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ClinicalTrials.gov public records Last synced Oct 1, 2026, 2:27 PM EDT

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Showing 1–24 of 28 matching trials from the live ClinicalTrials.gov search.
Filters: Sponsor: Blueprint Medicines Corporation Clear all
Not listed No phase listed Observational

External Control, Observational, Retrospective Study Comparing Pralsetinib to Best Available Therapy in Patients With RET-Fusion Positive NSCLC

NCT04697446
View directory record Official record
Conditions
RET-fusion Non Small Cell Lung Cancer, Lung Neoplasm, Carcinoma, Non-Small-Cell Lung, Respiratory Tract Neoplasms, Thoracic Neoplasms, Neoplasms by Site, Neoplasms, Lung Diseases, Respiratory Tract Diseases, Carcinoma, Bronchogenic, Bronchial Diseases, Head and Neck Neoplasms, Carcinoma, Neoplasms by Histologic Type, Neoplasms, Germ Cell and Embryonal, Neoplasms, Nerve Tissue, Metastatic Non Small Cell Lung Cancer
Interventions
Not listed
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
279 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 10, 2021 · Synced Oct 1, 2026, 2:27 PM EDT
Completed No phase listed Observational

Patient-Reported Outcome Questionnaire for Systemic Mastocytosis

NCT02380222
View directory record Official record
Conditions
Aggressive Systemic Mastocytosis (ASM), SM w Assoc Clonal Hema Non-mast Cell Lineage Disease (SM-AHNMD), Mast Cell Leukemia (MCL), Smoldering Systemic Mastocytosis (SSM), Indolent Systemic Mastocytosis (ISM) ISM Subgroup Fully Recruited
Interventions
Interview
Other
Lead sponsor
Adelphi Values LLC
Industry
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 10, 2017 · Synced Oct 1, 2026, 2:27 PM EDT
Terminated Phase 1 Interventional

Study of Elenestinib (BLU-263) in Advanced Systemic Mastocytosis (AdvSM) and and Other KIT Altered Hematologic Malignancies

NCT05609942
View directory record Official record
Conditions
Advanced Systemic Mastocytosis
Interventions
BLU-263, Azacitidine
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
4
States / cities
Palo Alto, California • Boston, Massachusetts • Ann Arbor, Michigan + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 10, 2025 · Synced Oct 1, 2026, 2:27 PM EDT
Completed Phase 1 Interventional

A Drug-drug Interaction Study of Avapritinib and Midazolam

NCT04908176
View directory record Official record
Conditions
Gastrointestinal Stromal Tumors, GIST, Non-resectable Advanced Solid Tumors, Recurrent or Unresectable Central Nervous System (CNS) Tumors
Interventions
Avapritinib, midazolam
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
3
States / cities
Jacksonville, Florida • Ann Arbor, Michigan • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 13, 2024 · Synced Oct 1, 2026, 2:27 PM EDT
Not listed No phase listed Observational

Project PEER: Understanding the Lung Cancer Patient ExperiEnce in the Real-World Setting

NCT05826756
View directory record Official record
Conditions
Lung Cancer Patients, Caregivers
Interventions
There are no interventions in this study. All participants complete online surveys.
Other
Lead sponsor
LUNGevity Foundation
Other
Eligibility
18 Years and older
Enrollment
1,300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 28, 2023 · Synced Oct 1, 2026, 2:27 PM EDT
Terminated Phase 1 Interventional

(SYMPHONY) Phase 1/2 Study Targeting EGFR Resistance Mechanisms in NSCLC

NCT04862780
View directory record Official record
Conditions
Lung Neoplasms, Carcinoma, Non-Small-Cell Lung, Respiratory Tract Neoplasms, Neoplasms, Neoplasms by Site, Lung Diseases, Respiratory Tract Disease, Carcinoma, Bronchogenic, Bronchial Neoplasms, Adenocarcinoma, Carcinoma, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, EGFR T790M, EGFR C797S, EGFR L858R, EGFR Gene Mutation, EGF-R Positive Non-Small Cell Lung Cancer, EGFR Exon 19 Deletion, EGFR Mutation Resulting in Tyrosine Kinase Inhibitor Resistance, EGFR Activating Mutation, Protein Kinase Inhibitors, Antineoplastic Agents, Thoracic Neoplasms
Interventions
BLU-945, osimertinib
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
177 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
10
States / cities
La Jolla, California • Los Angeles, California • Orange, California + 5 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2025 · Synced Oct 1, 2026, 2:27 PM EDT
Completed Phase 1 Interventional

(EXPLORER) Study of BLU-285 in Patients With Advanced Systemic Mastocytosis (AdvSM) and Relapsed or Refractory Myeloid Malignancies

