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

March 23, 2026 updated by: Aptamer Sciences, Inc.

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

70

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Aptamer Sciences Inc.
  • Phone Number: +82-70-5067-4275
  • Email: kjkim@aptsci.com

Study Locations

  • South Korea
      • Seongnam, South Korea, 13609
        • Recruiting
        • CHA Bundang Medical Center
        • Principal Investigator:
          • Hong Jae Chon, MD, PhD
      • Seoul, South Korea, 06351
        • Recruiting
        • Samsung Medical Center
        • Principal Investigator:
          • Jung Yong Hong, MD, PhD
      • Seoul, South Korea, 03722
        • Recruiting
        • Severance Hospital
        • Principal Investigator:
          • Hye Ryun Kim, MD, PhD.
    • Gyeonggi-do
      • Goyang-si, Gyeonggi-do, South Korea, 10408
        • Recruiting
        • National Cancer Center, Korea
        • Principal Investigator:
          • Sang Myung Woo, MD, PhD
        • Sub-Investigator:
          • Bo Hyun Kim, MD, PhD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: AST-201
Participants receive AST-201 administered intravenously according to the study dosing schedule.
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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].
4 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Pharmacokinetics (PK): Cmax
Time Frame: Cycle 1, Days 1-2 (cycle is 28 days)
Maximum Plasma Concentration (Cmax)
Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): Tmax
Time Frame: Cycle 1, Days 1-2 (cycle is 28 days)
Time to Reach Maximum Plasma Concentration (Tmax)
Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): AUC
Time Frame: Cycle 1, Days 1-2 (cycle is 28 days)
Area Under the Curve (AUC)
Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): Cl
Time Frame: Cycle 1, Days 1-2 (cycle is 28 days)
Clearance Rate
Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): t1/2
Time Frame: Cycle 1, Days 1-2 (cycle is 28 days)
Half-Life (t1/2)
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
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.
Overall Survival(OS) is defined as the period from the initial administration of the IP to death.
Baseline through the end of each 28-day cycle, up to 6 months.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Aptamer Sciences, Inc.

Collaborators

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

Investigators

  • Study Director: David Lee, Aptamer Sciences, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 11, 2025

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

May 1, 2028

Study Registration Dates

First Submitted

November 5, 2024

First Submitted That Met QC Criteria

November 12, 2024

First Posted (Actual)

November 14, 2024

Study Record Updates

Last Update Posted (Actual)

March 27, 2026

Last Update Submitted That Met QC Criteria

March 23, 2026

Last Verified

March 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AST-201-01

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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