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New targeted pill shows promise for rare blood cancer

NCT ID NCT03580655

Disease control 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 2 study tested avapritinib, a daily tablet that targets a specific genetic mutation, in 107 people with advanced systemic mastocytosis (a rare blood cancer). The main goal was to see how many patients had their tumors shrink or symptoms improve. Results suggest the drug may help control the disease and ease symptoms, but more research is needed.

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 successful, this could provide a new treatment option that shrinks tumors and improves symptoms for people with advanced systemic mastocytosis.
What could go wrong
This is a single-arm Phase 2 study with no comparison group, so results may not hold up in larger trials. The drug may cause side effects and not work for everyone.

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

107 people

The number who actually took part.

Started

Nov 2018

Finished

Dec 2024

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 a diagnosis of aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) or mast cell leukemia (MCL) based on World Health Organization diagnostic criteria. Before enrollment, the Study Steering Committee must confirm the diagnosis of AdvSM (based on Central Pathology Laboratory assessment of bone marrow). 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. Key Exclusion Criteria: 1. Patient has received prior treatment with avapritinib. 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.

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Conditions

The condition(s) this trial relates to.

aggressive systemic mastocytosis mast cell leukemia Mastocytosis, Systemic systemic mastocytosis with an associated clonal hematologic non-mast cell lineage disease

As listed by the trial registrant

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

Advanced systemic mastocytosis Aggressive systemic mastocytosis Mast cell leukemia Systemic mastocytosis with an associated hematologic neoplasm

Contacts and locations

Locations

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

    Salerno, 84131, Italy

  • Azienda Ospedaliero-Universitaria Careggi, CRIMM - Centro di Ricerca ed Innovazione per le Malattie Mieloproliferative

    Florence, 50134, Italy

  • Beatson West of Scotland Cancer Centre - NHS Greater Glasgow and Clyde

    Glasgow, G12 0YN, United Kingdom

  • CHU Toulouse - Hôpital Larrey

    Toulouse, 31059, France

  • Centro Ricerche Cliniche di Verona - Azienda Ospedaliera Universitaria Integrata di Verona

    Verona, 37134, Italy

  • Dana Farber Cancer Institute

    Boston, Massachusetts, 02215, United States

  • Guy's and St Thomas' NHS Foundation Trust - Guy's Hospital

    London, SE1 9RT, United Kingdom

  • Herbert Irving Comprehensive Cancer Center

    New York, New York, 10032, United States

  • Huntsman Cancer Institute

    Salt Lake City, Utah, 84112, United States

  • Hôpital Necker-Enfants Malades

    Paris, 75015, France

  • Klinik und Poliklinik für Innere Medizin III, Klinikum rechts der Isar der TU München

    Munich, 81675, Germany

  • Mays Cancer Center

    San Antonio, Texas, 78229, United States

  • Medizinische Universität Wien, Universitätsklinik für Innere Medizin I, Klinische Abteilung für Hämatologie und Hämostaseologie

    Vienna, 1090, Austria

  • Odense University Hospital, Department of Haematology

    Odense, DK-5000, Denmark

  • Oslo University Hospital-Rikshospitalet, Hematology

    Oslo, 0372, Norway

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14263, United States

  • 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

  • The University of Kansas Cancer Center

    Westwood, Kansas, 66205, United States

  • Uniklinik RWTH Aachen, Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltranslplantation

    Aachen, 52074, Germany

  • University Medical Center Groningen (UMCG)

    Groningen, 9713 GZ, Netherlands

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Pennsylvania, Abramson Cancer Center

    Philadelphia, Pennsylvania, 19104, United States

  • University of Texas, MD Anderson Cancer Center

    Houston, Texas, 77030, United States

  • Universitätsklinikum Hamburg-Eppendorf, Zentrum für Onkologie

    Hamburg, 20246, Germany

  • Universitätsklinikum Leipzig, Medizinische Klinik und Poliklinik I - Hämatologie und Zelltherapie, lnternistische Onkologie, Hämostaseologie

    Leipzig, 04103, Germany

  • Universitätsmedizin Mannheim III. Medizinische Klinik

    Mannheim, 68167, Germany

  • Uniwersytecki Szpital Kliniczny im. Jana Mikulicza-Radeckiego we Wrocławiu, Klinice Hematologii, Nowotworów Krwi i Transplantacji Szpiku

    Wroclaw, 50-367, Poland

  • Uniwersyteckie Centrum Kliniczne, Klinika Hematologii i Transplantologii

    Gdansk, 80-214, Poland

  • Washington University School of Medicine

    St Louis, Missouri, 63110, 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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