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New pill aims to tame rare mast cell disease

NCT ID NCT04910685

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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 05, 2026 · Updated 2 times

Summary

This study tests an experimental drug called elenestinib (BLU-263) in people with indolent systemic mastocytosis, a rare condition where too many mast cells cause symptoms like flushing, itching, and stomach issues. About 534 participants will receive either the drug or a placebo, plus standard symptom treatments. The goal is to see if elenestinib can better control symptoms that aren't helped by current therapies.

What this could mean

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

Active substance
Elenestinib (also called BLU-263), an oral tablet
What this could lead to
If it works, this could offer a new treatment option for people with indolent systemic mastocytosis whose symptoms are not controlled by current therapies.
What could go wrong
This is an early-to-mid-stage trial, so results are uncertain. The drug may cause side effects or fail to improve symptoms better than placebo.

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 2/3

Runs two stages together: whether the treatment works, then large-scale confirmation.

Participants

About 534 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2021

Expected to finish

Sep 2032

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: All Participants: -Participant must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2. Part 1 and PK groups: * Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review * Participant must have failed to achieve adequate symptom control for 1 or more Baseline symptoms, as determined by the Investigator, with at least 2 of the following symptom-directed therapies administered: H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab. * Participants must have SDT for ISM symptom management stabilized for at least 14 days prior to starting screening procedures. * For participants receiving corticosteroids, the dose must be ≤ 20 mg/day prednisone or equivalent, and the dose must be stable for ≥ 14 days. Part K: -Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review Part S: -Participant has confirmed diagnosis of SSM, confirmed by Central Pathology Review of BM biopsy and central review of B- and C-findings by WHO diagnostic criteria. Part 2: -Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review Key Exclusion Criteria: * Participant has been diagnosed with any of the following WHO systemic mastocytosis (SM) sub-classifications: cutaneous mastocytosis only, SM with an associated hematologic neoplasm of non-MC lineage (SM-AHN), aggressive SM, mast cell leukemia, or mast cell sarcoma. * Participant has been diagnosed with another myeloproliferative disorder. * Participant has organ damage attributable to SM. * Participant has clinically significant, uncontrolled, cardiovascular disease * Participant has a QT interval corrected using Fridericia's formula (QTcF) \> \> 470 milliseconds (msec) (for females) or \> 450 msec (for males). * Participant has a history of a primary malignancy that has been diagnosed or required therapy within 3 years. The following prior malignancies are not exclusionary: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site. * Time since any cytoreductive therapy including masitinib and midostaurin should be at least 5 half-lives or 14 days (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 screening period. * Participant has received radiotherapy or psoralen and ultraviolet A (PUVA) therapy \< 14 days before beginning the screening period. Other protocol-defined criteria apply.

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Conditions

The condition(s) this trial relates to.

indolent systemic mastocytosis Mastocytosis, Systemic Smouldering systemic mastocytosis

As listed by the trial registrant

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

Indolent systemic mastocytosis Smoldering systemic mastocytosis

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    75 sites in 22 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • AOU Policlinico G.Rodolico - San Marco

    RECRUITING

    Catania, 95123, Italy

  • Ankara Universitesi Tip Fakultesi Cebeci Hastanesi

    RECRUITING

    Ankara, 06620, Turkey (Türkiye)

  • Brigham and Women's Hospital

    RECRUITING

    Boston, Massachusetts, 02115, United States

  • CHP Centro Hematologico Pavlovsky

    RECRUITING

    Buenos Aires, 1113, Argentina

  • CHU Amiens-Picardie

    RECRUITING

    Amiens, 80000, France

  • CHU Grenoble

    RECRUITING

    Grenoble, 38043, France

  • CHU Tivoli

    RECRUITING

    La Louvière, 7100, Belgium

  • CHU Toulouse - Hopital Larrey

    RECRUITING

    Toulouse, France

  • CHU de Caen

    RECRUITING

    Caen, France

  • CHU de Limoges

    RECRUITING

    Limoges, 87042, France

  • CHU de Nantes

    RECRUITING

    Nantes, 44093, France

  • CHU de Poitiers

    RECRUITING

    Poitiers, 86000, France

  • CHUPorto, EPE - Hospital de Santo António

    RECRUITING

    Porto, 4099-001, Portugal

  • Cancer and Haematology Centre

    RECRUITING

    Oxford, OX3 7LE, United Kingdom

  • Centro Hospitalar Universitario Sao Joao, E.P.E.

