RecruitingRecruiting
A Study to Evaluate the Tolerability, Safety, and PK of AST-201 in Patients With GPC3-positive Advanced Solid Tumors
NCT06687941 · Aptamer Sciences, Inc.
In plain English
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Official title
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
About this study
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.
Eligibility 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
Study design
Enrollment target: 70 participants
Allocation: na
Masking: none
Age groups: adult, older_adult
Timeline
Starts: 2025-03-11
Estimated completion: 2028-05
Last updated: 2026-03-27
Interventions
Drug: AST-201
Primary outcomes
- • Dose Limiting Toxicity (DLT) (4 weeks)
Sponsor
Aptamer Sciences, Inc. · industry
With: CHA University, National Cancer Center, Korea, Samsung Medical Center, Severance Hospital
Contacts & investigators
ContactAptamer Sciences Inc. · contact · kjkim@aptsci.com · +82-70-5067-4275
InvestigatorDavid Lee · study_director, Aptamer Sciences, Inc.
All locations (4)
National Cancer Center, KoreaRecruiting
Goyang-si, Gyeonggi-do, South Korea
CHA Bundang Medical CenterRecruiting
Seongnam, South Korea
Severance HospitalRecruiting
Seoul, South Korea
Samsung Medical CenterRecruiting
Seoul, South Korea