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New treatment option for Advanced Systemic Mastocytosis (AdvSM)
Official title (Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
ClinicalTrials.gov ID: NCT04996875
What this study is testing
What is bezuclastinib?
bezuclastinib is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for advanced systemic mastocytosis (advsm).
Also referred to as CGT9486, PLX9486.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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).
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Key for Main Study:
- Diagnosed with one of the following advanced mastocytosis diagnoses by Eligibility Committee
- Aggressive Systemic Mastocytosis (ASM)
- Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN)
- Mast Cell Leukemia (MCL)
You likely can't join if
- 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...
- Seropositive for human immunodeficiency virus (HIV) 1 or 2, or positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody
See the full eligibility criteria
- Key for Main Study:
- Diagnosed with one of the following advanced mastocytosis diagnoses by Eligibility 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).
- ECOG (0 to 3)
- Have clinically acceptable local laboratory screening results (clinical chemistry, hematology) within certain limits Key
- 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 enrollment
- Seropositive for human immunodeficiency virus (HIV) 1 or 2, or positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody
- 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...
- 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 Criteria for Substudy Population: Rollover Cohort
- 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 side effects 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...
The study team makes the final eligibility decision.
Where it's taking place
- Birmingham, Alabama, United States
- Phoenix, Arizona, United States
- Duarte, California, United States
- Los Angeles, California, United States
- Stanford, California, United States
- Hialeah, Florida, United States
- Atlanta, Georgia, United States
- Chicago, Illinois, United States
- Boston, Massachusetts, United States
- New York, New York, United States
- Cleveland, Ohio, United States
- Charleston, South Carolina, United States
- Houston, Texas, United States
- Salt Lake City, Utah, United States
- Kingswood, New South Wales, Australia
- Southport, Queensland, Australia
- Melbourne N., Victoria, Australia
- Vienna, Austria
- Liège, Belgium
- Edmonton, Alberta, Canada
+ 21 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Birmingham, Alabama, United States; Phoenix, Arizona, United States; Duarte, California, United States; Los Angeles, California, United States; Stanford, California, United States; Hialeah, Florida, United States and 35 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.