    RECRUITING

    Porto, 4200-139, Portugal

  • Centro Hospitalar de Lisboa Central, E.P.E. - Hospital de Santo Antonio dos Capuchos

    RECRUITING

    Lisbon, 1169-050, Portugal

  • Charité - Universitätsmedizin Berlin Institute of Allergology

    RECRUITING

    Berlin, 12203, Germany

  • Columbia University Medical Center

    RECRUITING

    New York, New York, 10032, United States

  • Consultorios Medicos Dr. Doreski - Fundacion Respirar

    RECRUITING

    Buenos Aires, 1426, Argentina

  • Cork University Hospital

    RECRUITING

    Cork, T12 DC4A, Ireland

  • David Geffen School of Medicine at UCLA

    RECRUITING

    Los Angeles, California, 90095, United States

  • Duke Asthma, Allergy and Airway Center

    RECRUITING

    Durham, North Carolina, 27705, United States

  • ErasmusMC

    RECRUITING

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Fakultní nemocnice Brno, Interní hematologická a onkologická klinika

    RECRUITING

    Brno, 625 00, Czechia

  • Fakultní nemocnice Královské Vinohrady, Hematologická klinika 3. LF UK v Praze a FNKV

    RECRUITING

    Prague, 100 34, Czechia

  • Fundación Valle del Lili

    RECRUITING

    Cali, 760033, Colombia

  • General Hospital of Thessaloniki "G. Papanikolaou"

    RECRUITING

    Thessaloniki, 57010, Greece

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

    RECRUITING

    London, SE1 7EH, United Kingdom

  • Hospital Universitario Ramon y Cajal

    RECRUITING

    Madrid, 28034, Spain

  • Hospital Universitario Vall d'Hebron

    RECRUITING

    Barcelona, Spain

  • Hospital Virgen del Valle - Instituto de Estudios de Mastocitosis de Castilla-La Mancha

    RECRUITING

    Toledo, 45071, Spain

  • Hospital das Clínicas da Faculdade de Medicina da USP - HCFMUSP

    RECRUITING

    São Paulo, 05403-010, Brazil

  • Huntsman Cancer Institute, University of Utah

    RECRUITING

    Salt Lake City, Utah, 84112, United States

  • Hôpital Necker - Départementd 'HématologieA dultes

    RECRUITING

    Paris, 75015, France

  • Hôpital de la Pitié Salpétrière

    RECRUITING

    Paris, 75013, France

  • Kepler Universitatsklinikum, Med Campus III. Clinic of Internal Medicine 3 - Hematology and Oncology

    RECRUITING

    Linz, 4021, Austria

  • LMU Klinikum

    RECRUITING

    Munich, 80377, Germany

  • Luzerner Kantonsspital

    RECRUITING

    Lucerne, 6000, Switzerland

  • Maastricht UMC+

    RECRUITING

    Maastricht, 6229 HX, Netherlands

  • Mayo Clinic

    RECRUITING

    Rochester, Minnesota, 55902, United States

  • Mersin VM Medikal Park Hastanesi

    RECRUITING

    Mersin, 33200, Turkey (Türkiye)

