Evaluating AST-201 for GPC3-positive advanced solid tumors

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

Phase 1 Interventional Aptamer Sciences, Inc. · NCT06687941

This study is testing a new treatment called AST-201 to see if it can help people with advanced solid tumors that have a specific marker called GPC3.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment70 (estimated)
Ages19 Years and up
SexAll
SponsorAptamer Sciences, Inc. Industry-sponsored
Locations4 sites (Goyang-si, Gyeonggi-do and 3 other locations)
Trial IDNCT06687941 on ClinicalTrials.gov

What this trial studies

This clinical study is the first in humans to evaluate AST-201, a novel aptamer-drug conjugate, in patients with GPC3-positive advanced solid tumors. The study is divided into two phases: Phase 1a focuses on determining the maximum tolerated dose and pharmacokinetic properties of AST-201, while Phase 1b assesses its safety and preliminary efficacy across specific tumor types. Patients will receive AST-201 as a single agent, and the study aims to gather data to support future clinical development of this treatment for GPC3-positive tumors.

Who should consider this trial

Good fit: Ideal candidates are adults aged 19 and older with GPC3-positive advanced recurrent solid tumors.

Not a fit: Patients with ischemic heart disease or those who have received anti-tumor treatment within the last four weeks may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced solid tumors that express GPC3.

How similar studies have performed: While this approach is novel, preclinical studies have shown promising efficacy of AST-201 in GPC3-positive tumor models.

Eligibility criteria

Show full inclusion / exclusion criteria
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

Where this trial is running

Goyang-si, Gyeonggi-do and 3 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions NeoplasmsCarcinoma, HepatocellularCarcinoma, Non-Small-Cell LungLiver NeoplasmsGlypican-3GPC3-positive advanced solid tumorsAST-201Aptamer-drug conjugate
Last reviewed 2026-06-15 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.