Actively Recruiting
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Led by Blueprint Medicines Corporation · Updated on 2026-04-20
534
Participants Needed
64
Research Sites
565 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Participant must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2.
- Participant has confirmed diagnosis of indolent systemic mastocytosis (ISM) confirmed by Central Pathology Review (for Parts 1, 2, K, and PK groups).
- Participant has confirmed diagnosis of smoldering systemic mastocytosis (SSM) confirmed by central pathology review of bone marrow biopsy and WHO diagnostic criteria (for Part S).
- Participant's symptoms are not adequately controlled by at least two symptom-directed therapies, including H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab (for Parts 1 and PK groups).
- Symptom-directed therapy (SDT) for ISM symptom management must be stable for at least 14 days before starting screening.
- For participants on corticosteroids, the dose must be 20 mg/day prednisone or equivalent or less and stable for at least 14 days.
You will not qualify if you...
- Participants diagnosed with cutaneous mastocytosis only, SM with associated hematologic neoplasm of non-mast cell lineage, aggressive systemic mastocytosis, mast cell leukemia, or mast cell sarcoma.
- Participants diagnosed with another myeloproliferative disorder.
- Participants with organ damage caused by systemic mastocytosis.
- Participants with clinically significant, uncontrolled cardiovascular disease.
- Participants with a QTcF interval >470 msec for females or >450 msec for males.
- Participants with a history of primary malignancy diagnosed or treated within the past 3 years, except certain skin cancers and localized prostate cancer.
- Participants who received cytoreductive or antibody therapies within specified time frames before screening.
- Participants who have had radiotherapy or PUVA therapy within 14 days before screening.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 64 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
David Geffen School of Medicine at UCLA
Los Angeles, California, United States, 90095
Actively Recruiting
3
Stanford Cancer Institute
Palo Alto, California, United States, 94305
Actively Recruiting
4
UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus
Aurora, Colorado, United States, 80045
Actively Recruiting
5
Winship Cancer Institute, Emory University
Atlanta, Georgia, United States, 30322
Actively Recruiting
6
Brigham and Women's Hospital
Boston, Massachusetts, United States, 02115
Actively Recruiting
7
Michigan Medicine University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
8
Mayo Clinic
Rochester, Minnesota, United States, 55902
Actively Recruiting
9
Roswell Park Cancer Institute
Buffalo, New York, United States, 14263
Actively Recruiting
10
Columbia University Medical Center
New York, New York, United States, 10032
Actively Recruiting
11
Duke Asthma, Allergy and Airway Center
Durham, North Carolina, United States, 27705
Actively Recruiting
12
University of Cincinnati Medical Center
Cincinnati, Ohio, United States, 45219
Actively Recruiting
13
The University of Texas, MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
14
Huntsman Cancer Institute, University of Utah
Salt Lake City, Utah, United States, 84112
Actively Recruiting
15
Consultorios Medicos Dr. Doreski - Fundacion Respirar
Buenos Aires, Argentina, 1426
Actively Recruiting
16
Princess Alexandra Hospital
Woolloongabba, Queensland, Australia
Actively Recruiting
17
The Alfred Hospital
Melbourne, Victoria, Australia, 3004
Actively Recruiting
18
Kepler Universitatsklinikum, Med Campus III. Clinic of Internal Medicine 3 - Hematology and Oncology
Linz, Austria, 4021
Actively Recruiting
19
Unitversitair Ziekenhuis Antwerpen
Edegem, Antwerpen, Belgium
Actively Recruiting
20
CHU Tivoli
La Louvière, Belgium, 7100
Actively Recruiting
21
Fakultní nemocnice Královské Vinohrady, Hematologická klinika 3. LF UK v Praze a FNKV
Prague, Czechia, 100 34
Actively Recruiting
22
CHU Amiens-Picardie
Amiens, France, 80000
