Clinical trial NCT05895786: A Study to Understand How the Study Medicine (PF-06823859) Works in People With Active Idiopathic Inflammatory Myopathies [Dermatomyositis (DM) and Polymyositis (PM)]
Official title: A PHASE 3, MULTICENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PF-06823859 IN PARTICIPANTS WITH ACTIVE IDIOPATHIC INFLAMMATORY MYOPATHIES (INCLUDING PARTICIPANTS WITH ACTIVE DERMATOMYOSITIS OR POLYMYOSITIS)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Myositis
Keywords: Dermatomyositis
Polymyositis
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2023-06-09
Enrollment: 270
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Randomized, double-blind, placebo-controlled, parallel-group study to assess whether the study medicine will work to lessen muscle weakness and skin symptoms in adults with active polymyositis (PM) or dermatomyositis (DM). Eligible participants are required to have active disease at the time of enrollment and are required to enter the study on protocol-permitted standard of care (SOC) background medication.

The study will be conducted in 2 parallel cohorts: Cohort 1 (DM) and Cohort 2 (PM).
Primary purpose: TREATMENT

Brief summary: The purpose of the study is to understand how the study medicine PF-06823859 works in people with idiopathic inflammatory myopathies (DM and PM). These disorders cause inflammation that weakens the muscles that are important for movement and may also cause skin rash in people with DM.

This study is seeking participants who:

* Are 18 years of age or older or minimum legal adult age as defined per local regulation, whichever is greater
* Have active DM or active PM.
* Are receiving a stable dose of 1 corticosteroid taken by mouth and/or 1 traditional immunosuppressant.

  * Note: Corticosteroids and immunosuppressants are medicines that help reduce inflammation and may signal to the immune system not to attack the body.

Dermatomyositis (DM) is a rare disease that causes muscle inflammation that results in muscle weakness and low muscle stamina. Patients with DM have a characteristic skin rash. Polymyositis (PM) is a rare disease that involves mainly muscle inflammation resulting in muscle weakness, that can sometimes be painful. Patients with DM and PM may have trouble going up the steps, walking or getting to a standing position.

Some of the participants will receive the study medicine (PF-06823859) and some will receive placebo (which is similar to study medicine but contains no medicine in it).

The study medicine or placebo will be given as an intravenous (IV) infusion (directly into the veins), which takes about1 hour; every 4 weeks from Day 1 to Week 48 of the study. Both PF-06823859 and placebo and will be given at the study site.

The study will compare the experiences of people receiving study medication to those of the people who do not. This will help to see if PF-06823859 is safe and effective.

Participants will take part in this study for about 13 months. During this time, participants will have 15 study visits. These visits will be performed at the study site.


Eligibility criteria: Inclusion Criteria:

* Male or female adults (≥18 years old or minimum legal adult age as defined per local regulation, whichever is greater)
* Active dermatomyositis (DM) or polymyositis (PM) with age of onset

  * 18 years old.
* Must be receiving a stable dose of standard of care (SOC) background medications at the time of enrollment.

Exclusion Criteria:

* Myositis due to non-Idiopathic inflammatory myopathies (non-IIM)
* Existing diagnosis of inclusion body myositis (IBM)
* Presence of immune-mediated necrotizing myositis (IMNM)
* Myositis with end-stage organ involvement
* Active bacterial, viral or fungal infections or hospitalizations for serious infections within 60 days prior to enrollment
* History of recurrent bacterial, viral, fungal, mycobacterial or other infections
* Clinically significant finding on a chest x-ray
* Have cancer or a history of cancer within 5 years of screening
* Significant current or prior disease conditions that may interfere with the response to or safety of the study medicine, including but not limited to:
* history of major organ transplant
* acute coronary syndrome or any history of significant cerebrovascular disease within 24 weeks of screening
* preexisting demyelinating disorder such as multiple sclerosis, or other severe neurological disorder
* major surgery within 4 weeks of screening, or scheduled to occur during the study, excluding diagnostic surgery
* previous treatment with total lymphoid irradiation
* history of any lymphoproliferative disorder such as Epstein Barr Virus, history of lymphoma, leukemia, or symptoms of current lymphatic or lymphoid disease
* Clinically significant depression, suicidal ideation, or previous history of suicidal behaviors
* Other medical or laboratory abnormality that may increase the risk of study participation
* Previous administration with an investigational product (drug or vaccine) within 30 days or of the first dose of study medicine
* Current use or incomplete appropriate washout period of any prohibited medication(s) or known exposure to anti-interferon beta (PF-06823859) or any type of anti-interferon beta therapy
* Prior SOC medication that does not fulfill the criteria
* Certain laboratory results from screening assessments that may interfere with study participation.
* Investigator site staff directly involved in the conduct of the study and their family members, site staff and sponsor and sponsor delegate employees directly involved in the conduct of the study and their family members

