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RECRUITING

NCT04910685

PHASE2/PHASE3

(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis

Sponsor: Blueprint Medicines Corporation

View on ClinicalTrials.gov

Summary

This is a randomized, double-blind, placebo-controlled, Phase 2/3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by SDT. Parts 1 and 2 will enroll participants with ISM. Participants enrolled in Part 2 will roll over onto Part 3 to receive treatment with elenestinib in an open-label fashion following completion of the earlier Part. Part K will enroll participants with ISM who have previously received an approved selective KIT inhibitor. The study also includes pharmacokinetic (PK) groups that will enroll participants with ISM.

Official title: A Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study of BLU-263 in Indolent Systemic Mastocytosis

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

534

Start Date

2021-11-30

Completion Date

2032-09-30

Last Updated

2026-04-06

Healthy Volunteers

Conditions

Indolent Systemic Mastocytosis Smoldering Systemic Mastocytosis

Interventions

DRUG

Elenestinib

Elenestinib oral tablet

DRUG

Placebo

Placebo oral tablet

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.

Locations (63)

University of Alabama at Birmingham

Birmingham, Alabama, United States

David Geffen School of Medicine at UCLA

Los Angeles, California, United States

Stanford Cancer Institute

Palo Alto, California, United States

UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus

Aurora, Colorado, United States

Winship Cancer Institute, Emory University

Atlanta, Georgia, United States

Brigham and Women's Hospital

Boston, Massachusetts, United States

Michigan Medicine University of Michigan

Ann Arbor, Michigan, United States

Mayo Clinic

Rochester, Minnesota, United States

Roswell Park Cancer Institute

Buffalo, New York, United States

Columbia University Medical Center

New York, New York, United States

Duke Asthma, Allergy and Airway Center

Durham, North Carolina, United States

University of Cincinnati Medical Center

Cincinnati, Ohio, United States

The University of Texas, MD Anderson Cancer Center

Houston, Texas, United States

Huntsman Cancer Institute, University of Utah

Salt Lake City, Utah, United States

Consultorios Medicos Dr. Doreski - Fundacion Respirar

Buenos Aires, Argentina

Princess Alexandra Hospital

Woolloongabba, Queensland, Australia

The Alfred Hospital

Melbourne, Victoria, Australia

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

Linz, Austria

Unitversitair Ziekenhuis Antwerpen

Edegem, Antwerpen, Belgium

CHU Tivoli

La Louvière, Belgium

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

Prague, Czechia

CHU Amiens-Picardie

Amiens, France

CHU de Caen

Caen, France

CHU Grenoble

Grenoble, France

CHU de Limoges

Limoges, France

CHU de Nantes

Nantes, France

Hôpital de la Pitié Salpétrière

Paris, France

Hôpital Necker - Départementd 'HématologieA dultes

Paris, France

CHU de Poitiers

Poitiers, France

CHU Toulouse - Hopital Larrey

Toulouse, France

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

Aachen, Germany

Charité - Universitätsmedizin Berlin Institute of Allergology

Berlin, Germany

University Clinic Erlangen

Erlangen, Germany

University Clinic Hamburg Eppendorf

Hamburg, Germany

Universitätsmedizin Mannheim III. Medizinische Klinik Universität Heidelberg Medizinische Fakultät Mannheim

Mannheim, Germany

LMU Klinikum

Munich, Germany

University General Hospital - General Hospital of West Attica

Chaïdári, Greece

General Hospital of Thessaloniki "G. Papanikolaou"

Thessaloniki, Greece

UOC Ematologia

Milan, Lombardy, Italy

SOD Ematologia (Ambulatori)- AOUC Azienda Ospedaliero Universitaria Careggi

Florence, Tuscany, Italy

Unita Operativa di Ematologia AOU Policlinico S. Orsola-Malpighi

Bologna, Italy

AOU Policlinico G.Rodolico - San Marco

Catania, Italy

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

Pavia, Italy

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

Salerno, Italy

Unità Operativa di Allergologia Azienda Ospedaliera Universitaria Integrata di Verona

Verona, Italy

ErasmusMC

Rotterdam, South Holland, Netherlands

University Medical Center Groningen

Groningen, Netherlands

Oslo University Hospital

Oslo, Norway

Uniwersyteckie Centrum Kliniczne Klinika Alergologii

Gdansk, Poland

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

Lisbon, Portugal

CHUPorto, EPE - Hospital de Santo António

Porto, Portugal

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

Porto, Portugal

Hospital Universitario Vall d'Hebron

Barcelona, Spain

Hospital Universitario Ramon y Cajal

Madrid, Spain

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

Toledo, Spain

Uppsala University Hospital

Uppsala, Sweden

University Hospital Basel

Basel, Switzerland

Luzerner Kantonsspital

Lucerne, Switzerland

University Hospital of Wales

Cardiff, Wales, United Kingdom

University Hospital of Wales

Cardiff, United Kingdom

Guy's and St Thomas's NHS Foundation Trust

London, United Kingdom

Cancer and Haematology Centre

Oxford, United Kingdom

University Hospital Plymouth NHS Trust

Plymouth, United Kingdom