A Study to Evaluate the Tolerability, Safety, and PK of AST-201 in Patients With GPC3-positive Advanced Solid Tumors
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: AST-201.
- Who it may be relevant to
- Registry conditions: Neoplasms, Carcinoma, Hepatocellular, Carcinoma, Non-Small-Cell Lung, Liver Neoplasms. Basic parameters: from 19 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- South Korea
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Multi-center, Open-label, Dose Escalation and Expansion, Phase 1 Study to Evaluate the Tolerability, Safety and Pharmacokinetics of AST-201 in Patients With GPC3-positive Advanced Solid Tumors
Overview
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.
Detailed description
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.
Interventions
- Drug AST-201
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.
Primary outcome measures
- Dose Limiting Toxicity (DLT) [Time frame: 4 weeks]
Secondary outcome measures (11)
- Pharmacokinetics (PK): Cmax [Time frame: Cycle 1, Days 1-2 (cycle is 28 days)]
- Pharmacokinetics (PK): Tmax [Time frame: Cycle 1, Days 1-2 (cycle is 28 days)]
- Pharmacokinetics (PK): AUC [Time frame: Cycle 1, Days 1-2 (cycle is 28 days)]
- Pharmacokinetics (PK): Cl [Time frame: Cycle 1, Days 1-2 (cycle is 28 days)]
- Pharmacokinetics (PK): t1/2 [Time frame: Cycle 1, Days 1-2 (cycle is 28 days)]
- Objective Response Rate (ORR) [Time frame: Baseline through the end of each 28-day cycle, up to 6 months.]
- Disease Control Rate (DCR) [Time frame: Baseline through the end of each 28-day cycle, up to 6 months.]
- Duration of Response (DOR) [Time frame: Baseline through the end of each 28-day cycle, up to 6 months.]
- Time to Progression (TTP) [Time frame: Baseline through the end of each 28-day cycle, up to 6 months.]
- Progression-Free Survival (PFS) [Time frame: Baseline through the end of each 28-day cycle, up to 6 months.]
- Overall Survival(OS) [Time frame: Baseline through the end of each 28-day cycle, up to 6 months.]
Eligibility criteria
Inclusion criteria
- Male and female aged ≥19 years
- Histologically and/or cytologically diagnosed as the advanced recurrent solid tumor
- GPC3-positive confirmed by IHC test
- At least 1 measurable or non-measurable but evaluable lesion as defined per RECIST v1.1 (modified RECIST for hepatocellular carcinoma)
- ECOG performance status of 0 or 1
- Life expectancy at least 12 weeks
- Adequate hematologic, hepatic, renal, and heart/coagulation function
- Child-Pugh Class of A for HCC
Exclusion criteria
- Subjects with ischemic heart disease
- Subjects with anti-tumor treatment within 4 weeks
- Subjects with comorbidities such as uncontrolled hypertension, heart failure, etc.
- Pregnant or potentially pregnant and lactating woman
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
South Korea · 4 centers
- National Cancer Center, Korea — Goyang-si
- CHA Bundang Medical Center — Seongnam
- Severance Hospital — Seoul
- Samsung Medical Center — Seoul
Identifiers
NCT: NCT06687941 · AST-201-01