NCT02561988
View directory record Official record
Conditions
Aggressive Systemic Mastocytosis, Systemic Mastocytosis-associated Hematologic Non-mast Cell Disease, Mast Cell Leukemia, Relapsed or Refractory Myeloid Malignancies
Interventions
Avapritinib
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
9
States / cities
Stanford, California • Denver, Colorado • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Mar 17, 2023 · Synced Oct 1, 2026, 2:27 PM EDT
Completed No phase listed Observational

Mast Cell Connect: A Registry for Patients With Mastocytosis

NCT02620254
View directory record Official record
Conditions
Mastocytosis
Interventions
Not listed
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
Not listed
Enrollment
743 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
1
States / cities
Cambridge, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 4, 2021 · Synced Oct 1, 2026, 2:27 PM EDT
Recruiting Phase 2Phase 3 Interventional

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

NCT04910685
View directory record Official record
Conditions
Indolent Systemic Mastocytosis, Smoldering Systemic Mastocytosis
Interventions
Elenestinib, Placebo
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
534 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2032
U.S. locations
14
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 1, 2026, 2:27 PM EDT
Recruiting Phase 1 Interventional

Avapritinib With Decitabine in Patients With SM-AHN

NCT06327685
View directory record Official record
Conditions
Systemic Mastocytosis With an Associated Hematologic Neoplasm
Interventions
Avapritinib, Decitabine, Decitabine/Cedazuridine
Drug · Combination Product
Lead sponsor
H. Lee Moffitt Cancer Center and Research Institute
Other
Eligibility
18 Years and older
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
7
States / cities
Phoenix, Arizona • Palo Alto, California • Tampa, Florida + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced Oct 1, 2026, 2:27 PM EDT
Recruiting No phase listed Observational

An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)

NCT07264959
View directory record Official record
Conditions
Indolent Systemic Mastocytosis
Interventions
Not listed
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
7
States / cities
Aurora, Colorado • Normal, Illinois • Wheaton, Maryland + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 4, 2026 · Synced Oct 1, 2026, 2:27 PM EDT
Completed Phase 1Phase 2 Interventional

A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling

NCT04773782
View directory record Official record
Conditions
Solid Tumor, Unspecified, Child, Relapsed Solid Neoplasm, CNS Tumor
Interventions
avapritinib
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
2 Years to 17 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
15
States / cities
Phoenix, Arizona • San Francisco, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 1, 2026, 2:27 PM EDT
Recruiting Phase 1Phase 2 Interventional

A First-in-Human Trial of BLU-924 (SAR449336) in Advanced Solid Tumors Harboring KRAS Mutations

NCT07629960
View directory record Official record
Conditions
Advanced Solid Tumor, Non-Small Cell Lung Cancer, Colorectal Neoplasms, Pancreatic Ductal Adenocarcinoma
Interventions
BLU-924
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
265 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
10
States / cities
Sarasota, Florida • Tampa, Florida • Baton Rouge, Louisiana + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 1, 2026, 2:27 PM EDT
Active, not recruiting Phase 2 Interventional

(PIONEER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, Versus Placebo in Patients With Indolent Systemic Mastocytosis

NCT03731260
View directory record Official record
Conditions
Indolent Systemic Mastocytosis
Interventions
Avapritinib, Placebo
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
251 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
19
States / cities
Birmingham, Alabama • Phoenix, Arizona • Stanford, California + 15 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2025 · Synced Oct 1, 2026, 2:27 PM EDT
Recruiting No phase listed Observational

Screening Study for KIT D816V Mutated Mast Cell Disease in Select Populations

NCT07143669
View directory record Official record
Conditions
Clonal Mast Cell Disease, KIT D816V Mutation, Suspected KITD816V Mutated Clonal Mast Cell Disease
Interventions
Screening
Other
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
750 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
22
States / cities
Birmingham, Alabama • San Diego, California • Walnut Creek, California + 18 more
Source: ClinicalTrials.gov public record
Updated Aug 12, 2026 · Synced Oct 1, 2026, 2:27 PM EDT
Terminated Phase 1 Interventional

(Concerto) Study of BLU-451 in Advanced Cancers With EGFR Exon 20 Insertion Mutations

NCT05241873
View directory record Official record
Conditions
Lung Neoplasm Malignant, Carcinoma, Non-Small-Cell Lung, Respiratory Tract Neoplasms, Neoplasms, Neoplasms by Site, Lung Diseases, Respiratory Tract Disease, Carcinoma, Bronchogenic, Bronchial Neoplasms, Adenocarcinoma, Carcinoma, Neoplasms by Histologic Type, EGFR Exon 20 Mutation, EGFR Exon 20 Insertion Mutation, EGFR Activating Mutation, Antineoplastic Agents, Metastatic Lung Cancer, Brain Metastases, EGFR-mutated NSCLC, EGFR Atypical Mutations, Including G719X and L861Q
Interventions
BLU-451, Carboplatin, Pemetrexed
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
103 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
13
States / cities
Duarte, California • Los Angeles, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2025 · Synced Oct 1, 2026, 2:27 PM EDT
Completed No phase listed Observational

