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NCT Number: NCT06687941

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

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.

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Key information

Conditions

Neoplasms Adenocarcinoma Bronchial Neoplasms Carcinoma Carcinoma, Bronchogenic Carcinoma, Hepatocellular Carcinoma, Non-Small-Cell Lung Digestive System Diseases Digestive System Neoplasms Liver Diseases Liver Neoplasms Lung Diseases Lung Neoplasms Neoplasm Metastasis Neoplasms by Histologic Type Neoplasms by Site Neoplasms, Glandular and Epithelial Neoplastic Processes Pathologic Processes Pathological Conditions, Signs and Symptoms Respiratory Tract Diseases Respiratory Tract Neoplasms Simpson-Golabi-Behmel syndrome Thoracic Neoplasms

Age range

19 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

National Cancer Center, Korea, Goyang-si, Gyeonggi-do, South Korea

Loading trial locations.

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

AST-201

Drug

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 outcomes

  1. Dose Limiting Toxicity (DLT)

    Time frame: 4 weeks

    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].

Secondary outcomes

  1. Pharmacokinetics (PK): Cmax

    Time frame: Cycle 1, Days 1-2 (cycle is 28 days)

    Maximum Plasma Concentration (Cmax)

  2. Pharmacokinetics (PK): Tmax

    Time frame: Cycle 1, Days 1-2 (cycle is 28 days)

    Time to Reach Maximum Plasma Concentration (Tmax)

  3. Pharmacokinetics (PK): AUC

    Time frame: Cycle 1, Days 1-2 (cycle is 28 days)

    Area Under the Curve (AUC)

  4. Pharmacokinetics (PK): Cl

    Time frame: Cycle 1, Days 1-2 (cycle is 28 days)

    Clearance Rate

  5. Pharmacokinetics (PK): t1/2

    Time frame: Cycle 1, Days 1-2 (cycle is 28 days)

    Half-Life (t1/2)

  6. Objective Response Rate (ORR)

    Time frame: Baseline through the end of each 28-day cycle, up to 6 months.

    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.

  7. Disease Control Rate (DCR)

    Time frame: Baseline through the end of each 28-day cycle, up to 6 months.

    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.

  8. Duration of Response (DOR)

    Time frame: Baseline through the end of each 28-day cycle, up to 6 months.

    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.

  9. Time to Progression (TTP)

    Time frame: Baseline through the end of each 28-day cycle, up to 6 months.

    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.

  10. Progression-Free Survival (PFS)

    Time frame: Baseline through the end of each 28-day cycle, up to 6 months.

    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.

  11. Overall Survival(OS)

    Time frame: Baseline through the end of each 28-day cycle, up to 6 months.

    Overall Survival(OS) is defined as the period from the initial administration of the IP to death.

Study contacts

Contact information is provided by the study sponsor or research team.

Aptamer Sciences Inc.

CONTACT

[email protected]

+82-70-5067-4275

Sponsors and collaborators

Lead sponsor

Aptamer Sciences, Inc.

Industry

Collaborators

  • CHA University
  • National Cancer Center, Korea
  • Samsung Medical Center
  • Severance Hospital

Registry information

Official study 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

Important dates

Study start
2025
Primary completion
2027
Study completion
2028
First posted
Nov 14, 2024
Registry last updated
Mar 27, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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