Primary outcomes:
measure: Moderate change in Total Improvement Score (TIS)
timeFrame: 24 weeks outside of the United States (US) and 52 weeks in the US
Secondary outcomes:
measure: Change from baseline in Manual Muscle Testing - 8 designated muscles (MMT-8)
timeFrame: 24 weeks outside of the US and 52 weeks in the US
measure: Change from baseline in Cutaneous Dermatomyositis Disease Area and Severity Index Activity Score (CDASI-A) in participants with dermatomyositis (DM)
timeFrame: Week 24 outside the US
measure: Change from baseline in Investigator Global Assessment severity scale (IGA) in participants with dermatomyositis
timeFrame: 24 and 52 weeks in the US only
measure: Corticosteroid (CS) dose assessment
timeFrame: 52 weeks
measure: Moderate change in Total Improvement Score
timeFrame: 24 weeks in the US and 52 weeks outside of the US
measure: Change from baseline in Patient-Reported Outcomes Measurement Information System - Physical Function (PROMIS-PF)
timeFrame: 24 weeks outside of the US and 52 weeks in the US
measure: Change from baseline in 5-D Itch Scale Score
timeFrame: 24 weeks outside of the US and 52 weeks in the US
measure: Change from baseline in Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-F)
timeFrame: 24 weeks outside of the US and 52 weeks in the US
measure: Response in corticosteroid tapering
timeFrame: 52 weeks US only
Other outcomes:
Locations:
city: Glendale
country: United States
facility: AARA Clinical Research - Arizona Arthritis & Rheumatology Associates - Glendale
state: Arizona
city: Glendale
country: United States
facility: Arizona Arthritis & Rheumatology Associates, P.C.
state: Arizona
city: Phoenix
country: United States
facility: Mayo Clinic Hospital
state: Arizona
city: Scottsdale
country: United States
facility: Mayo Clinic in Arizona - Scottsdale
state: Arizona
city: Los Angeles
country: United States
facility: 200 UCLA Medical Plaza
state: California
city: Los Angeles
country: United States
facility: UCLA Clinical & Translational Research Center (CTRC)
state: California
city: Los Angeles
country: United States
facility: UCLA
state: California
city: Orange
country: United States
facility: Center for Clinical Research - Chapman Pavilion
state: California
city: Orange
country: United States
facility: UCI Douglas Hospital
state: California
city: Orange
country: United States
facility: UCI Health Center for Innovative Health Therapies
state: California
city: Orange
country: United States
facility: University of California - Irvine
state: California
city: Coral Gables
country: United States
facility: University of Miami
state: Florida
city: Jacksonville
country: United States
facility: Mayo Clinic in Florida
state: Florida
city: Margate
country: United States
facility: Lal Bhagchandani, M.D
state: Florida
city: Miami
country: United States
facility: University of Miami Dermatology Clinical Trials Unit
state: Florida
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Orlando
country: United States
facility: Omega Research Orlando
state: Florida
city: Plantation
country: United States
facility: IRIS Research and Development, LLC
state: Florida
city: Plantation
country: United States
facility: West Broward Pulmonary Consultants
state: Florida
city: Tamarac
country: United States
facility: West Broward Rheumatology Associates
state: Florida
city: Fairway
country: United States
facility: KU Clinical Research Center - Clinical and Translational Science Unit (CTSU)
state: Kansas
city: Kansas City
country: United States
facility: KU Clinical Research Center - Clinical and Translational Science Unit (CTSU) Rainbow
state: Kansas
city: Kansas City
country: United States
facility: University of Kansas Medical Center - Hoglund Brain Imaging Center
state: Kansas
city: Kansas City
country: United States
facility: University of Kansas Medical Center
state: Kansas
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Boston
country: United States
facility: CTH - Brigham & Women's Hospital
state: Massachusetts
city: Saint Louis
country: United States
facility: Washington University Pulmonary Function Laboratory
state: Missouri
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: Brooklyn
country: United States
facility: Joseph S. and Diane H. Steinberg Ambulatory Care Center
state: New York
city: New York
country: United States
facility: NYU Langone Health - Clinical and Translational Science Institute Research Pharmacy
