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A Study to Evaluate the Tolerability, Safety, and PK of AST-201 in Patients With GPC3-positive Advanced Solid Tumors

A Study to Evaluate the Tolerability, Safety, and PK of AST-201 in Patients With GPC3-positive Advanced Solid Tumors

Recruiting
19 years and older
All
Phase 1

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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.

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.

Eligibility

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

Study details
    Neoplasms
    Carcinoma
    Hepatocellular
    Carcinoma
    Non-Small-Cell Lung
    Liver Neoplasms

NCT06687941

Aptamer Sciences, Inc.

27 August 2025

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