Taking people in SwitzerlandPhase 2
Tests bezuclastinib in adults with advanced systemic mastocytosis
Tests bezuclastinib in adults with advanced systemic mastocytosis, a rare blood disease.
Key facts
- Who runs it
- Cogent Biosciences, Inc.
- Where
- Basel, SwitzerlandSee the siteAlso in 13 other countries, 41 sites
- Registry title
- (Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
- Status
- Recruiting
- Phase
- Phase 2
- People taking part
- 140 people, planned
- Started
- 9 Nov 2021
- Expected to end
- Jul 2027
- Type of study
- Interventional
- Treatments tested
- bezuclastinib
- Record updated
- 15 September 2026
Who can take part
AI summary
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for adults with advanced systemic mastocytosis: aggressive disease, disease with another blood cancer, or mast cell leukemia. All receive bezuclastinib.
- Age
- 18 years or older
- Sex
- All sexes can join
- Disease stage
- Advanced systemic mastocytosis: aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematologic neoplasm (SM-AHN), or mast cell leukemia (MCL). Disease that can be measured. ECOG 0 to 3.
- Earlier treatment
- Side effects from earlier treatment must have eased to Grade 1 or less. No cytoreductive therapy or investigational drug in the last 14 days (28 days for cladribine, interferon alpha, pegylated interferon and antibody therapy). No growth factor support in the last 14 days. No strong CYP3A4 inhibitors or inducers in the last 14 days or 5 drug half-lives.
- Biomarker or test result
- People with the FIP1L1 PDGFRA fusion cannot join. People with eosinophilia and no detectable KIT D816V mutation must show no PDGFRA fusion.
Main reasons for not taking part
- Another blood cancer that needs cancer treatment right away
- Clinically significant heart disease
- HIV, hepatitis B or hepatitis C infection
- Significant bleeding in the last 30 days, or need for blood thinners
- Another cancer diagnosed or treated in the last 3 years
The registry criteria, word for word
Show all 30 criteriaShow less
Who can take part
- 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
- 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
Who cannot take part
- Persistent toxicity from previous therapy for AdvSM that has not resolved to ≤ Grade 1
- Associated hematologic neoplasm requiring immediate antineoplastic therapy
- 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
- 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
- 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
- Clinically significant cardiac disease
- Seropositive for human immunodeficiency virus (HIV) 1 or 2, or positive for hepatitis B surface antigen or hepatitis C virus (HCV) antibody
- Diagnosis of Philadelphia chromosome-positive malignancy
- Diagnosis of acute myeloid leukemia (AML)
Check if you might fit
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- How old are you?
- Do you have serious heart disease, such as a recent heart attack or heart failure?
- Do you have an infection such as HIV, hepatitis B or hepatitis C?
Where in Switzerland
Also in 13 other countries, 41 sites
- Universitätsspital BaselBasel, SwitzerlandContact: Cogent Biosciences, Inc.Taking people
About the trial
What the trial wants to find out
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).
The group in the trial
- bezuclastinibTreatment being tested
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorData and sources
- Registry record
- Listed on ClinicalTrials.gov and on the EU's official list of trials (CTIS).
- Official title
- 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
- Plain-language title
- Tests bezuclastinib in adults with advanced systemic mastocytosisGenerated from the registry record. The registry title is the official one.
- AI summary
- From the trial's eligibility criteria. Read by machine on 12 November 2026.
- Fit questions
- Built from NCT04996875, who can take part
- Record updated
- 15 September 2026