(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
Condition(s) studied
Investigational drug(s) / intervention(s)
Avapritinib: Avapritinib tablet
Placebo: Placebo tablet
Study 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.
Eligibility
Primary outcome measure(s)
- Part 1: Recommended Phase 2 dose (RP2D) in patients with ISM — 9 months
- Part 2: Mean change in ISM Symptom Assessment Form (ISM-SAF) total symptom score (TSS) as compared to placebo — 6 months
0 - 110 points (higher value represents worse symptom outcomes) - Part 3: Number of Participants with Adverse Events — Up to 5 years
Trial sites (49)
| Facility | City | Region | Status |
|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | |
| Mayo Clinic Hospital | Phoenix | Arizona | |
| Stanford Cancer Institute | Stanford | California | |
| Mayo Clinic Florida | Jacksonville | Florida | |
| H. Lee Moffitt Cancer Center | Tampa | Florida | |
| Winship Cancer Institute, Emory University | Atlanta | Georgia | |
| Rush University Medical Center | Chicago | Illinois | |
| University of Kansas Hospital | Kansas City | Kansas | |
| Brigham & Women's Hospital | Boston | Massachusetts | |
| Dana Farber Cancer Institute | Boston | Massachusetts | |
| Michigan Medicine, University of Michigan | Ann Arbor | Michigan | |
| Mayo Clinic | Rochester | Minnesota | |
| Washington University School of Medicine | St Louis | Missouri | |
| Herbert Irving Comprehensive Cancer Center | New York | New York | |
| Duke University Health System (DUHS) | Durham | North Carolina | |
| University Hospitals Cleveland Medical Center | Cleveland | Ohio | |
| University of Texas, MD Anderson Cancer Center | Houston | Texas | |
| Huntsman Cancer Institute | Salt Lake City | Utah | |
| Virginia Commonwealth University Medical Center | Richmond | Virginia | |
| University Hospital Antwerp | Edegem | Belgium | |
| Tom Baker Cancer Centre | Calgary | Alberta | |
| University of Alberta Hospital | Edmonton | Alberta | |
| St. Michael's Hospital | Toronto | Ontario | |
| 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 |
+ 9 more sites — see the full list on the official registry below.
More Blueprint Medicines Corporation trials in Italy
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT03731260 on ClinicalTrials.gov ↗ ← All trials in Italy