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MPN Clinical Trial Finder

Clinical Trial Finder

Search Results

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

Study Purpose

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.

Recruitment Criteria

Accepts Healthy Volunteers

Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms

No
Study Type

An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.


An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.


Searching Both is inclusive of interventional and observational studies.

Interventional
Eligible Ages 18 Years and Over
Gender All
More Inclusion & Exclusion Criteria

Key Inclusion Criteria :

  • - 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: 1. Aggressive SM (ASM). 2. 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. 3. Mast cell leukemia (MCL), including diagnoses with an AHN component, that does not require a C-finding. 4. 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). Key

Exclusion Criteria:

  • - 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.
  • - 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 Details

Trial ID:

This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.

NCT05609942
Phase

Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.

Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.

Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.

Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.

Phase 1/Phase 2
Lead Sponsor

The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.

Blueprint Medicines Corporation
Principal Investigator

The person who is responsible for the scientific and technical direction of the entire clinical study.

N/A
Principal Investigator Affiliation N/A
Agency Class

Category of organization(s) involved as sponsor (and collaborator) supporting the trial.

Industry
Overall Status Recruiting
Countries Belgium, France, Germany, Netherlands, Norway, Spain, United States
Conditions

The disease, disorder, syndrome, illness, or injury that is being studied.

Advanced Systemic Mastocytosis
Additional Details

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).

Arms & Interventions

Arms

Experimental: Monotherapy

Participants with AdvSM (ASM, SM-AHN, or MCL) will receive BLU-263 monotherapy.

Experimental: Combination therapy

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.

Interventions

Drug: - BLU-263

BLU-263 Oral Tablets

Drug: - Azacitidine

Azacitidine powder for suspension for intravenous infusion / subcutaneous injection

Contact a Trial Team

If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.

Stanford Cancer Institute, Palo Alto, California

Status

Not yet recruiting

Address

Stanford Cancer Institute

Palo Alto, California, 94305

Site Contact

[email protected]

617-714-6707

Dana-Farber Cancer Institute, Boston, Massachusetts

Status

Recruiting

Address

Dana-Farber Cancer Institute

Boston, Massachusetts, 02215

Site Contact

[email protected]

617-714-6707

University of Michigan, Ann Arbor, Michigan

Status

Recruiting

Address

University of Michigan

Ann Arbor, Michigan, 48109

Site Contact

[email protected]

617-714-6707

Huntsman Cancer Institute, Salt Lake City, Utah

Status

Recruiting

Address

Huntsman Cancer Institute

Salt Lake City, Utah, 84112

Site Contact

[email protected]

617-714-6707

International Sites

Antwerp University Hospital, Edegem, Belgium

Status

Recruiting

Address

Antwerp University Hospital

Edegem, , 2650

Site Contact

[email protected]

617-714-6707

University Hospital Ghent, Ghent, Belgium

Status

Recruiting

Address

University Hospital Ghent

Ghent, , 9000

Site Contact

[email protected]

617-714-6707

Caen, France

Status

Recruiting

Address

CHU Caen - Institut d'Hematologie de Basse Normandie

Caen, , 14033

Site Contact

[email protected]

617-714-6707

University Medical Centre Mannheim, Mannheim, Germany

Status

Recruiting

Address

University Medical Centre Mannheim

Mannheim, , 68167

Site Contact

[email protected]

617-714-6707

Maastricht University Medical Center, Maastricht, Netherlands

Status

Recruiting

Address

Maastricht University Medical Center

Maastricht, , 6229 HX

Site Contact

[email protected]

617-714-6707

Oslo University Hospital, Oslo, Norway

Status

Recruiting

Address

Oslo University Hospital

Oslo, , 0450

Site Contact

[email protected]

617-714-6707

Toledo, Spain

Status

Recruiting

Address

Instituto de Estudios de Mastocitosis de Castilla La Mancha (CLMast)

Toledo, , 45071

Site Contact

[email protected]

617-714-6707

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The content provided on clinical trials is for informational purposes only and is not a substitute for medical consultation with your healthcare provider. We do not recommend or endorse any specific study and you are advised to discuss the information shown with your healthcare provider. While we believe the information presented on this website to be accurate at the time of writing, we do not guarantee that its contents are correct, complete, or applicable to any particular individual situation. We strongly encourage individuals to seek out appropriate medical advice and treatment from their physicians. We cannot guarantee the availability of any clinical trial listed and will not be responsible if you are considered ineligible to participate in a given clinical trial. We are also not liable for any injury arising as a result of participation.

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