Testing bezuclastinib for advanced systemic mastocytosis (advsm)
Official title: (Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
A Phase 2 Open-Label, Multicenter Clinical Study of the Safety, Efficacy, Pharmacokinetic, and Pharmacodynamic Profiles of CGT9486 as a Single Agent in Patients With Advanced Systemic Mastocytosis
- Phase 2
- 1 group
- Sites in Edmonton and Toronto
- Recruiting
Interventions
- Medication
bezuclastinib
Bezuclastinib is administered as tablets to be taken orally, continuously in 28-day cycles.
Canadian Sites (2)
2 of 2 recruiting
- Recruiting
University of Alberta Hospital
Edmonton, Alberta
- Recruiting
St. Michael's Hospital - Unity Health Toronto
Toronto, Ontario
Eligibility Criteria
See who this study is looking for76 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +ECOG (0 to 3)
- +Seropositive for human immunodeficiency virus (HIV) 1 or 2, or positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody
- +for Main Study:
- +Diagnosed with one of the following advanced mastocytosis diagnoses by EligibilityEligibility criteriaThe full list of requirements for taking part in a study.Read more → Committee
- +Aggressive Systemic Mastocytosis (ASM)
- +Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN)
- +Mast Cell Leukemia (MCL)
- +Measurable disease according to modified IWG-MRT-ECNM criteria. (A subset of patients inevaluble per mIWG-MRT-ECNM will be included in the study).
- +Have clinically acceptable local laboratory screeningScreeningThe checks done before joining, to see whether a study fits.Read more → results (clinical chemistry, hematology) within certain limits
- +Key Exclusion CriteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → for Main Study:
- +Persistent toxicity from previous therapy for AdvSM that has not resolved to ≤ Grade 1
- +Associated hematologic neoplasm requiring immediate antineoplastic therapy
- +Clinically significant cardiac disease
- +Known positivity for the FIP1L1 PDGFRA fusion. Patients with eosinophilia without detectable KIT D816V mutation must demonstrate lack of PDGFRA fusion mutation prior to enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →
- +History of clinically significant bleeding event within 30 days before the first dose of study drug or need for therapeutic anticoagulation on study
- +Diagnosed with or treated for malignancy other than the disease under study within the prior 3 years before enrollment
- +Received any cytoreductive therapy or any investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon, and any antibody therapy less than 28 days, before screening bone marrow biopsy
- +Received hematopoietic growth factor support within 14 days before the first dose of study drug
- +Received strong CYP3A4 inhibitors or inducers within 14 days or 5 drug half-lives, whichever is longer, before the first dose of study drug
- +Need for treatment with high dose steroids
- +Key Inclusion CriteriaInclusion criteriaThe things you must have or be for a study to consider you.Read more → for Substudy Population:
- +Rollover CohortCohortA group of participants sharing a characteristic, followed together.Read more →
- +Demonstrate AHN progression requiring immediate AHN-directed therapy while receiving bezuclastinib
- +Demonstrated clinical benefit from bezuclastinib therapy
- +Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits
- +High-Risk Cohort
- +Receiving or indicated for AHN-directed therapy.
- +Diagnosed with one of the following pathologic diagnoses of SM-AHN:
- +Myelodysplastic syndrome (MDS) that is high- or very high-risk
- +Accelerated phase myeloproliferative neoplasm (MPN)
- +MDS with excessive blasts in bone marrow or peripheral blood
- +Chronic myelomonocytic leukemia-2 (CMML-2)
- +Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits.
