(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis
NCT ID: NCT03580655
Last Updated: 2025-04-30
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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COMPLETED
PHASE2
107 participants
INTERVENTIONAL
2018-11-21
2024-12-18
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Avapritinib
Avapritinib will be administered as an immediate release tablet, orally, continuously, in 28-day cycles
Avapritinib
Avapritinib tablet
Interventions
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Avapritinib
Avapritinib tablet
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Patient must have a serum tryptase ≥ 20 ng/mL.
3. Patient must have Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0 to 3.
Exclusion Criteria
2. Patient has received any cytoreductive therapy (including midostaurin and other TKIs, hydroxyurea, azacitidine) or an investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon and any antibody therapy (eg, brentuximab vedotin) less than 28 days before obtaining screening BM biopsy for this study.
3. Patient has eosinophilia and known positivity for the FIP1L1 PGDFRA fusion, unless the patient has demonstrated relapse or PD on prior imatinib therapy. Patients with eosinophilia (\> 1.5 × 10\^9/L), who do not have a detectable KIT D816 mutation, must be tested for a PDGFRA fusion mutation by fluorescence in situ hybridization (FISH) or polymerase chain reaction (PCR).
4. Patient has history of another primary malignancy that has been diagnosed or required therapy within 3 years before the first dose of study drug. The following are exempt from the 3-year limit: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site.
5. Patient has a QT interval corrected using Fridericia's formula (QTcF) \> 480 msec.
6. Patient has a known risk or recent history (12 months before the first dose of study drug) of intracranial bleeding (eg, brain aneurysm, concomitant vitamin K antagonist use).
7. Platelet count \< 50,000/μL (within 4 weeks of the first dose of study drug) or receiving platelet transfusion(s).
8. Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) \>3 x the upper limit of normal (ULN); no restriction if due to suspected liver infiltration by mast cells.
9. Bilirubin \>1.5 × ULN; no restriction if due to suspected liver infiltration by mast cells or Gilbert's disease. (In the case of Gilbert's disease, a direct bilirubin \>2 × ULN would be an exclusion.)
10. Estimated glomerular filtration rate (eGFR) \< 30 mL/min/1.73m2 or creatinine \> 1.5 × ULN.
11. Patient has a primary brain malignancy or metastases to the brain.
12. Patient has a history of a seizure disorder (eg, epilepsy) or requirement for antiseizure medication.
18 Years
ALL
No
Sponsors
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Blueprint Medicines Corporation
INDUSTRY
Responsible Party
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Locations
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Stanford Cancer Institute
Stanford, California, United States
Rush University Medical Center
Chicago, Illinois, United States
The University of Kansas Cancer Center
Westwood, Kansas, United States
Dana Farber Cancer Institute
Boston, Massachusetts, United States
University of Michigan
Ann Arbor, Michigan, United States
Washington University School of Medicine
St Louis, Missouri, United States
Roswell Park Comprehensive Cancer Center
Buffalo, New York, United States
Herbert Irving Comprehensive Cancer Center
New York, New York, United States
University of Pennsylvania, Abramson Cancer Center
Philadelphia, Pennsylvania, United States
University of Texas, MD Anderson Cancer Center
Houston, Texas, United States
Mays Cancer Center
San Antonio, Texas, United States
Huntsman Cancer Institute
Salt Lake City, Utah, United States
Medizinische Universität Wien, Universitätsklinik für Innere Medizin I, Klinische Abteilung für Hämatologie und Hämostaseologie
Vienna, , Austria
St. Michael's Hospital
Toronto, Ontario, Canada
Odense University Hospital, Department of Haematology
Odense, , Denmark
Hôpital Necker-Enfants Malades
Paris, , France
CHU Toulouse - Hôpital Larrey
Toulouse, , France
Uniklinik RWTH Aachen, Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltranslplantation
Aachen, , Germany
Universitätsklinikum Hamburg-Eppendorf, Zentrum für Onkologie
Hamburg, , Germany
Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I - Hämatologie und Zelltherapie, lnternistische Onkologie, Hämostaseologie
Leipzig, , Germany
Universitätsmedizin Mannheim III. Medizinische Klinik
Mannheim, , Germany
Klinik und Poliklinik für Innere Medizin III, Klinikum rechts der Isar der TU München
Munich, , Germany
Azienda Ospedaliero-Universitaria Careggi, CRIMM - Centro di Ricerca ed Innovazione per le Malattie Mieloproliferative
Florence, , Italy
A.O. OO.RR. S.Giovanni di Dio e Ruggi d'Aragona, University of Salerno
Salerno, , Italy
Centro Ricerche Cliniche di Verona - Azienda Ospedaliera Universitaria Integrata di Verona
Verona, , Italy
University Medical Center Groningen (UMCG)
Groningen, , Netherlands
Oslo University Hospital-Rikshospitalet, Hematology
Oslo, , Norway
Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii
Gdansk, , Poland
Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu, Klinice Hematologii, Nowotworów Krwi i Transplantacji Szpiku
Wroclaw, , Poland
lnstituto de Estudios de Mastocitosis de Castilla la Mancha, Hospital Virgen del Valle - Complejo Hospitalario de Toledo
Toledo, , Spain
Beatson West of Scotland Cancer Centre - NHS Greater Glasgow and Clyde
Glasgow, , United Kingdom
Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital
London, , United Kingdom
Countries
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References
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Reiter A, Gotlib J, Alvarez-Twose I, Radia DH, Lubke J, Bobbili PJ, Wang A, Norregaard C, Dimitrijevic S, Sullivan E, Louie-Gao M, Schwaab J, Galinsky IA, Perkins C, Sperr WR, Sriskandarajah P, Chin A, Sendhil SR, Duh MS, Valent P, DeAngelo DJ. Efficacy of avapritinib versus best available therapy in the treatment of advanced systemic mastocytosis. Leukemia. 2022 Aug;36(8):2108-2120. doi: 10.1038/s41375-022-01615-z. Epub 2022 Jul 5.
Reiter A, Schwaab J, DeAngelo DJ, Gotlib J, Deininger MW, Pettit KM, Alvarez-Twose I, Vannucchi AM, Panse J, Platzbecker U, Hermine O, Dybedal I, Lin HM, Rylova SN, Ehlert K, Dimitrijevic S, Radia DH. Efficacy and safety of avapritinib in previously treated patients with advanced systemic mastocytosis. Blood Adv. 2022 Nov 8;6(21):5750-5762. doi: 10.1182/bloodadvances.2022007539.
Gotlib J, Reiter A, Radia DH, Deininger MW, George TI, Panse J, Vannucchi AM, Platzbecker U, Alvarez-Twose I, Mital A, Hermine O, Dybedal I, Hexner EO, Hicks LK, Span L, Mesa R, Bose P, Pettit KM, Heaney ML, Oh ST, Sen J, Lin HM, Mar BG, DeAngelo DJ. Efficacy and safety of avapritinib in advanced systemic mastocytosis: interim analysis of the phase 2 PATHFINDER trial. Nat Med. 2021 Dec;27(12):2192-2199. doi: 10.1038/s41591-021-01539-8. Epub 2021 Dec 6.
Other Identifiers
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2017-004836-13
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
BLU-285-2202
Identifier Type: -
Identifier Source: org_study_id
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