This is the first in human trial clinical study of AST-201 in patients with GPC3-positive advanced solid tumors. This study aims to evaluate the safety, tolerability, pharmacokinetic properties, and preliminary efficacy of AST-201 across various tumor types.
AST-201 is a novel aptamer drug conjugate (ApDC) investigational agent with demonstrated preclinical efficacy in GPC3-positive tumor models. This Phase 1 clinical study aims to investigate the safety, tolerability, and preliminary efficacy of AST-201, targeting GPC3-positive advanced solid tumors. The study consists of two parts: Phase 1a and Phase 1b. In Phase 1a, AST-201 will be administered in a dose escalating manner across cohorts of patients to determine the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D). In this dose-escalation phase, patients will receive AST-201 as a single agent, with safety, tolerability, and pharmacokinetic (PK) profiles assessed. In Phase 1b, patients will receive AST-201 at the RP2D across specific GPC3-positive tumor types to further explore safety and efficacy. This expansion phase focuses on assessing anti-tumor efficacy and overall safety in a broader patient population. Data collected from this study will support future clinical development of AST-201 in GPC3-positive advanced solid tumors.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
70
AST-201 is administered intravenously on Days 1, 8, and 15 of each 28-day cycle, followed by a one-week rest period. Dosing is repeated until DLT or disease progression is occurred.
National Cancer Center, Korea
Goyang-si, Gyeonggi-do, South Korea
RECRUITINGCHA Bundang Medical Center
Seongnam, South Korea
RECRUITINGSeverance Hospital
Seoul, South Korea
RECRUITINGDose Limiting Toxicity (DLT)
Dose-limiting toxicity (DLT) is defined as any treatment-related Grade 3 or higher adverse event, or other clinically significant toxicity occurring during the first cycle (4 weeks), that meets the protocol-defined criteria for dose limitation, based on the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE), version \[5.0\].
Time frame: 4 weeks
Pharmacokinetics (PK): Cmax
Maximum Plasma Concentration (Cmax)
Time frame: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): Tmax
Time to Reach Maximum Plasma Concentration (Tmax)
Time frame: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): AUC
Area Under the Curve (AUC)
Time frame: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): Cl
Clearance Rate
Time frame: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): t1/2
Half-Life (t1/2)
Time frame: Cycle 1, Days 1-2 (cycle is 28 days)
Objective Response Rate (ORR)
Objective Response Rate (ORR) is defined as the proportion of subjects with the best overall response (BOR) assessed as complete response (CR) and partial response (PR). ORR assessed by the Investigator and evaluated according to RECIST 1.1 criteria.
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
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Samsung Medical Center
Seoul, South Korea
RECRUITINGDisease Control Rate (DCR)
Disease Control Rate (DCR) is defined as the proportion of subjects with the BOR assessed as CR, PR, or stable disease (SD). DCR assessed by the Investigator and evaluated according to RECIST 1.1 criteria.
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
Duration of Response (DOR)
Duration of Response (DOR) is defined as the period from the initial assessment date confirming CR or PR to disease progression (PD) or death. DOR assessed by the Investigator and evaluated according to RECIST 1.1 criteria.
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
Time to Progression (TTP)
Time to Progression (TTP) is defined as the period from the initial administration of the investigational product (IP) to disease progression (PD). TTP assessed by the Investigator and evaluated according to RECIST 1.1 criteria.
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
Progression-Free Survival (PFS)
Progression-Free Survival (PFS) is defined as the period from the initial administration of the IP to disease progression (PD) or death. PFS assessed by the Investigator and evaluated according to RECIST 1.1 criteria.
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
Overall Survival(OS)
Overall Survival(OS) is defined as the period from the initial administration of the IP to death.
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.