New drug aims to ease symptoms of rare mast cell disease

NCT ID NCT03731260

Ongoing Symptom relief Sponsor: Blueprint Medicines Corporation Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This phase 2 study tests whether avapritinib, a targeted drug taken as a tablet, can reduce symptoms in people with indolent systemic mastocytosis whose symptoms are not well controlled by standard care. About 251 participants will receive either avapritinib or a placebo, and all will eventually get the active drug. The main goal is to see if symptoms improve and to check for side effects.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: avapritinib (a targeted cancer drug taken as a tablet)
What this could lead to: If it works, this could provide a new option for people with indolent systemic mastocytosis whose symptoms are not well controlled by current treatments.
What could go wrong: This is a mid-stage trial, so results are not yet proven. The drug may cause side effects, and not all participants may see symptom improvement.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Get updates

Get notified about this study

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

indolent systemic mastocytosis Mastocytosis, Systemic

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

INDOLENT SYSTEMIC MASTOCYTOSIS

Contacts and locations

Show contact details

Locations

A.O. OO.RR. S.Giovanni di Dio e Ruggi d'Aragona, University of Salerno

Salerno, 84131, Italy
A.O.U di Bologna - IRCCS, Istituto di Ematologia Lorenzo e Ariosto Seragnoli, Ematologia

Bologna, 40138, Italy
Akademiska sjukhuset, Hematologmottagningen/101A

Uppsala, 751 85, Sweden
Azienda Ospedaliera Universitaria Integrata di Verona

Verona, 37126, Italy
Brigham & Women's Hospital

Boston, Massachusetts, 02115, United States
CHU Toulouse Larrey, CEREMAST, Service de Dermatologie et Allergologie cutanée

Toulouse, 31059, France
Charité Universitätsmedizin Berlin

Berlin, 10117, Germany
Clatterbridge Cancer Centre NHS Foundation Trust

Metropolitan Borough of Wirral, CH63 4JY, United Kingdom
Dana Farber Cancer Institute

Boston, Massachusetts, 02215, United States
Duke University Health System (DUHS)

Durham, North Carolina, 27710, United States
Erasmus Medical Center

Rotterdam, 3015 GD, Netherlands
Fondazione IRCCS Ca' Granda Ospedale Maggiore Poloclinico, UOC Ematologia

Milan, 20122, Italy
Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital

London, SE1 9RT, United Kingdom
H. Lee Moffitt Cancer Center

Tampa, Florida, 33612, United States
Herbert Irving Comprehensive Cancer Center

New York, New York, 10032, United States
Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain
Huntsman Cancer Institute

Salt Lake City, Utah, 84112, United States
Hôpital Pitié-Salpêtrière, Service de Dermatologie

Paris, 75013, France
Hôpital de la Timone, Service de dermatologie

Marseille, 13385, France
Karolinska University Hospital, Hematologimottagningen R51

Stockholm, 141 86, Sweden
Klinikum rechts der Isar, Technische Universität München

Munich, 80802, Germany
Mayo Clinic

Rochester, Minnesota, 55905, United States
Mayo Clinic Florida

Jacksonville, Florida, 32224, United States
Mayo Clinic Hospital

Phoenix, Arizona, 85054, United States
Michigan Medicine, University of Michigan

Ann Arbor, Michigan, 48106, United States
NHS Greater Glasgow and Clyde, Beatson West of Scotland Cancer Centre

Glasgow, G12 OXL, United Kingdom
Odense Universitetshospital, ORCA/Allergicentret, Hudafdeling I og Allergicenter

Odense, DK-5000, Denmark
Oslo Universitetssykehus, Rikshospitalet, Department of Hematology

Oslo, 0372, Norway
Rush University Medical Center

Chicago, Illinois, 60612, United States
St. Michael's Hospital

Toronto, Ontario, M5B 1W8, Canada
Stanford Cancer Institute

Stanford, California, 94305, United States
Tom Baker Cancer Centre

Calgary, Alberta, T2N 4N2, Canada
Uniklinik RWTH Aachen

Aachen, 52074, Germany
University Clinic Hamburg Eppendorf, University Cancer Center Hamburg (UCCH)

Hamburg, 20246, Germany
University Hospital Antwerp

Edegem, 2650, Belgium
University Hospital Basel

Basel, 4031, Switzerland
University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States
University Medical Center Groningen (UMCG)

Groningen, 9713 GZ, Netherlands
University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States
University of Alberta Hospital

Edmonton, Alberta, T6G 2B7, Canada
University of Kansas Hospital

Kansas City, Kansas, 66160, United States
University of Texas, MD Anderson Cancer Center

Houston, Texas, 77030, United States
Universitätsklinik Mainz, Universitäts-Hautklinik, Clinical Research Center

Mainz, 55131, Germany
Universitätsklinikum Schleswig-Holstein, Hämatologie/Onkologie

Lübeck, 23538, Germany
Universitätsmedizin Mannheim, III. Medizinische Klinik

Mannheim, 68167, Germany
Virginia Commonwealth University Medical Center

Richmond, Virginia, 23298, United States
Washington University School of Medicine

St Louis, Missouri, 63110, United States
Winship Cancer Institute, Emory University

Atlanta, Georgia, 30322, United States
lnstituto de Estudios de Mastocitosis de Castilla la Mancha, Hospital Virgen del Valle - Complejo Hospitalario de Toledo

Toledo, 45071, Spain

More trials for these conditions

Other studies related to the condition(s) this trial covers.