(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
NCT ID: NCT03731260
Last Updated: 2025-09-23
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE2
251 participants
INTERVENTIONAL
2019-04-16
2027-06-23
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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(Part 1) Avapritinib Dose 1 + BSC
Avapritinib will be administered orally in continuous 28-day cycles
Avapritinib
Avapritinib tablet
(Part 1) Avapritinib Dose 2 + BSC
Avapritinib will be administered orally in continuous 28-day cycles
Avapritinib
Avapritinib tablet
(Part 1) Avapritinib Dose 3 + BSC
Avapritinib will be administered orally in continuous 28-day cycles
Avapritinib
Avapritinib tablet
(Part 1) Placebo + BSC
Placebo will be administered orally in continuous 28-day cycles
Placebo
Placebo tablet
(Part 2) Avapritinib RP2D + BSC
Avapritinib will be administered orally in continuous 28-day cycles
Avapritinib
Avapritinib tablet
(Part 2) Placebo + BSC
Placebo will be administered orally in continuous 28-day cycles
Placebo
Placebo tablet
(Part 3) Avapritinib RP2D + BSC
Avapritinib will be administered orally in continuous 28-day cycles
Avapritinib
Avapritinib tablet
Interventions
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Avapritinib
Avapritinib tablet
Placebo
Placebo tablet
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 2\. Patient must have moderate-to-severe symptoms based on minimum mean total symptom score (TSS) of the ISM Symptom Assessment Form (ISM-SAF) over the 14-day eligibility screening period.
* 3\. Patient must have failed to achieve adequate symptom control for 1 or more Baseline symptoms.
* 4\. For patients receiving corticosteroids, the dose must be ≤ 20 mg/d prednisone or equivalent, and the dose must be stable for ≥ 14 days.
* 5\. Patient must have an Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 2.
Exclusion Criteria
* 2\. Patient must not have received prior treatment with avapritinib.
* 3\. Patient must not have had any cytoreductive therapy including but not limited to masitinib and midostaurin, or investigational agent for \< 14 days or 5 half-lives of the drug (whichever is longer), and for cladribine, interferon alpha, pegylated interferon, or antibody therapy \< 28 days or 5 half-lives of the drug (whichever is longer), before beginning the 14-day ISM-SAF eligibility TSS assessment.
* 4\. Patient must not have received radiotherapy or psoralen and ultraviolet A (PUVA) therapy \< 14 days before beginning the 14-day ISM-SAF eligibility TSS assessment.
* 5\. Patient must not have received any hematopoietic growth factor the preceding 14 days before beginning the 14-day ISM-SAF eligibility TSS assessment.
* 6\. Patient must not have a QT interval corrected using Fridericia's formula (QTcF) of \> 480 msec.
18 Years
ALL
No
Sponsors
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Blueprint Medicines Corporation
INDUSTRY
Responsible Party
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Locations
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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
Countries
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References
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Gotlib J, Castells M, Elberink HO, Siebenhaar F, Hartmann K, Broesby-Olsen S, George TI, Panse J, Alvarez-Twose I, Radia DH, Tashi T, Bulai Livideanu C, Sabato V, Heaney M, Van Daele P, Cerquozzi S, Dybedal I, Reiter A, Pongdee T, Barete S, Ustun C, Schwartz L, Ward BR, Schafhausen P, Vadas P, Bose P, DeAngelo DJ, Rein L, Vachhani P, Triggiani M, Bonadonna P, Rafferty M, Butt NM, Oh ST, Wortmann F, Ungerstedt J, Guilarte M, Taparia M, Kuykendall AT, Arana Yi C, Ogbogu P, Gaudy-Marqueste C, Mattsson M, Shomali W, Giannetti MP, Bidollari I, Lin HM, Sulllivan E, Mar B, Scherber R, Roche M, Akin C, Maurer M. Avapritinib versus Placebo in Indolent Systemic Mastocytosis. NEJM Evid. 2023 Jun;2(6):EVIDoa2200339. doi: 10.1056/EVIDoa2200339. Epub 2023 May 23.
Padilla B, Shields AL, Taylor F, Li X, Mcdonald J, Green T, Boral AL, Lin HM, Akin C, Siebenhaar F, Mar B. Psychometric evaluation of the Indolent Systemic Mastocytosis Symptom Assessment Form (ISM-SAF) in a phase 2 clinical study. Orphanet J Rare Dis. 2021 Oct 18;16(1):434. doi: 10.1186/s13023-021-02037-3.
Other Identifiers
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2018-000588-99
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
BLU-285-2203
Identifier Type: -
Identifier Source: org_study_id
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