(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Start Date
11/30/2021
Completion Date
9/30/2032
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.
Eligibility Criteria
Age Range: 18 years to No maximum
Interventions
Elenestinib
Placebo
Conditions
Locations
University of Alabama at Birmingham
Birmingham, Alabama 35294
United States
David Geffen School of Medicine at UCLA
Los Angeles, California 90095
United States
Stanford Cancer Institute
Palo Alto, California 94305
United States
UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus
Aurora, Colorado 80045
United States
Winship Cancer Institute, Emory University
Atlanta, Georgia 30322
United States
Brigham and Women's Hospital
Boston, Massachusetts 02115
United States
Michigan Medicine University of Michigan
Ann Arbor, Michigan 48109
United States
Mayo Clinic
Rochester, Minnesota 55902
United States
Roswell Park Cancer Institute
Buffalo, New York 14263
United States
Columbia University Medical Center
New York, New York 10032
United States
Duke Asthma, Allergy and Airway Center
Durham, North Carolina 27705
United States
University of Cincinnati Medical Center
Cincinnati, Ohio 45219
United States
The University of Texas, MD Anderson Cancer Center
Houston, Texas 77030
United States
Huntsman Cancer Institute, University of Utah
Salt Lake City, Utah 84112
United States
CHP Centro Hematologico Pavlovsky
Buenos Aires, 1113
Argentina
Consultorios Medicos Dr. Doreski - Fundacion Respirar
Buenos Aires, 1426
Argentina
Princess Alexandra Hospital
Woolloongabba, Queensland
Australia
The Alfred Hospital
Melbourne, Victoria 3004
Australia
Kepler Universitatsklinikum, Med Campus III. Clinic of Internal Medicine 3 - Hematology and Oncology
Linz, 4021
Austria
Unitversitair Ziekenhuis Antwerpen
Edegem, Antwerpen
Belgium
Universitair Ziekenhuis Gent
Ghent, 9000
Belgium
CHU Tivoli
La Louvière, 7100
Belgium
Hospital das Clínicas da Faculdade de Medicina da USP - HCFMUSP
São Paulo, 05403-010
Brazil
Fakultní nemocnice Brno, Interní hematologická a onkologická klinika
Brno, 625 00
Czechia
Fakultní nemocnice Královské Vinohrady, Hematologická klinika 3. LF UK v Praze a FNKV
Prague, 100 34
Czechia
CHU Amiens-Picardie
Amiens, 80000
France
CHU de Caen
Caen,
France
CHU Grenoble
Grenoble, 38043
France
CHU de Limoges
Limoges, 87042
France
CHU de Nantes
Nantes, 44093
France
Hôpital de la Pitié Salpétrière
Paris, 75013
France
Hôpital Necker - Départementd 'HématologieA dultes
Paris, 75015
France
CHU de Poitiers
Poitiers, 86000
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, 12203
Germany
University Clinic Erlangen
Erlangen, 91054
Germany
University Clinic Hamburg Eppendorf
Hamburg, 20246
Germany
Universitätsmedizin Mannheim III. Medizinische Klinik Universität Heidelberg Medizinische Fakultät Mannheim
Mannheim, 68167
Germany
LMU Klinikum
Munich, 80377
Germany
University General Hospital - General Hospital of West Attica
Chaïdári, 12462
Greece
General Hospital of Thessaloniki "G. Papanikolaou"
Thessaloniki, 57010
Greece
Cork University Hospital
Cork, T12 DC4A
Ireland
UOC Ematologia
Milan, Lombardy 20122
Italy
SOD Ematologia (Ambulatori)- AOUC Azienda Ospedaliero Universitaria Careggi
Florence, Tuscany 50134
Italy
Unita Operativa di Ematologia AOU Policlinico S. Orsola-Malpighi
Bologna, 40138
Italy
AOU Policlinico G.Rodolico - San Marco
Catania, 95123
Italy
S.C. Ematologia Fondazione I.R.C.C.S. Policlinico San Matteo
Pavia, 27100
Italy
S.S.D. Immunologia Clinica e Allergologia Azienda Ospedaliera Universitaria San Giovanni di Dio e Ruggi d'Aragona
Salerno, 84131
Italy
Unità Operativa di Allergologia Azienda Ospedaliera Universitaria Integrata di Verona
Verona, 37126
Italy
ErasmusMC
Rotterdam, South Holland 3015 GD
Netherlands
University Medical Center Groningen
Groningen, 9713 GZ
Netherlands
Oslo University Hospital
Oslo, N-0424
Norway
Uniwersyteckie Centrum Kliniczne Klinika Alergologii
Gdansk, 80-214
Poland
Centro Hospitalar de Lisboa Central, E.P.E. - Hospital de Santo Antonio dos Capuchos
Lisbon, 1169-050
Portugal
CHUPorto, EPE - Hospital de Santo António
Porto, 4099-001
Portugal
Centro Hospitalar Universitario Sao Joao, E.P.E.
Porto, 4200-139
Portugal
Hospital Universitario Vall d'Hebron
Barcelona,
Spain
Hospital Universitario Ramon y Cajal
Madrid, 28034
Spain
Hospital Virgen del Valle - Instituto de Estudios de Mastocitosis de Castilla-La Mancha
Toledo, 45071
Spain
Uppsala University Hospital
Uppsala, 751 85
Sweden
University Hospital Basel
Basel, C-4031
Switzerland
Luzerner Kantonsspital
Lucerne, 6000
Switzerland
lstanbul Universitesi lstanbul Tip Fakultesi Hastanesi
Istanbul, 34093
Turkey (Türkiye)
Mersin VM Medikal Park Hastanesi
Mersin, 33200
Turkey (Türkiye)
University Hospital of Wales
Cardiff, Wales CF14 4XW
United Kingdom
University Hospital of Wales
Cardiff, CF14 4XW
United Kingdom
University College London Hospitals (UCLH), Haematology Cancer Clinical Trials Unit
London, NW1 2PG
United Kingdom
Guy's and St Thomas's NHS Foundation Trust
London, SE1 7EH
United Kingdom
Cancer and Haematology Centre
Oxford, OX3 7LE
United Kingdom
University Hospital Plymouth NHS Trust
Plymouth, PL6 8DH
United Kingdom