state: New York
city: New York
country: United States
facility: NYU Langone Health Clinical Research Center
state: New York
city: New York
country: United States
facility: NYU Langone Health
state: New York
city: New York
country: United States
facility: NYU Langone Radiology
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Allen
country: United States
facility: Arthritis & Rheumatology Research Institute
state: Texas
city: Edinburg
country: United States
facility: Integrative Rheumatology of South Texas
state: Texas
city: Harlingen
country: United States
facility: Integrative Rheumatology of South Texas - Harlingen
state: Texas
city: Houston
country: United States
facility: Nerve & Muscle Center of Texas
state: Texas
city: Weslaco
country: United States
facility: Pulmonary and Sleep Center of the Valley
state: Texas
city: Beckley
country: United States
facility: Rheumatology & Pulmonary Clinic
state: West Virginia
city: Zárate
country: Argentina
facility: Instituto de Investigaciones Clinicas Zarate
state: Buenos Aires
city: Quilmes
country: Argentina
facility: CER medical Institute
state: Ciudad Autónoma DE Buenos Aires
city: SAN M. DE Tucuman
country: Argentina
facility: Centro de Investigaciones Médicas Tucuman
state: Tucumán
city: Ciudad Autónoma de Buenos Aires
country: Argentina
facility: Hospital J. M. Ramos Mejía
city: Mendoza
country: Argentina
facility: Instituto de Reumatología
city: Leuven
country: Belgium
facility: UZ Leuven
state: Vlaams-brabant
city: Plovdiv
country: Bulgaria
facility: Medical Center Artmed
city: Sofia
country: Bulgaria
facility: Diagnostic Consultative Centre (DCC) - Foкus 5
city: Hefei
country: China
facility: Anhui Provincial Hospital
state: Anhui
city: Beijing
country: China
facility: China-Japan Friendship Hospital
state: Beijing
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
city: Guangzhou
country: China
facility: Guangdong Provincial People's Hospital
state: Guangdong
city: Guangzhou
country: China
facility: Guangdong Provincial People's Hospital
state: Guangdong
city: Cangzhou
country: China
facility: Cangzhou People's Hospital
state: Hebei
city: Changsha
country: China
facility: Xiangya Hospital Central South University
state: Hunan
city: Nanchang
country: China
facility: The Second Affiliated Hospital of Nanchang University
state: Jiangxi
city: Jinan
country: China
facility: Qilu Hospital of Shandong University
state: Shandong
city: Shanghai
country: China
facility: Ruijin Hospital Shanghai Jiaotong University School of Medicine
state: Shanghai
city: Shanghai
country: China
facility: Huashan Hospital, Fudan University
state: Shanghai
city: Shanghai
country: China
facility: Shanghai Jiaotong University School of Medicine, Renji Hospital
state: Shanghai
city: Cheng Du
country: China
facility: West China Hospital, Sichuan University
state: Sichuan
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
state: Tianjin
city: Kunming
country: China
facility: First Affiliated Hospital of Kunming Medical University
state: Yunnan Sheng
city: Hangzhou
country: China
facility: The first Affiliated Hospital, Zhejiang University School of Medicine
state: Zhejiang
city: Reims
country: France
facility: Centre Hospitalier Universitaire de Reims - l'Hôpital Maison Blanche
city: Strasbourg
country: France
facility: Hopitaux Universitaires de Strasbourg, Nouvel Hopital Civil
city: Erlangen
country: Germany
facility: Universitaetsklinikum Erlangen
state: Bayern
city: Munchen
country: Germany
facility: Ludwig Maximilians University Munich Klinikum
city: Debrecen
country: Hungary
facility: Debreceni Egyetem Klinikai Kozpont
city: Surat
country: India
facility: Institute of Neurosciences
state: Gujarat
city: Gurugram (Gurgaon)
country: India
facility: Medanta-The Medicity
state: Haryana
city: Cochin
country: India
facility: CHARM HEALTHCARE PRIVATE LIMITED (Previously Dr Shenoy's Care Private Limited)
state: Kerala
city: Kolkata
country: India
facility: Institute of Post Graduate Medical Education and Research and Seth Sukhlal Karnani Memorial Hospital
state: WEST Bengal
city: Petah Tikva
country: Israel
facility: Rabin Medical Center
state: Hamerkaz
city: Ramat Gan
country: Israel
facility: Sheba Medical Center
state: Hamerkaz
city: Tel Aviv
country: Israel
facility: Sourasky Medical Center
state: Tell Abīb
city: Rome
country: Italy
facility: Fondazione Policlinico Tor Vergata
state: Lazio
city: Rozzano