- +Key Exclusion Criteria for Substudy Population:
- +Diagnosis of Philadelphia chromosome-positive malignancy
- +Diagnosis of acute myeloid leukemia (AML)
- +Appropriate for allogenic hematopoietic stem cell transplantation
- +Any contraindication to selected concomitant therapy
- +Rollover Cohort: Have not demonstrated acceptable tolerability of previous bezuclastinib therapy
- +High-Risk Cohort: Previously treated with investigational therapy for AdvSM
- +High-Risk Cohort: Previously treated with cytoreductive therapy and discontinued due to treatment-related toxicity
- +High-Risk Cohort: Received any cytoreductive therapy or any investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon, and any antibody therapy less than 28 days, before screening or archival bone marrow biopsy
Exclusion
- −Seropositive for human immunodeficiency virus (HIV) 1 or 2, or positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody
- −for Main Study:
- −Persistent toxicity from previous therapy for AdvSM that has not resolved to ≤ Grade 1
- −Associated hematologic neoplasm requiring immediate antineoplastic therapy
- −Clinically significant cardiac disease
- −Known positivity for the FIP1L1 PDGFRA fusion. Patients with eosinophilia without detectable KIT D816V mutation must demonstrate lack of PDGFRA fusion mutation prior to enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →
- −History of clinically significant bleeding event within 30 days before the first dose of study drug or need for therapeutic anticoagulation on study
- −Diagnosed with or treated for malignancy other than the disease under study within the prior 3 years before enrollment
- −Received any cytoreductive therapy or any investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon, and any antibody therapy less than 28 days, before screeningScreeningThe checks done before joining, to see whether a study fits.Read more → bone marrow biopsy
- −Received hematopoietic growth factor support within 14 days before the first dose of study drug
- −Received strong CYP3A4 inhibitors or inducers within 14 days or 5 drug half-lives, whichever is longer, before the first dose of study drug
- −Need for treatment with high dose steroids
- −Key Inclusion CriteriaInclusion criteriaThe things you must have or be for a study to consider you.Read more → for Substudy Population:
- −Rollover CohortCohortA group of participants sharing a characteristic, followed together.Read more →
- −Demonstrate AHN progression requiring immediate AHN-directed therapy while receiving bezuclastinib
- −Demonstrated clinical benefit from bezuclastinib therapy
- −Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits
- −High-Risk Cohort
- −Receiving or indicated for AHN-directed therapy.
- −Diagnosed with one of the following pathologic diagnoses of SM-AHN:
- −Myelodysplastic syndrome (MDS) that is high- or very high-risk
- −Accelerated phase myeloproliferative neoplasm (MPN)
- −MDS with excessive blasts in bone marrow or peripheral blood
- −Chronic myelomonocytic leukemia-2 (CMML-2)
- −Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits.
- −Key Exclusion CriteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → for Substudy Population:
- −Diagnosis of Philadelphia chromosome-positive malignancy
- −Diagnosis of acute myeloid leukemia (AML)
- −Appropriate for allogenic hematopoietic stem cell transplantation
- −Any contraindication to selected concomitant therapy
- −Rollover Cohort: Have not demonstrated acceptable tolerability of previous bezuclastinib therapy
- −High-Risk Cohort: Previously treated with investigational therapy for AdvSM
- −High-Risk Cohort: Previously treated with cytoreductive therapy and discontinued due to treatment-related toxicity
- −High-Risk Cohort: Received any cytoreductive therapy or any investigational agent less than 14 days, and for cladribine, interferon alpha, pegylated interferon, and any antibody therapy less than 28 days, before screening or archival bone marrow biopsy
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more → — a middle-stage study in a few hundred people at most, looking at what the treatment does and watching for side effectsSide effectAn unwanted effect thought to be caused by the treatment itself.Read more →.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
Everyone in this study is in one group and receives bezuclastinib.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
There is only one group in this study, so there is no assignment to different treatments.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists a minimum age of 18 years, with no upper limit given.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 140 people.
The study is currently expected to finish around July 2027.
The main measurement is taken over: 18 months.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Part I: Identify clinically active and tolerable exposures of bezuclastinib in patients with AdvSM — measured over 18 months.
Part II: - Determine efficacy of bezuclastinib as measured by mIWG Objective Response Rate (ORR) - Confirm the exposure-response relationship of bezuclastinib — measured over 18 months.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT04996875 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About bezuclastinibDrug
Bezuclastinib is administered as tablets to be taken orally, continuously in 28-day cycles.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: a minimum age of 18 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
There is only one group in this study, so there is no assignment to different treatments.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists a minimum age of 18 years, with no upper limit given.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: 18 months.
The study as a whole is currently expected to finish around 2027-07.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 140 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 3 locations, including: Edmonton, Alberta, Canada; Toronto, Ontario, Canada; New York, New York, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
This is an open-label, two-part Phase 2 study investigating CGT9486 for the treatment of patients with Advanced Systemic Mastocytosis (AdvSM), including patients with Aggressive SM (ASM), SM with Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL).
Other Sites (1)
Columbia University Irving Medical Center
New York, New York, United States
Think this trial might be right for you?
Complete a quick intake form and we will match you with this and other relevant trials based on your medical profile.
See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.