New pill aims to tame rare mast cell disease

NCT ID NCT04910685

Recruiting now Disease control Sponsor: Blueprint Medicines Corporation Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

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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

Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

AOU Policlinico G.Rodolico - San Marco
RECRUITING

Catania, 95123, Italy
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
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
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
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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