | 225 |
| Est. completion date | December 2024 |
| Est. primary completion date | December 2024 |
| Accepts healthy volunteers | No |
| Gender | All |
| Age group | 18 Years to 75 Years |
| Eligibility | 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 |
| Country | Name | City | State |
|---|---|---|---|
| Argentina | Clinical Trial Site | Caba | |
| Argentina | Clinical Trial Site | Mendoza | |
| Argentina | Clinical Trial Site | Quilmes | Buenos Aires |
| Belgium | Clinical Trial Site | Leuven | |
| Bulgaria | Clinical Trial Site | Plovdiv | |
| Bulgaria | Clinical Trial Site | Plovdiv | |
| Bulgaria | Clinical Trial Site | Plovdiv | |
| Bulgaria | Clinical Trial Site | Sofia | |
| Canada | Clinical Trial Site | Newmarket | Ontario |
| Canada | Clinical Trial Site | Vancouver | British Colombia |
| Chile | Clinical Trial Site | Concepción | Region Del Bio Bio |
| Chile | Clinical Trial Site | Recoleta | |
| Chile | Clinical Trial Site | Santiago | |
| Chile | Clinical Trial Site | Santiago | |
| Chile | Clinical Trial Site | Temuco | |
| Czechia | Clinical Trial Site | Prague | |
| Germany | Clinical Trial Site | Berlin | |
| Germany | Clinical Trial Site | Berlin | |
| Germany | Clinical Trial Site | Dresden | |
| Germany | Clinical Trial Site | Essen | |
| Germany | Clinical Trial Site | Freiburg | |
| Germany | Clinical Trial Site | Mainz | Rheinland-Pfalz |
| Hungary | Clinical Trial Site | Debrecen | |
| Hungary | Clinical Trial Site | Pécs | |
| Hungary | Clinical Trial Site | Szeged | |
| Israel | Clinical Trial Site | Ashkelon | |
| Israel | Clinical Trial Site | Haifa | |
| Israel | Clinical Trial Site | Poriyya | |
| Israel | Clinical Trial Site | Tel Aviv | |
| Israel | Clinical Trial Site | Tel HaShomer | |
| Italy | Clinical Trial Site | Bari | |
| Italy | Clinical Trial Site | Pavia | |
| Italy | Clinical Trial Site | Roma | |
| Italy | Clinical Trial Site | Torino | |
| Korea, Republic of | Clinical Trial Site | Seoul | |
| Korea, Republic of | Clinical Trial Site | Suwon | |
| Mexico | Clinical Trial Site | Guadalajara | |
| Mexico | Clinical Trial Site | Mérida | Yucatan |
| Mexico | Clinical Trial Site | Mexico City | |
| Mexico | Clinical Trial Site | Monterrey | Nuevo Leon |
| Mexico | Clinical Trial Site | San Luis Potosí | |
| Netherlands | Clinical Trial Site | Amsterdam | |
| Netherlands | Clinical Trial Site | Nijmegen | Gelderland |
| Poland | Clinical Trial Site | Bialystok | Podlaskie |
| Poland | Clinical Trial Site | Kraków | |
| Poland | Clinical Trial Site | Lublin | |
| Poland | Clinical Trial Site | Lublin | |
| Poland | Clinical Trial Site | Nowa Sól | |
| Poland | Clinical Trial Site | Poznan | |
| Poland | Clinical Trial Site | Warsaw | |
| Portugal | Clinical Trial Site | Guimarães | |
| Portugal | Clinical Trial Site | Lisboa | |
| Portugal | Clinical Trial Site | Porto | |
| Portugal | Clinical Trial Site | Vila Nova De Gaia | Porto |
| Romania | Clinical Trial Site | Bucharest | |
| Romania | Clinical Trial Site | Cluj-Napoca | |
| Serbia | Clinical Trial Site | Belgrad | |
| Serbia | Clinical Trial Site | Belgrade | |
| Spain | Clinical Trial Site | Madrid | |
| Taiwan | Clinical Trial Site | Kaohsiung | |
| Taiwan | Clinical Trial Site | Taichung | |
| Taiwan | Clinical Trial Site | Tainan | |
| Taiwan | Clinical Trial Site | Taipei | |
| Turkey | Clinical Trial Site | Ankara | |
| Turkey | Clinical Trial Site | Antalya | |
| Turkey | Clinical Trial Site | Istanbul | |
| Turkey | Clinical Trial Site | Izmir | |
| Turkey | Clinical Trial Site | Izmit | |
| United Kingdom | Clinical Trial Site | Bath | |
| United Kingdom | Clinical Trial Site | Manchester | |
| United Kingdom | Clinical Trial Site | Wolverhampton | |
