(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis
NCT ID: NCT04910685
Last Updated: 2026-01-30
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2/PHASE3
534 participants
INTERVENTIONAL
2021-11-30
2032-09-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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(Part 1) Placebo + SDT
Participants will receive SDT and matching placebo. SDT will be determined on a per participant basis. Placebo will be administered orally, once daily until completion of Part 1.
Placebo
Placebo oral tablet
(Part 2) Elenestinib Dose 1 + SDT
Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for approximately 48 weeks.
Elenestinib
Elenestinib oral tablet
(Part 2) Placebo + SDT
Participants will receive SDT and matching placebo. SDT will be determined on a per participant basis. Placebo will be administered orally, once daily for approximately 48 weeks.
Placebo
Placebo oral tablet
(Part 3) Elenestinib + SDT
Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
Elenestinib
Elenestinib oral tablet
(Part S) Elenestinib Dose 1 + SDT
Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
Elenestinib
Elenestinib oral tablet
(Part K) Elenestinib Dose 1 + SDT
Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
Elenestinib
Elenestinib oral tablet
(PK groups) Elenestinib + SDT
Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily for up to approximately 5 years.
Elenestinib
Elenestinib oral tablet
(Part 1) Elenestinib Dose 1 + SDT
Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1.
Elenestinib
Elenestinib oral tablet
(Part 1) Elenestinib Dose 2 + SDT
Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1.
Elenestinib
Elenestinib oral tablet
(Part 1) Elenestinib Dose 3 + SDT
Participants will receive SDT and elenestinib. SDT will be determined on a per participant basis. Elenestinib will be administered orally, once daily until completion of Part 1.
Elenestinib
Elenestinib oral tablet
Interventions
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Elenestinib
Elenestinib oral tablet
Placebo
Placebo oral tablet
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
-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
Exclusion Criteria
* 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.
18 Years
ALL
No
Sponsors
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Blueprint Medicines Corporation
INDUSTRY
Responsible Party
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Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Stanford Cancer Institute
Palo Alto, California, United States
UCHealth Blood Disorders and Cell Therapies Center - Anschutz Medical Campus
Aurora, Colorado, 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
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
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 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
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
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
Countries
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Central Contacts
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Other Identifiers
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BLU-263-1201
Identifier Type: -
Identifier Source: org_study_id
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