Actively Recruiting
A Phase 2 Open-Label Study of Bezuclastinib for Advanced Systemic Mastocytosis Evaluating Safety, Efficacy, and Pharmacology
Led by Cogent Biosciences, Inc. · Updated on 2026-05-05
140
Participants Needed
42
Research Sites
92 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are investigating CGT9486 (bezuclastinib) in a Phase 2 open-label study for adults with Advanced Systemic Mastocytosis (AdvSM), including Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), and Mast Cell Leukemia (MCL). This study aims to evaluate the drug's safety, efficacy, pharmacokinetics, and pharmacodynamics in these conditions. Participants receive bezuclastinib tablets taken orally in continuous 28-day cycles. The study has two parts conducted over 18 months each: Part I focuses on identifying active and tolerable doses, while Part II assesses treatment effectiveness by measuring the modified IWG Objective Response Rate (ORR) and confirms exposure-response relationships. During the study, participants will undergo evaluations including blood and bone marrow tests, imaging for spleen and liver size, and monitoring of mutation levels and serum tryptase. Researchers will track adverse events, duration and time to response, progression-free and overall survival. The total study duration includes these assessments over 18 months.
CONDITIONS
Brief Title
(Apex) Bezuclastinib in Patients With Advanced Systemic Mastocytosis
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 years or older
- Diagnosed with Aggressive Systemic Mastocytosis (ASM), Systemic Mastocytosis with an Associated Hematologic Neoplasm (SM-AHN), or Mast Cell Leukemia (MCL) confirmed by Eligibility Committee
- Measurable disease by modified IWG-MRT-ECNM criteria or included subset
- ECOG performance status of 0 to 3
- Clinically acceptable local laboratory screening results in clinical chemistry and hematology
- For Substudy Population (Rollover or High-Risk Cohorts), additional specific diagnostic and therapy-related criteria apply
You will not qualify if you...
- Persistent toxicity from previous AdvSM therapy not resolved to Grade 1 or less
- Associated hematologic neoplasm requiring immediate antineoplastic therapy
- Clinically significant cardiac disease
- Positive for FIP1L1 PDGFRA fusion or related mutations if KIT D816V mutation not detected
- Positive serology for HIV 1 or 2, hepatitis B surface antigen, or hepatitis C antibody
- Significant bleeding event within 30 days before first study drug dose or need for therapeutic anticoagulation
- Recent diagnosis or treatment of malignancy other than AdvSM within past 3 years
- Recent use of cytoreductive therapy or investigational agents within 14 to 28 days before screening
- Recent hematopoietic growth factor support within 14 days before first study drug dose
- Recent use of strong CYP3A4 inhibitors or inducers within 14 days or 5 drug half-lives
- Need for treatment with high dose steroids
- Substudy exclusions include Philadelphia chromosome-positive malignancy, acute myeloid leukemia, allogenic stem cell transplant eligibility, contraindications to therapy, and prior investigational or cytoreductive therapy toxicity
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 18 months
Participants take bezuclastinib tablets orally, continuously in 28-day cycles to treat advanced systemic mastocytosis.
Regular visits to monitor safety and efficacy during treatment
Trial Site Locations
Total: 42 locations
1
University of Alabama at Birmingham (UAB) Hospital
Birmingham, Alabama, United States, 35233
Active, Not Recruiting
2
Mayo Clinic Arizona
Phoenix, Arizona, United States, 85054
Actively Recruiting
3
City of Hope Comprehensive Cancer Center
Duarte, California, United States, 91010
Actively Recruiting
4
UCLA Medical Center
Los Angeles, California, United States, 90095
Actively Recruiting
5
Stanford Cancer Institute
Stanford, California, United States, 94305
Actively Recruiting
6
Galiz Research
Hialeah, Florida, United States, 33016
Withdrawn
7
Winship Cancer Institute - Emory University
Atlanta, Georgia, United States, 30322
Actively Recruiting
8
Rush University Medical Center
Chicago, Illinois, United States, 60612
Withdrawn
9
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
10
Columbia University Irving Medical Center
New York, New York, United States, 10032
Withdrawn
11
Cleveland Clinic Taussig Cancer Center
Cleveland, Ohio, United States, 44106
Active, Not Recruiting
12
MUSC Health University Medical Center
Charleston, South Carolina, United States, 29425
Actively Recruiting
13
The University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Actively Recruiting
14
Huntsman Cancer Institute - University of Utah Health
Salt Lake City, Utah, United States, 84112
Actively Recruiting
15
Nepean Hospital
Kingswood, New South Wales, Australia, 2747
Actively Recruiting
16
Gold Coast University Hospital
Southport, Queensland, Australia, 4215
Actively Recruiting
17
Peter MacCallum Cancer Centre
Melbourne N., Victoria, Australia, 3051
Actively Recruiting
18
AKH Wien, Universitatsklinikum
Vienna, Austria, 1090
Active, Not Recruiting
19
CHU de Liege
Liège, Belgium, 4000
Withdrawn
20
University of Alberta Hospital
Edmonton, Alberta, Canada, T6G 2G3
Actively Recruiting
21
St. Michael's Hospital - Unity Health Toronto
Toronto, Ontario, Canada, M5B 1W8
Actively Recruiting
22
Necker-Enfants Malades Hospital
Paris, France, 75015
Active, Not Recruiting
23
Centre Hospitalier Universitaire (CHU) de Poitiers
Poitiers, France, 86000
Active, Not Recruiting
24
Centre Hospitalier Universitaire (CHU) de Toulouse
Toulouse, France, 31300
Active, Not Recruiting
25
University Hospital Aachen
Aachen, Germany, 52074
Active, Not Recruiting
26
Universitätsklinikum Freiburg
Freiburg im Breisgau, Germany, 79104
Active, Not Recruiting
27
UKSH Campus Lubeck
Lübeck, Germany, 23562
Active, Not Recruiting
28
Universitätsklinikum Mannheim
Mannheim, Germany, 68167
Active, Not Recruiting
29
IRCCS Azienda Ospedaliero Universitaria di Bologna
Bologna, Italy, 40138
Active, Not Recruiting
30
Azienda Ospedaliero Universitaria Careggi
Florence, Italy, 50134
Active, Not Recruiting
31
AOU San Giovanni di Dio e Ruggi dAragonia
Salerno, Italy, 84131
Active, Not Recruiting
32
Azienda Ospidaleira Universitaria Integrata Verona
Verona, Italy, 37124
Withdrawn
33
University Medical Center Groningen
Groningen, Netherlands, 9713
Active, Not Recruiting
34
Oslo University Hospital
Oslo, Norway, 0450
Active, Not Recruiting
35
Public University Hospital No. 1 in Lublin
Lublin, Poland, 20-400
Active, Not Recruiting
36
Hospital Universitario Vall d'Hebron
Barcelona, Spain, 08740
Active, Not Recruiting
37
Institut Català d'Oncologia - Hospital Duran i Reynals
Barcelona, Spain, 08908
Active, Not Recruiting
38
Hospital Universitario Ramón y Cajal
Madrid, Spain, 28034
Active, Not Recruiting
39
Universitätsspital Basel
Basel, Switzerland, 4031
Actively Recruiting
40
Guy's Hospital - NHS Foundation Trust
London, London, United Kingdom, SE1 9RT
Actively Recruiting
41
Leeds Teaching Hospitals NHS Trust
Leeds, United Kingdom, LS9 7TF
Actively Recruiting
42
University College London Hospital - NHS Foundation Trust
London, United Kingdom, NW1 2BU
Actively Recruiting
Research Team
C
Cogent Biosciences, Inc.
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Frequently Asked Questions
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