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

NCT ID: NCT06687941

Last Updated: 2025-03-21

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

70 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-03-11

Study Completion Date

2028-05-31

Brief Summary

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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

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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.

Conditions

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Neoplasms Carcinoma, Hepatocellular Carcinoma, Non-Small-Cell Lung Liver Neoplasms

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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AST-201

Group Type EXPERIMENTAL

AST-201

Intervention Type 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.

Interventions

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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.

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

19 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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CHA University

OTHER

Sponsor Role collaborator

National Cancer Center, Korea

OTHER_GOV

Sponsor Role collaborator

Samsung Medical Center

OTHER

Sponsor Role collaborator

Severance Hospital

OTHER

Sponsor Role collaborator

Aptamer Sciences, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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National Cancer Center, Korea

Gyeonggi-do, , South Korea

Site Status RECRUITING

CHA Bundang Medical Center

Seongnam, , South Korea

Site Status RECRUITING

Severance Hospital

Seoul, , South Korea

Site Status RECRUITING

Samsung Medical Center

Seoul, , South Korea

Site Status RECRUITING

Countries

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South Korea

Central Contacts

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Aptamer Sciences Inc.

Role: CONTACT

+82-70-5067-4275

Other Identifiers

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AST-201-01

Identifier Type: -

Identifier Source: org_study_id

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