Advanced Systemic MastocytosisPhase 1AZURENCT05609942
Study of Elenestinib (BLU-263) in Advanced Systemic Mastocytosis (AdvSM) and and Other KIT Altered Hematologic Malignancies
A Phase 1 study testing BLU-263 and Azacitidine in people with Advanced Systemic Mastocytosis, run by Blueprint Medicines Corporation at 11 sites in 7 countries.
Trial summary
The goal of this clinical trial is to evaluate elenestinib (BLU-263) in participants with Advanced Systemic Mastocytosis (AdvSM), SM with an associated hematologic neoplasm (SM-AHN), and other hematologic malignancies. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Systemic Mastocytosis
- What is being tested?
- BLU-263 and Azacitidine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 11 sitesin 7 countries
- Study dates
- 25 Sep 2023to 10 Mar 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant has Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0-2
- Participant must have a new Bone Marrow (BM) biopsy or may use archival tissue if taken within 56 days prior to C1D1 and participant must be willing to have follow-up BM Biopsies.
- Participants receiving antineoplastic therapy within the preceding 12 weeks must have discontinued therapy due to disease progression, refractory disease, lack of efficacy, or intolerance.
- Participants treated with 1 prior selective KIT inhibitor (such as avapritinib or CGT9486) will be permitted on study after confirmation of KIT D816V mutation and with written approval of the study Sponsor. Participants who discontinued treatment with a prior selective KIT inhibitor due to a severe AE that was thought to be related to prior treatment will not be eligible to participate in the study.
- Arm 1 (Monotherapy): Participants must have one of the following AdvSM diagnoses, based on World Health Organization (WHO) diagnostic criteria.
- Before enrollment, diagnosis of AdvSM must be confirmed based on Central Pathology Laboratory assessment of BM:
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- Aggressive SM (ASM).
- SM-AHN that in the opinion of the Investigator is not considered to be a candidate for Hypomethylating agent (HMA) monotherapy. Incidental indolent, low-grade lymphoid AHNs (eg, chronic lymphocytic leukemia) not requiring treatment are eligible.
- Mast cell leukemia (MCL), including diagnoses with an AHN component, that does not require a C-finding.
- Upon discussion with the Sponsor, other relapsed or refractory hematologic neoplasms with evidence of aberrant KIT or PDGFR may be considered for enrollment. (eg, participants with chronic myeloid neoplasms, such as subvariants of MDS/MPN that harbor activating KIT exon 17 mutations but do not fulfill the diagnostic criteria of SM-AHN, and participants with myeloid/lymphoid neoplasms with PDGFRa/b fusion genes and mutations conferring resistance to imatinib, such as T674I or D842V).
Who cannot join
- Diagnosis of a Philadelphia chromosome positive malignancy
- Acute myeloid leukemia.
- If the participant is receiving corticosteroids, and the dose has not been stable for ≥7 days.
- Within the 14 days prior to enrollment, participant has received any antineoplastic therapy (including midostaurin, avapritinib and other tyrosine kinase inhibitors [TKIs]) or an investigational agent.
- Participant has received hydroxyurea within 7 days prior to the first dose of elenestinib (BLU-263).
- Participant received prior HMA therapy (e.g., azacitidine, decitabine) for the current diagnosis.
Show all 20Show fewer
- Participant must not be eligible for allogenic hematopoietic stem cell transplantation.
- Participant received prior radiotherapy within 14 days of screening BM biopsy.
- Participant received any hematopoietic growth factor (except erythropoietin) within 14 days of screening BM biopsy, or requiring growth factors to maintain adequate neutrophil or platelet levels.
- Those participants maintained on a chronic dose of erythropoietin, whose hemoglobin is stable, and dose of erythropoietin has not been changed in the prior 28 days are allowed on study.
- Participant received >1 prior selective KIT inhibitor (eg: avapritinib or bezuclastinib).