country: Italy
facility: IRCCS Istituto Clinico Humanitas
state: Milano
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
state: RM
city: Brescia
country: Italy
facility: Azienda Ospedaliera Spedali Civili di Brescia
city: Catania
country: Italy
facility: Azienda Ospedaliero Universitaria Policlinico G.Rodolico-San Marco Di Catania
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Careggi
city: Milano
country: Italy
facility: Irccs Ospedale San Raffaele
city: Kitakyushu-shi
country: Japan
facility: Hospital of the university of occupational and environmental health
state: Fukuoka
city: Kawasaki
country: Japan
facility: St. Marianna University School of Medicine Hospital
state: Kanagawa
city: Sendai
country: Japan
facility: Tohoku University Hospital
state: Miyagi
city: Okayama-shi
country: Japan
facility: Okayama City General Medical Center Okayama City Hospital
state: Okayama
city: Kawachinagano
country: Japan
facility: National Hospital Organization Osaka Minami Medical Center
state: Osaka
city: Otsu
country: Japan
facility: Shiga University of Medical Science Hospital
state: Shiga
city: Bunkyo-ku
country: Japan
facility: Institute of Science Tokyo Hospital
state: Tokyo
city: Bunkyo-ku
country: Japan
facility: Nippon Medical School Hospital
state: Tokyo
city: Shinjuku-ku
country: Japan
facility: Tokyo Medical University Hospital
state: Tokyo
city: Gwangju
country: Korea, Republic of
facility: Chonnam National University Bitgoeul Hospital
state: Kwangju-kwangyokshi
city: Seoul
country: Korea, Republic of
facility: Kyung Hee University Hospital
state: Seoul-teukbyeolsi [seoul]
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Cuauhtémoc
country: Mexico
facility: Cryptex Investigación Clínica S.A. de C.V.
state: Distrito Federal
city: Mexico City
country: Mexico
facility: CITER Centro de Investigación y Tratamiento de las Enfermedades Reumáticas
state: Distrito Federal
city: Guadalajara
country: Mexico
facility: Centro Integral en Reumatologia
state: Jalisco
city: Cuernavaca
country: Mexico
facility: Private Practice - Dr. Miguel Cortes
state: Morelos
city: Warsaw
country: Poland
facility: Narodowy Instytut Geriatrii, Reumatologii i Rehabilitacji
state: Mazowieckie
city: Krakow
country: Poland
facility: Centrum Medyczne Plejady
state: Małopolskie
city: Kraków
country: Poland
facility: Pracownia Radiologiczna WIDOK-MED
state: Małopolskie
city: Kraków
country: Poland
facility: Małopolskie Centrum Kliniczne
state: Małopolskie
city: Kraków
country: Poland
facility: Lux Med
state: Małopolskie
city: Kraków
country: Poland
facility: 5 Wojskowy Szpital Kliniczny z Polikliniką SPZOZ
state: Małopolskie
city: Bialystok
country: Poland
facility: Centrum Medycyny Oddechowej Robert Mroz spolka jawna
state: Podlaskie
city: Bialystok
country: Poland
facility: Nova Reuma Domyslawska i Rusilowicz Spolka Partnerska Lekarza Reumatologa i Fizjoterapeuty
state: Podlaskie
city: Białystok
country: Poland
facility: INTER CLINIC Piotr Adrian Klimiuk
state: Podlaskie
city: Białystok
country: Poland
facility: Zaklad Diagnostyki Obrazowej HEM s.c.
state: Podlaskie
city: Piešťany
country: Slovakia
facility: Narodny ustav reumatickych chorob
city: La Laguna
country: Spain
facility: Hospital Universitario de Canarias
state: Santa CRUZ DE Tenerife
city: Madrid
country: Spain
facility: Hospital General Universitario Gregorio Marañon
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Stockholm
country: Sweden
facility: Karolinska University Hospital Solna
city: Tainan City
country: Taiwan
facility: Chi Mei Medical Center
state: Tainan
city: Taichung
country: Taiwan
facility: China Medical University Hospital
city: Taichung
country: Taiwan
facility: Taichung Veterans General Hospital
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Taipei
country: Taiwan
facility: Taipei Veterans General Hospital
city: Taoyuan
country: Taiwan
facility: Chang Gung Medical Foundation-Linkou Branch
city: Ankara
country: Turkey
facility: Ankara University Ibni Sina Hospital
city: Ankara
country: Turkey
facility: Hacettepe Universite Hastaneleri
city: Ankara
country: Turkey
facility: Memorial Ankara Hastanesi
city: Ankara
country: Turkey
facility: Memorial Ankara Hastanesi
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital
state: Glasgow CITY
city: Salford
country: United Kingdom
facility: Salford Royal Hospital
state: Manchester
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: