NCT06687941

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
70

participants targeted

Target at P75+ for phase_1

Timeline
24mo left

Started Mar 2025

Typical duration for phase_1

Geographic Reach
1 country

4 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress37%
Mar 2025May 2028

First Submitted

Initial submission to the registry

November 5, 2024

Completed
9 days until next milestone

First Posted

Study publicly available on registry

November 14, 2024

Completed
4 months until next milestone

Study Start

First participant enrolled

March 11, 2025

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2027

Expected
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2028

Last Updated

March 27, 2026

Status Verified

March 1, 2026

Enrollment Period

2.7 years

First QC Date

November 5, 2024

Last Update Submit

March 23, 2026

Conditions

NeoplasmsCarcinoma, HepatocellularCarcinoma, Non-Small-Cell LungLiver Neoplasms

Keywords

Glypican-3 (GPC3)GPC3-positive advanced solid tumorsAST-201Aptamer-drug conjugateHepatocellular carcinoma (HCC)Advanced solid tumorsMetastaticDose Escalation and Expansion

Outcome Measures

Primary Outcomes (1)

  • Dose Limiting Toxicity (DLT)

    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 Outcomes (11)

  • Pharmacokinetics (PK): Cmax

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

  • Pharmacokinetics (PK): Tmax

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

  • Pharmacokinetics (PK): AUC

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

  • Pharmacokinetics (PK): Cl

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

  • Pharmacokinetics (PK): t1/2

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

  • +6 more secondary outcomes

Study Arms (1)

AST-201

EXPERIMENTAL

Participants receive AST-201 administered intravenously according to the study dosing schedule.

Drug: AST-201

Interventions

AST-201DRUG

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.

AST-201

Eligibility Criteria

Age19 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

National Cancer Center, Korea

Goyang-si, Gyeonggi-do, 10408, South Korea

RECRUITING

CHA Bundang Medical Center

Seongnam, 13609, South Korea

RECRUITING

Severance Hospital

Seoul, 03722, South Korea

RECRUITING

Samsung Medical Center

Seoul, 06351, South Korea

RECRUITING

MeSH Terms

Conditions

NeoplasmsCarcinoma, HepatocellularCarcinoma, Non-Small-Cell LungLiver NeoplasmsSimpson-Golabi-Behmel syndromeNeoplasm Metastasis

Condition Hierarchy (Ancestors)

AdenocarcinomaCarcinomaNeoplasms, Glandular and EpithelialNeoplasms by Histologic TypeDigestive System NeoplasmsNeoplasms by SiteDigestive System DiseasesLiver DiseasesCarcinoma, BronchogenicBronchial NeoplasmsLung NeoplasmsRespiratory Tract NeoplasmsThoracic NeoplasmsLung DiseasesRespiratory Tract DiseasesNeoplastic ProcessesPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • David Lee

    Aptamer Sciences, Inc.

    STUDY DIRECTOR

Central Study Contacts

Aptamer Sciences Inc.

CONTACT

+82-70-5067-4275kjkim@aptsci.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

November 5, 2024

First Posted

November 14, 2024

Study Start

March 11, 2025

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

May 1, 2028

Last Updated

March 27, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share

Locations

KR(4)