- Participant have any of the following laboratory abnormalities on last laboratory assessment within 14 days prior to the first dose of initiation of study drug: a. Alanine aminotransferase and aspartate aminotransferase > 3 × ULN; > 5 × ULN if associated with clinically suspected liver infiltration by mastocytosis or another disease for which the patient enrolled into the study b. Total bilirubin > 1.5 × ULN; > 3 × ULN if associated with liver infiltration by the disease being treated or in the presence of Gilbert's Disease. (In the case of Gilbert's disease, a direct bilirubin > 2.0 ULN would be an exclusion) c. Estimated (Cockcroft-Gault formula) or measured creatinine clearance < 40 mL/min d. Absolute neutrophil count < 0.5 × 10^9/L
- Participant has had a major surgical procedure within 14 days of the first dose of study drug.
- History of another primary malignancy that has been diagnosed or required therapy within 1 year prior to the first dose of study drug. The following are exempt from the 1-year limit: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, GI stromal tumor, and completely resected carcinoma in situ of any site.
- Mean resting QTcF > 480 msec, a history of prolonged QT syndrome or Torsades de pointes, or a familial history of prolonged QT syndrome.
- Clinically significant, uncontrolled, cardiovascular disease.
- Arm 1 (Monotherapy):
- Myelodysplastic Syndrome (MDS) that is very high- or high-risk as defined by the International Prognostic Scoring System for Myelodysplastic Syndromes-Revised (IPSS-R).
- A myeloid AHN with ≥10% BM or peripheral blood blasts.
- Platelet count <50 x 10^9/L (within 4 weeks prior to the first dose of study drug) or receiving platelet transfusions or thrombopoietin receptor agonists (TPO-RA) within the prior 14 days.
Trial locations and contacts
11 sites in 7 countries.
11 sites in 7 countries
United States4 sites
University of Michigan
Ann Arbor, Michigan, 48109
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Stanford Cancer Institute
Palo Alto, California, 94305
Huntsman Cancer Institute
Salt Lake City, Utah, 84112
Belgium2 sites
Antwerp University Hospital
Edegem, 2650
University Hospital Ghent
Ghent, 9000
Netherlands1 site
Maastricht University Medical Center
Maastricht, 6229 HX
Spain1 site
Instituto de Estudios de Mastocitosis de Castilla La Mancha (CLMast)
Toledo, 45071
About this study
Official title: A Phase 1/2, Open-label, 2-arm Study Evaluating BLU-263 as Monotherapy and in Combination With Azacitidine, in Patients With KIT Altered Hematologic Malignancies
The goal of this clinical trial is to evaluate elenestinib (BLU-263) in participants with Advanced Systemic Mastocytosis (AdvSM), SM with an associated hematologic neoplasm (SM-AHN), and other hematologic malignancies. The main questions it aims to answer are:
- Determine Recommended Dose of elenestinib (BLU-263) monotherapy for participants with AdvSM
- Safety and tolerability of elenestinib (BLU-263) monotherapy
- Efficacy of elenestinib (BLU-263) monotherapy in participants with AdvSM
- Determine Recommended Dose of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM
- Safety and tolerability of elenestinib (BLU-263) in combination with azacitidine
- Efficacy of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM
The estimated study duration for each participant will be approximately 4 years: 2 years of treatment followed by 2 years of follow-up. Participants may be required to attend monthly visits for the first six months, followed by quarterly visits for the remainder of the study.
Read the detailed description
Systemic mastocytosis includes five major subtypes: Indolent SM (ISM), SM with an associated hematologic neoplasm (SM-AHN), aggressive SM (ASM), and MC leukemia (MCL). In 2016, the smoldering subtype of SM, a former provisional ISM subvariant, was designated as a distinct variant of SM by the World Health Organization (WHO). Aggressive SM, SM-AHN, and MCL together are referred to as Advanced SM (AdvSM).
- Conditions
- Advanced Systemic Mastocytosis
- Being tested
- BLU-263, Azacitidine
- Keywords
- BLU-263, elenestinib, azacitidine, AdvSM, KIT D816V, Advanced systemic mastocytosis, Mast cell leukemia, MCL, Systemic mastocytosis, SM, SM-AHN, CMML-2, Myelodysplastic/myeloproliferative neoplasms, MDS/MPN, Myeloid neoplasms, ASM
- Medical subject headings
- Mastocytosis, Systemic, Leukemia, Mast-Cell, Myelodysplastic-Myeloproliferative Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BLU-263Drug
Also called elenestinib
BLU-263 Oral Tablets
AzacitidineDrug
Azacitidine powder for suspension for intravenous infusion / subcutaneous injection
| Study group | Role | What they receive |
|---|---|---|
| Monotherapy | Experimental | Participants with AdvSM (ASM, SM-AHN, or MCL) will receive BLU-263 monotherapy. Receives: Drug: BLU-263 |
| Combination therapy | Experimental | Participants with high risk and very high risk systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) of non-MC lineage will receive BLU-263 in combination with azacitidine. Receives: Drug: BLU-263; Drug: Azacitidine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Dose Escalation: Number of Dose-limiting Toxicities (DLTs) (monotherapy only)
Monotherapy: The Recommended Dose (RD) will be primarily determined by the number of DLTs in the first 28 days of treatment with elenestinib (BLU-263) monotherapy.
Measured: 28 Days
Dose Escalation: Number of DLTs (combination therapy only)
Combination therapy: The RD will be primarily determined by the number of DLTs (during 28 days starting from Day 15 of C1 or Day 15 of C2) with elenestinib (BLU-263) in combination with azacitidine.
Measured: 28 Days
Dose Escalation and Expansion: Pure Pathological Response (PPR) Rate for SM in Selective KIT Inhibitor-naïve Participants (monotherapy only)
PPR Rate is defined as complete remission (resolution of palpable splenomegaly/hepatomegaly) (CR) + complete remission with partial recovery of peripheral blood counts (CRh) + partial remission (≥35% reduction in spleen volume) (PR)
Measured: Up to approximately 4 years
Dose Escalation and Expansion: Number of Participants with Adverse Events (AEs)
Measured: Up to approximately 4 years
Dose Escalation and Expansion: Number of Participants with Serious Adverse Events (SAEs)
Measured: Up to approximately 4 years
Secondary outcomes (22)
Dose Escalation and Expansion: Overall Response Rate (ORR) for AdvSM using modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (IWG-MRT-ECNM) (monotherapy only)
ORR is defined as CR + CRh + PR + Clinical Improvement (CI)
Measured: Up to approximately 4 years
Dose Escalation and Expansion: ORR for SM Using Modified IWG-MRT-ECNM (combination therapy only)
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Maximum Plasma Concentration (Cmax) of BLU-263
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Cmax of Azacitidine (combination therapy only)
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Time to Maximum Concentration (Tmax) of BLU-263
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Tmax of Azacitidine (combination therapy only)
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Area Under the Curve From Time Zero to 24 Hours (AUC(0-24)) of BLU-263
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: AUC(0-24) of Azacitidine (combination therapy only)
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Apparent Volume of Distribution (Vz/F) of BLU-263
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Vz/F of Azacitidine (combination therapy only)
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Terminal Elimination Half-life (t1/2) of BLU-263
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: t1/2 of Azacitidine (combination therapy only)
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Apparent Oral Clearance (CL/F) of BLU-263
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: CL/F of Azacitidine (combination therapy only)
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Accumulation Ratio of BLU-263
Measured: Up to approximately 4 years
Dose Escalation and Dose Expansion: Accumulation Ratio of Azacitidine (combination therapy only)
Measured: Up to approximately 4 years
Dose Escalation and Expansion: Overall Survival (OS) (monotherapy only)
Measured: Up to approximately 4 years
Dose Escalation and Expansion: Time to Response (TtR) (monotherapy only)
Measured: Up to approximately 4 years
Dose Escalation and Expansion: Duration of Response (DOR) (monotherapy only)
Measured: Up to approximately 4 years
Dose Escalation and Expansion: Progression-Free Survival (PFS) (monotherapy only)
Measured: Up to approximately 4 years
Dose Escalation and Expansion: Proportion of Participants Pursuing Stem Cell Transplant (monotherapy only)
Measured: Up to approximately 4 years
Dose Escalation and Expansion: PPR Rate for SM (combination therapy only)
Measured: Up to approximately 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Nov 2022
- Study start25 Sep 2023
- Primary completion14 Jan 2025
- Study completion10 Mar 2025
Why it stopped: Terminated - alignment of strategic priorities
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Systemic MastocytosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Blueprint Medicines Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05609942
- Sponsor's protocol ID
- BLU-263-2101
- Other IDs
- 2022-001535-87
- Acronym
- AZURE
- Status last verified
- Mar 2025
- First submitted
- 2 Nov 2022
- Last updated
- 10 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2, Open-label, 2-arm Study Evaluating BLU-263 as Monotherapy and in Combination With Azacitidine, in Patients With KIT Altered Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT05609942
- Acronym / sponsor ID
- AZURE / BLU-263-2101
- Conditions
- Advanced Systemic Mastocytosis
- Keywords
- BLU-263, elenestinib, azacitidine, AdvSM, KIT D816V, Advanced systemic mastocytosis, Mast cell leukemia, MCL
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Terminated - alignment of strategic priorities
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Blueprint Medicines CorporationIndustry (pharma, biotech, devices)
- Study start
- 25 Sep 2023 (actual)
- Primary completion
- 14 Jan 2025 (actual)
- Study completion
- 10 Mar 2025 (actual)
- First posted
- 8 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical trial is to evaluate elenestinib (BLU-263) in participants with Advanced Systemic Mastocytosis (AdvSM), SM with an associated hematologic neoplasm (SM-AHN), and other hematologic malignancies. The main questions it aims to answer are:
- Determine Recommended Dose of elenestinib (BLU-263) monotherapy for participants with AdvSM
- Safety and tolerability of elenestinib (BLU-263) monotherapy
- Efficacy of elenestinib (BLU-263) monotherapy in participants with AdvSM
- Determine Recommended Dose of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM
- Safety and tolerability of elenestinib (BLU-263) in combination with azacitidine
- Efficacy of elenestinib (BLU-263) in combination with azacitidine in participants with AdvSM
The estimated study duration for each participant will be approximately 4 years: 2 years of treatment followed by 2 years of follow-up. Participants may be required to attend monthly visits for the first six months, followed by quarterly visits for the remainder of the study.
Detailed description
Systemic mastocytosis includes five major subtypes: Indolent SM (ISM), SM with an associated hematologic neoplasm (SM-AHN), aggressive SM (ASM), and MC leukemia (MCL). In 2016, the smoldering subtype of SM, a former provisional ISM subvariant, was designated as a distinct variant of SM by the World Health Organization (WHO). Aggressive SM, SM-AHN, and MCL together are referred to as Advanced SM (AdvSM).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Monotherapy | Experimental | Drug: BLU-263Participants with AdvSM (ASM, SM-AHN, or MCL) will receive BLU-263 monotherapy. |
| Combination therapy | Experimental | Drug: BLU-263, Drug: AzacitidineParticipants with high risk and very high risk systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) of non-MC lineage will receive BLU-263 in combination with azacitidine. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Participant has Eastern Cooperative Oncology Group (ECOG) performance status (PS) of 0-2
- Participant must have a new Bone Marrow (BM) biopsy or may use archival tissue if taken within 56 days prior to C1D1 and participant must be willing to have follow-up BM Biopsies.
- Participants receiving antineoplastic therapy within the preceding 12 weeks must have discontinued therapy due to disease progression, refractory disease, lack of efficacy, or intolerance.
- Participants treated with 1 prior selective KIT inhibitor (such as avapritinib or CGT9486) will be permitted on study after confirmation of KIT D816V mutation and with written approval of the study Sponsor. Participants who discontinued treatment with a prior selective KIT inhibitor due to a severe AE that was thought to be related to prior treatment will not be eligible to participate in the study.
- Arm 1 (Monotherapy): Participants must have one of the following AdvSM diagnoses, based on World Health Organization (WHO) diagnostic criteria.
Key exclusion criteria (20 listed)
- Diagnosis of a Philadelphia chromosome positive malignancy
- Acute myeloid leukemia.
