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New drug aims to ease symptoms of rare mast cell disease

NCT ID NCT03731260

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

251 people

The number who actually took part.

Started

Apr 2019

Expected to finish

Jun 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Key Inclusion Criteria: * 1\. Patient must have SM, confirmed by Central Pathology Review of BM biopsy, and central review of B- and C-findings by WHO diagnostic 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. Key Exclusion Criteria: * 1\. Patient has been diagnosed with any of the following WHO SM subclassifications: cutaneous mastocytosis only, smoldering SM, SM with associated hematologic neoplasm, aggressive SM, mast cell leukemia, or mast cell sarcoma. * 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.

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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

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

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