Study CBX-250-001 is a Phase 1, open-label, dose-escalation study of CBX-250 in participants with relapsed/refractory AML, HR-MDS, CMML, and CML. Participants aged ≥ 12 years are planned to be enrolled. CBX-250 will initially be investigated on a fixed step-up dosing schedule. CBX-250 will be administered subcutaneously in 28-day cycles, with the first study drug dose administered on Cycle 1, Day 1. Cycle 1 will consist of a priming phase over 7 days, and a target phase over 28 days. Participants will continue CBX-250 until progressive disease (PD) or unacceptable toxicity. All subsequent treatment cycles will be 28 days.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
72
subcutaneous CBX-250
City of Hope
Duarte, California, United States
RECRUITINGStanford Medical Center
Palo Alto, California, United States
RECRUITINGMoffitt Cancer Center
Tampa, Florida, United States
RECRUITINGNorthwestern Medicine
Chicago, Illinois, United States
RECRUITINGDana Farber Cancer Institute
Boston, Massachusetts, United States
RECRUITINGWashington University in St. Louis
St Louis, Missouri, United States
RECRUITINGMemorial Sloan Kettering Cancer Center
New York, New York, United States
RECRUITINGThomas Jefferson University Hospital
Philadelphia, Pennsylvania, United States
RECRUITINGSarah Cannon Cancer Institute
Nashville, Tennessee, United States
RECRUITINGVanderbilt University Medical Center
Nashville, Tennessee, United States
RECRUITING...and 1 more locations
To determine the safety, tolerability, RP2D, or MTD if different, and dosing regimen of CBX-250.
Frequency and type of DLT
Time frame: Until the end of study (approximately 24 months)
To determine the safety, tolerability, RP2D, or MTD if different, and dosing regimen of CBX-250.
Frequency, duration, and severity of TEAEs, TRAEs, AESIs, and SAEs
Time frame: Until the end of study (approximately 24 months)
To determine the safety, tolerability, RP2D, or MTD if different, and dosing regimen of CBX-250.
Frequency and severity of CRS AEs
Time frame: Until the end of study (approximately 24 months)
To determine the safety, tolerability, RP2D, or MTD if different, and dosing regimen of CBX-250.
Drug withdrawals, drug interruptions, or dose reductions due to adverse events
Time frame: Until the end of study (approximately 24 months)
To determine the safety, tolerability, RP2D, or MTD if different, and dosing regimen of CBX-250.
Incidence/shifts of clinical laboratory abnormalities
Time frame: Until the end of study (approximately 24 months)
AUC0-t (first dose and at steady state)
Area under the plasma concentration-time curve from time 0 to time of last measurable concentration (AUC0-t) of CBX-250 and relevant metabolites
Time frame: Approximately 1 year
Cmax
Maximum plasma concentration (Cmax) of CBX-250 and relevant metabolites
Time frame: Approximately 1 year
Tmax
Time to observed maximum plasma concentration of CBX-250 and relevant metabolites
Time frame: Approximately 1 year
ADAs
Incidence and severity of anti-drug antibodies
Time frame: Approximately 1 year
ORR
To assess the overall response rate (ORR) of CBX-250
Time frame: Approximately 1 year
CR+CRh rate in AML
To assess the complete remission (CR) and complete remission with partial hematologic recovery (CRh) rate
Time frame: Approximately 1 year
EFS
To assess the event free survival (EFS) of CBX-250
Time frame: Approximately 3 years
OS
To assess overall survival (OS) of CBX-250
Time frame: Approximately 3 years
DOR
To assess the duration of response (DOR) of CBX-250
Time frame: Approximately 3 years
CRc rate in AML
To assess the composite definition of complete remission (CRc) rate
Time frame: Approximately 3 years
Remission rate in HR-MDS
Remission rate as defined by Zeidan 2023: CR, CR equivalent, partial remission (PR), CRL (CRuni and CRbi), CRh, Hematologic improvement (HI)
Time frame: Approximately 1 year
PFS
To assess the progression free survival (PFS) of CBX-250
Time frame: Approximately 3 years
CR+CRh rate in HR-MDS
CR+ CR equivalent+ CRL + CRh
Time frame: Approximately 1 year
Transfusion independence
Any transfusion-free period lasting for at least 56 consecutive days, during which the participant is either on CBX-250 therapy or after cessation of CBX-250 therapy but prior to the start of new therapy
Time frame: Approximately 3 years
To assess the preliminary anti-leukemic activity of CBX-250 in participants with CMML
Proportion of participants achieving complete response (CR), complete cytogenetic remission, partial response (PR), marrow response, and clinical benefit according to the 2015 International Consortium Proposal of Uniform Response Criteria for Myelodysplastic Syndromes (MDS)/Myeloproliferative Neoplasms (MPN).
Time frame: Approximately 1 year.
To assess the preliminary anti-CML activity of CBX-250 in participants with CML
Proportion of participants achieving a ≥1-log10 reduction in BCR::ABL1 transcript levels from pre-treatment baseline at Week 12 (Day 169 ± 7 days), measured on the International Scale (IS) by centralized realtime quantitative PCR (RT-qPCR)
Time frame: Approximately 1 year.
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