- If the participant is receiving corticosteroids, and the dose has not been stable for ≥7 days.
- Within the 14 days prior to enrollment, participant has received any antineoplastic therapy (including midostaurin, avapritinib and other tyrosine kinase inhibitors [TKIs]) or an investigational agent.
- Participant has received hydroxyurea within 7 days prior to the first dose of elenestinib (BLU-263).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BLU-263Also: elenestinibBLU-263 Oral Tablets | Drug | Combination therapy, Monotherapy |
| AzacitidineAzacitidine powder for suspension for intravenous infusion / subcutaneous injection | Drug | Combination therapy |
Primary outcome
Dose Escalation: Number of Dose-limiting Toxicities (DLTs) (monotherapy only)
Time frame: 28 Days
Monotherapy: The Recommended Dose (RD) will be primarily determined by the number of DLTs in the first 28 days of treatment with elenestinib (BLU-263) monotherapy.
Dose Escalation: Number of DLTs (combination therapy only)
Time frame: 28 Days
Combination therapy: The RD will be primarily determined by the number of DLTs (during 28 days starting from Day 15 of C1 or Day 15 of C2) with elenestinib (BLU-263) in combination with azacitidine.
Dose Escalation and Expansion: Pure Pathological Response (PPR) Rate for SM in Selective KIT Inhibitor-naïve Participants (monotherapy only)
Time frame: Up to approximately 4 years
PPR Rate is defined as complete remission (resolution of palpable splenomegaly/hepatomegaly) (CR) + complete remission with partial recovery of peripheral blood counts (CRh) + partial remission (≥35% reduction in spleen volume) (PR)
Dose Escalation and Expansion: Number of Participants with Adverse Events (AEs)
Time frame: Up to approximately 4 years
Dose Escalation and Expansion: Number of Participants with Serious Adverse Events (SAEs)
Time frame: Up to approximately 4 years
22 secondary outcomes
Dose Escalation and Expansion: Overall Response Rate (ORR) for AdvSM using modified International Working Group-Myeloproliferative Neoplasms Research and Treatment and European Competence Network on Mastocytosis (IWG-MRT-ECNM) (monotherapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Expansion: ORR for SM Using Modified IWG-MRT-ECNM (combination therapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Maximum Plasma Concentration (Cmax) of BLU-263
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Cmax of Azacitidine (combination therapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Time to Maximum Concentration (Tmax) of BLU-263
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Tmax of Azacitidine (combination therapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Area Under the Curve From Time Zero to 24 Hours (AUC(0-24)) of BLU-263
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: AUC(0-24) of Azacitidine (combination therapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Apparent Volume of Distribution (Vz/F) of BLU-263
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Vz/F of Azacitidine (combination therapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Terminal Elimination Half-life (t1/2) of BLU-263
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: t1/2 of Azacitidine (combination therapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Apparent Oral Clearance (CL/F) of BLU-263
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: CL/F of Azacitidine (combination therapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Accumulation Ratio of BLU-263
Time frame: Up to approximately 4 years
Dose Escalation and Dose Expansion: Accumulation Ratio of Azacitidine (combination therapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Expansion: Overall Survival (OS) (monotherapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Expansion: Time to Response (TtR) (monotherapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Expansion: Duration of Response (DOR) (monotherapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Expansion: Progression-Free Survival (PFS) (monotherapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Expansion: Proportion of Participants Pursuing Stem Cell Transplant (monotherapy only)
Time frame: Up to approximately 4 years
Dose Escalation and Expansion: PPR Rate for SM (combination therapy only)
Time frame: Up to approximately 4 years
Results and safety
The study was stopped early (reason given: Terminated - alignment of strategic priorities). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
11 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BLU-263 and Azacitidine in people with Advanced Systemic Mastocytosis, run by Blueprint Medicines Corporation at 11 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 10 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in United States, Belgium, France, Germany, Netherlands and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: Terminated - alignment of strategic priorities). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose Escalation: Number of Dose-limiting Toxicities (DLTs) (monotherapy only) (time frame: 28 Days). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Apr 2025.