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(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis
Sponsor: Blueprint Medicines Corporation
Summary
This is a Phase 2, randomized, double-blind, placebo-controlled study comparing the efficacy and safety of avapritinib + best supportive care (BSC) with placebo + BSC in patients with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by BSC. The study will be conducted in 3 parts. All patients will receive treatment with avapritinib during Part 3 including those rolling over from the placebo group.
Official title: A 3-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate Safety and Efficacy of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Indolent and Smoldering Systemic Mastocytosis With Symptoms Inadequately Controlled With Standard Therapy
Key Details
Gender
All
Age Range
18 Years - Any
Study Type
INTERVENTIONAL
Enrollment
251
Start Date
2019-04-16
Completion Date
2027-06-23
Last Updated
2025-09-23
Healthy Volunteers
No
Conditions
Interventions
Avapritinib
Avapritinib tablet
Placebo
Placebo tablet
Locations (49)
University of Alabama at Birmingham
Birmingham, Alabama, United States
Mayo Clinic Hospital
Phoenix, Arizona, United States
Stanford Cancer Institute
Stanford, California, United States
Mayo Clinic Florida
Jacksonville, Florida, United States
H. Lee Moffitt Cancer Center
Tampa, Florida, United States
Winship Cancer Institute, Emory University
Atlanta, Georgia, United States
Rush University Medical Center
Chicago, Illinois, United States
University of Kansas Hospital
Kansas City, Kansas, United States
Brigham & Women's Hospital
Boston, Massachusetts, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
Michigan Medicine, University of Michigan
Ann Arbor, Michigan, United States
Mayo Clinic
Rochester, Minnesota, United States
Washington University School of Medicine
St Louis, Missouri, United States
Herbert Irving Comprehensive Cancer Center
New York, New York, United States
Duke University Health System (DUHS)
Durham, North Carolina, United States
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States
University of Texas, MD Anderson Cancer Center
Houston, Texas, United States
Huntsman Cancer Institute
Salt Lake City, Utah, United States
Virginia Commonwealth University Medical Center
Richmond, Virginia, United States
University Hospital Antwerp
Edegem, Belgium
Tom Baker Cancer Centre
Calgary, Alberta, Canada
University of Alberta Hospital
Edmonton, Alberta, Canada
St. Michael's Hospital
Toronto, Ontario, Canada
Odense Universitetshospital, ORCA/Allergicentret, Hudafdeling I og Allergicenter
Odense, Denmark
Hôpital de la Timone, Service de dermatologie
Marseille, France
Hôpital Pitié-Salpêtrière, Service de Dermatologie
Paris, France
CHU Toulouse Larrey, CEREMAST, Service de Dermatologie et Allergologie cutanée
Toulouse, France
Uniklinik RWTH Aachen
Aachen, Germany
Charité Universitätsmedizin Berlin
Berlin, Germany
University Clinic Hamburg Eppendorf, University Cancer Center Hamburg (UCCH)
Hamburg, Germany
Universitätsklinikum Schleswig-Holstein, Hämatologie/Onkologie
Lübeck, Germany
Universitätsklinik Mainz, Universitäts-Hautklinik, Clinical Research Center
Mainz, Germany
Universitätsmedizin Mannheim, III. Medizinische Klinik
Mannheim, Germany
Klinikum rechts der Isar, Technische Universität München
Munich, Germany
A.O.U di Bologna - IRCCS, Istituto di Ematologia Lorenzo e Ariosto Seragnoli, Ematologia
Bologna, Italy
Fondazione IRCCS Ca' Granda Ospedale Maggiore Poloclinico, UOC Ematologia
Milan, Italy
A.O. OO.RR. S.Giovanni di Dio e Ruggi d'Aragona, University of Salerno
Salerno, Italy
Azienda Ospedaliera Universitaria Integrata di Verona
Verona, Italy
University Medical Center Groningen (UMCG)
Groningen, Netherlands
Erasmus Medical Center
Rotterdam, Netherlands
Oslo Universitetssykehus, Rikshospitalet, Department of Hematology
Oslo, Norway
Hospital Universitari Vall d'Hebron
Barcelona, Spain
lnstituto de Estudios de Mastocitosis de Castilla la Mancha, Hospital Virgen del Valle - Complejo Hospitalario de Toledo
Toledo, Spain
Karolinska University Hospital, Hematologimottagningen R51
Stockholm, Sweden
Akademiska sjukhuset, Hematologmottagningen/101A
Uppsala, Sweden
University Hospital Basel
Basel, Switzerland
NHS Greater Glasgow and Clyde, Beatson West of Scotland Cancer Centre
Glasgow, United Kingdom
Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital
London, United Kingdom
Clatterbridge Cancer Centre NHS Foundation Trust
Metropolitan Borough of Wirral, United Kingdom