| United States | Clinical Trial Site | Ann Arbor | Michigan |
| United States | Clinical Trial Site | Atlanta | Georgia |
| United States | Clinical Trial Site | Augusta | Georgia |
| United States | Clinical Trial Site | Aurora | Colorado |
| United States | Clinical Trial Site | Austin | Texas |
| United States | Clinical Trial Site | Baltimore | Maryland |
| United States | Clinical Trial Site | Boston | Massachusetts |
| United States | Clinical Trial Site | Boynton Beach | Florida |
| United States | Clinical Trial Site | Chicago | Illinois |
| United States | Clinical Trial Site | Cincinnati | Ohio |
| United States | Clinical Trial Site | Cleveland | Ohio |
| United States | Clinical Trial Site | Denver | Colorado |
| United States | Clinical Trial Site | Gainesville | Florida |
| United States | Clinical Trial Site | Houston | Texas |
| United States | Clinical Trial Site | Iowa City | Iowa |
| United States | Clinical Trial Site | Irvine | California |
| United States | Clinical Trial Site | Irving | Texas |
| United States | Clinical Trial Site | Jackson | Tennessee |
| United States | Clinical Trial Site | Jacksonville | Florida |
| United States | Clinical Trial Site | Kansas City | Kansas |
| United States | Clinical Trial Site | Los Angeles | California |
| United States | Clinical Trial Site | Manhasset | New York |
| United States | Clinical Trial Site | Marietta | Georgia |
| United States | Clinical Trial Site | Minneapolis | Minnesota |
| United States | Clinical Trial Site | New Orleans | Louisiana |
| United States | Clinical Trial Site | New Orleans | Louisiana |
| United States | Clinical Trial Site | New York | New York |
| United States | Clinical Trial Site | New York | New York |
| United States | Clinical Trial Site | Oklahoma City | Oklahoma |
| United States | Clinical Trial Site | Philadelphia | Pennsylvania |
| United States | Clinical Trial Site | Phoenix | Arizona |
| United States | Clinical Trial Site | Pittsburgh | Pennsylvania |
| United States | Clinical Trial Site | Plantation | Florida |
| United States | Clinical Trial Site | Portland | Oregon |
| United States | Clinical Trial Site | Rochester | Minnesota |
| United States | Clinical Trial Site | San Francisco | California |
| United States | Clinical Trial Site | Scottsdale | Arizona |
| United States | Clinical Trial Site | Scottsdale | Arizona |
| United States | Clinical Trial Site | Tampa | Florida |
| Lead Sponsor | Collaborator |
|---|---|
| Priovant Therapeutics, Inc. |
United States, Argentina, Belgium, Bulgaria, Canada, Chile, Czechia, Germany, Hungary, Israel, Italy, Korea, Republic of, Mexico, Netherlands, Poland, Portugal, Romania, Serbia, Spain, Taiwan, Turkey, United Kingdom,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | 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 | TIS responder rate after 52 weeks of brepocitinib administration QD, in comparison to placebo | Proportion of responders, defined as achieving TIS = 40 points (moderate improvement) at Week 52 | 52 weeks | |
| Secondary | 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 | |
| Secondary | Cutaneous Dermatomyositis Area and Severity Index (CDASI) Activity Score after 52 weeks of brepocitinib administration QD, in comparison to placebo | Change from baseline in Cutaneous Dermatomyositis Area and Severity Index (CDASI) Activity score at Week 52. Cutaneous Dermatomyositis Disease Area and Severity Index Activity Score 0 to 100 with higher scores indicating a worse outcome. | 52 weeks | |
| Secondary | Average daily prednisone-equivalent dose from Week 36 to Week 52 minus baseline | 52 weeks |
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