Actively Recruiting
23
CHU de Caen
Caen, France
Actively Recruiting
24
CHU Grenoble
Grenoble, France, 38043
Actively Recruiting
25
CHU de Limoges
Limoges, France, 87042
Actively Recruiting
26
CHU de Nantes
Nantes, France, 44093
Actively Recruiting
27
Hôpital de la Pitié Salpétrière
Paris, France, 75013
Actively Recruiting
28
Hôpital Necker - Départementd 'HématologieA dultes
Paris, France, 75015
Actively Recruiting
29
CHU de Poitiers
Poitiers, France, 86000
Actively Recruiting
30
CHU Toulouse - Hopital Larrey
Toulouse, France
Actively Recruiting
31
Universitätsklinikum RWTH Aachen Klinik für Hämatologie, Onkologie, Hämostaseologie und Stammzelltransplantation
Aachen, Germany
Actively Recruiting
32
Charité - Universitätsmedizin Berlin Institute of Allergology
Berlin, Germany, 12203
Actively Recruiting
33
University Clinic Erlangen
Erlangen, Germany, 91054
Actively Recruiting
34
University Clinic Hamburg Eppendorf
Hamburg, Germany, 20246
Actively Recruiting
35
Universitätsmedizin Mannheim III. Medizinische Klinik Universität Heidelberg Medizinische Fakultät Mannheim
Mannheim, Germany, 68167
Actively Recruiting
36
LMU Klinikum
Munich, Germany, 80377
Actively Recruiting
37
University General Hospital - General Hospital of West Attica
Chaïdári, Greece, 12462
Actively Recruiting
38
General Hospital of Thessaloniki "G. Papanikolaou"
Thessaloniki, Greece, 57010
Actively Recruiting
39
UOC Ematologia
Milan, Lombardy, Italy, 20122
Actively Recruiting
40
SOD Ematologia (Ambulatori)- AOUC Azienda Ospedaliero Universitaria Careggi
Florence, Tuscany, Italy, 50134
Actively Recruiting
41
Unita Operativa di Ematologia AOU Policlinico S. Orsola-Malpighi
Bologna, Italy, 40138
Actively Recruiting
42
AOU Policlinico G.Rodolico - San Marco
Catania, Italy, 95123
Actively Recruiting
43
S.C. Ematologia Fondazione I.R.C.C.S. Policlinico San Matteo
Pavia, Italy, 27100
Actively Recruiting
44
S.S.D. Immunologia Clinica e Allergologia Azienda Ospedaliera Universitaria San Giovanni di Dio e Ruggi d'Aragona
Salerno, Italy, 84131
Actively Recruiting
45
Unità Operativa di Allergologia Azienda Ospedaliera Universitaria Integrata di Verona
Verona, Italy, 37126
Actively Recruiting
46
ErasmusMC
Rotterdam, South Holland, Netherlands, 3015 GD
Actively Recruiting
47
University Medical Center Groningen
Groningen, Netherlands, 9713 GZ
Actively Recruiting
48
Oslo University Hospital
Oslo, Norway, N-0424
Actively Recruiting
49
Uniwersyteckie Centrum Kliniczne Klinika Alergologii
Gdansk, Poland, 80-214
Actively Recruiting
50
Centro Hospitalar de Lisboa Central, E.P.E. - Hospital de Santo Antonio dos Capuchos
Lisbon, Portugal, 1169-050
Actively Recruiting
51
CHUPorto, EPE - Hospital de Santo António
Porto, Portugal, 4099-001
Actively Recruiting
52
Centro Hospitalar Universitario Sao Joao, E.P.E.
Porto, Portugal, 4200-139
Actively Recruiting
53
Hospital Universitario Vall d'Hebron
Barcelona, Spain
Actively Recruiting
54
Hospital Universitario Ramon y Cajal
Madrid, Spain, 28034
Actively Recruiting
55
Hospital Virgen del Valle - Instituto de Estudios de Mastocitosis de Castilla-La Mancha
Toledo, Spain, 45071
Actively Recruiting
56
Uppsala University Hospital
Uppsala, Sweden, 751 85
Actively Recruiting
57
University Hospital Basel
Basel, Switzerland, C-4031
Actively Recruiting
58
Luzerner Kantonsspital
Lucerne, Switzerland, 6000
Actively Recruiting
59
University Hospital of Wales
Cardiff, Wales, United Kingdom, CF14 4XW
Actively Recruiting
60
University Hospital of Wales
Cardiff, United Kingdom, CF14 4XW
Actively Recruiting
61
University College London Hospitals (UCLH), Haematology Cancer Clinical Trials Unit
London, United Kingdom, NW1 2PG
Actively Recruiting
62
Guy's and St Thomas's NHS Foundation Trust
London, United Kingdom, SE1 7EH
Actively Recruiting
63
Cancer and Haematology Centre
Oxford, United Kingdom, OX3 7LE
Actively Recruiting
64
University Hospital Plymouth NHS Trust
Plymouth, United Kingdom, PL6 8DH
Actively Recruiting
Research Team
B
Blueprint Medicines
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
10
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