Retrospective Study Assessing the Effect of Avapritinib Versus Best Available Therapy in Patients With AdvSM

NCT04695431
View directory record Official record
Conditions
Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis, Systemic Mastocytosis With an Associated Hematological Neoplasm, Mast Cell Leukemia
Interventions
Not listed
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
317 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
2
States / cities
Palo Alto, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 10, 2022 · Synced Oct 1, 2026, 2:27 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study of Elenestinib in Healthy Adult Female Participants

NCT07388511
View directory record Official record
Conditions
Healthy Participants
Interventions
Elenestinib, Midazolam, Levonorgestrel/Ethinyl Estradiol
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years to 65 Years · Female only
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Tempe, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 20, 2026 · Synced Oct 1, 2026, 2:27 PM EDT
Terminated Phase 1 Interventional

(VELA) Study of BLU-222 in Advanced Solid Tumors

NCT05252416
View directory record Official record
Conditions
Advanced Solid Tumors, HR+ Breast Cancer, CCNE1 Amplification, HER2-negative Breast Cancer, Ovarian Cancer, Endometrial Cancer, Gastric Cancer, Esophageal Adenocarcinoma, Carcinosarcoma
Interventions
BLU-222, Carboplatin, Ribociclib, Fulvestrant
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
19
States / cities
Little Rock, Arkansas • San Francisco, California • Stanford, California + 13 more
Source: ClinicalTrials.gov public record
Updated Nov 28, 2025 · Synced Oct 1, 2026, 2:27 PM EDT
Completed Phase 3 Interventional Results available

(VOYAGER) Study of Avapritinib vs Regorafenib in Patients With Locally Advanced Unresectable or Metastatic GIST

NCT03465722
View directory record Official record
Conditions
GIST
Interventions
avapritinib, regorafenib
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
476 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
30
States / cities
Phoenix, Arizona • Santa Monica, California • Aurora, Colorado + 26 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2022 · Synced Oct 1, 2026, 2:27 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety, Tolerability, Pharmacokinetics, and Food Effect of BLU-782 in Healthy Adults

NCT03858075
View directory record Official record
Conditions
Healthy Volunteers
Interventions
BLU-782, Placebo, BLU-782 taken with food
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
19 Years to 65 Years
Enrollment
108 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019
U.S. locations
1
States / cities
Lincoln, Nebraska
Source: ClinicalTrials.gov public record
Updated Nov 8, 2019 · Synced Oct 1, 2026, 2:27 PM EDT
Terminated Phase 1 Interventional

(HARMONY) Study of BLU-701 in EGFR-mutant NSCLC

NCT05153408
View directory record Official record
Conditions
Lung Neoplasm, Carcinoma, Non-Small-Cell Lung, Respiratory Tract Neoplasms, Neoplasms, Neoplasms by Site, Lung Diseases, Respiratory Tract Diseases, Carcinoma, Bronchogenic, Bronchial Neoplasms, Adenocarcinoma, Carcinoma, Neoplasms by Histologic Type, Neoplasms, Nerve Tissue, EGFR C797S, EGFR C797A, EGFR L858R, EGFR Exon 19 Deletion, EGFR Gene Mutation, EGF-R Positive Non-Small Cell Lung Cancer, EGFR Mutation Resulting in Tyrosine Kinase Inhibitor Resistance, EGFR Activating Mutation, Thoracic Neoplasms, Antineoplastic Agents, Protein Kinase Inhibitors, EGFR C797G, EGFR C797X, Non Small Cell Lung Cancer
Interventions
BLU-701, osimertinib, carboplatin, pemetrexed
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
7
States / cities
Boston, Massachusetts • Detroit, Michigan • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 18, 2023 · Synced Oct 1, 2026, 2:27 PM EDT
Completed Phase 1 Interventional Results available

(NAVIGATOR) Study of BLU-285 in Patients With Gastrointestinal Stromal Tumors (GIST) and Other Relapsed and Refractory Solid Tumors

NCT02508532
View directory record Official record
Conditions
Gastrointestinal Stromal Tumors (GIST), Other Relapsed or Refractory Solid Tumors
Interventions
Avapritinib
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
250 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
9
States / cities
Scottsdale, Arizona • Santa Monica, California • Miami, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 21, 2022 · Synced Oct 1, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

(PATHFINDER) Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients With Advanced Systemic Mastocytosis

NCT03580655
View directory record Official record
Conditions
Advanced Systemic Mastocytosis, Aggressive Systemic Mastocytosis, Systemic Mastocytosis With an Associated Hematologic Neoplasm, Mast Cell Leukemia
Interventions
Avapritinib
Drug
Lead sponsor
Blueprint Medicines Corporation
Industry
Eligibility
18 Years and older
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
12
States / cities
Stanford, California • Chicago, Illinois • Westwood, Kansas + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 24, 2026 · Synced Oct 1, 2026, 2:27 PM EDT