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

Phase 1

Recruiting

• Conditions: Neoplasms; Carcinoma, Hepatocellular; Carcinoma, Non-Small-Cell Lung; Liver Neoplasms
• Interventions: Drug: AST-201

• Registration Number: NCT06687941
• Lead Sponsor: Aptamer Sciences, Inc.

Brief Summary

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.

Study Timeline

• Study First Submitted: 2024-11-05
• Study First Submitted QC: 2024-11-12
• Study First Posted: 2024-11-14
• Status Verified: 2025-03
• Study Start: 2025-03-11
• Last Update Submitted: 2025-03-19
• Last Update Posted: 2025-03-21
• Primary Completion: 2027-12
• Study Completion: 2028-05

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 70

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
AST-201	AST-201	-

Primary Outcome Measures

Name	Time	Method
Dose Limiting Toxicity (DLT)	4 weeks

Secondary Outcome Measures

Name	Time	Method
Pharmacokinetics (PK)	Cycle 1, Days 1-2 (cycle is 28 days)	Half-Life (t1/2)
Preliminary Antitumor Efficacy	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.

Trial Locations

Locations (4)

National Cancer Center, Korea; 🇰🇷 Gyeonggi, Korea, Republic of

CHA Bundang Medical Center; 🇰🇷 Seongnam, Korea, Republic of

Severance Hospital; 🇰🇷 Seoul, Korea, Republic of

Samsung Medical Center; 🇰🇷 Seoul, Korea, Republic of