  • Michigan Medicine University of Michigan

    RECRUITING

    Ann Arbor, Michigan, 48109, United States

  • Oslo University Hospital

    RECRUITING

    Oslo, N-0424, Norway

  • Princess Alexandra Hospital

    RECRUITING

    Woolloongabba, Queensland, Australia

  • Roswell Park Cancer Institute

    RECRUITING

    Buffalo, New York, 14263, United States

  • S.C. Ematologia Fondazione I.R.C.C.S. Policlinico San Matteo

    RECRUITING

    Pavia, 27100, Italy

  • S.S.D. Immunologia Clinica e Allergologia Azienda Ospedaliera Universitaria San Giovanni di Dio e Ruggi d'Aragona

    RECRUITING

    Salerno, 84131, Italy

  • SOD Ematologia (Ambulatori)- AOUC Azienda Ospedaliero Universitaria Careggi

    RECRUITING

    Florence, Tuscany, 50134, Italy

  • Samodzielny Publiczny Za kład Opieki Zdrowotnej Szpital Uniwersytecki w Krakowie

    RECRUITING

    Krakow, 30-688, Poland

  • Stanford Cancer Institute

    RECRUITING

    Palo Alto, California, 94305, United States

  • The Alfred Hospital

    RECRUITING

    Melbourne, Victoria, 3004, Australia

  • The University of Texas, MD Anderson Cancer Center

    RECRUITING

    Houston, Texas, 77030, United States

  • UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus

    RECRUITING

    Aurora, Colorado, 80045, United States

  • UOC Ematologia

    RECRUITING

    Milan, Lombardy, 20122, Italy

  • Unita Operativa di Ematologia AOU Policlinico S. Orsola-Malpighi

    RECRUITING

    Bologna, 40138, Italy

  • Unitversitair Ziekenhuis Antwerpen

    RECRUITING

    Edegem, Antwerpen, Belgium

  • Unità Operativa di Allergologia Azienda Ospedaliera Universitaria Integrata di Verona

    RECRUITING

    Verona, 37126, Italy

  • Universitair Ziekenhuis Gent

    RECRUITING

    Ghent, 9000, Belgium

  • University Clinic Erlangen

    RECRUITING

    Erlangen, 91054, Germany

  • University Clinic Hamburg Eppendorf

    RECRUITING

    Hamburg, 20246, Germany

  • University College London Hospitals (UCLH), Haematology Cancer Clinical Trials Unit

    RECRUITING

    London, NW1 2PG, United Kingdom

  • University General Hospital - General Hospital of West Attica

    RECRUITING

    Chaïdári, 12462, Greece

  • University Hospital Basel

    RECRUITING

    Basel, C-4031, Switzerland

  • University Hospital Plymouth NHS Trust

    RECRUITING

    Plymouth, PL6 8DH, United Kingdom

  • University Hospital of Wales

    RECRUITING

    Cardiff, Wales, CF14 4XW, United Kingdom

  • University Hospital of Wales

    RECRUITING

    Cardiff, CF14 4XW, United Kingdom

  • University Medical Center Groningen

    RECRUITING

    Groningen, 9713 GZ, Netherlands

  • University of Alabama at Birmingham

    RECRUITING

    Birmingham, Alabama, 35294, United States

  • University of Cincinnati Medical Center

    RECRUITING

    Cincinnati, Ohio, 45219, United States

  • Universitätsklinikum RWTH Aachen Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltransplantation

    RECRUITING

    Aachen, Germany

  • Universitätsmedizin Mannheim III. Medizinische Klinik Universität Heidelberg Medizinische Fakultät Mannheim

    RECRUITING

    Mannheim, 68167, Germany

  • Uniwersyteckie Centrum Kliniczne Klinika Alergologii

    RECRUITING

    Gdansk, 80-214, Poland

  • Uppsala University Hospital

    RECRUITING

    Uppsala, 751 85, Sweden

  • Winship Cancer Institute, Emory University

    RECRUITING

    Atlanta, Georgia, 30322, United States

  • lstanbul Universitesi lstanbul Tip Fakultesi Hastanesi

    RECRUITING

    Istanbul, 34093, Turkey (Türkiye)

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