NeoplasmsPhase 1NCT06687941
A Study to Evaluate the Tolerability, Safety, and PK of AST-201 in Patients With GPC3-positive Advanced Solid Tumors
A Phase 1 study testing AST-201 in people with Neoplasms and Carcinoma, Hepatocellular, run by Aptamer Sciences, Inc. at 4 sites in South Korea.
Trial 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. Read more about this study
- Who is it for?
- Ages 19 years and older
- Conditions the study is for
- Neoplasms, Carcinoma, Hepatocellular, Carcinoma, Non-Small-Cell Lung
- What is being tested?
- AST-201
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 4 sitesSouth Korea
- Study dates
- 11 Mar 2025to May 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 19 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
South Korea4 sites, 4 recruiting
- Recruiting
National Cancer Center, Korea
Goyang-si, Gyeonggi-do, 10408
Sang Myung Woo, MD, PhD
Bo Hyun Kim, MD, PhD
- Recruiting
CHA Bundang Medical Center
Seongnam, 13609
Hong Jae Chon, MD, PhD
- Recruiting
Severance Hospital
Seoul, 03722
Hye Ryun Kim, MD, PhD.
- Recruiting
Samsung Medical Center
Seoul, 06351
Jung Yong Hong, MD, PhD
About this study
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
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.
Read the 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.
- Being tested
- AST-201
- Keywords
- Glypican-3 (GPC3), GPC3-positive advanced solid tumors, AST-201, Aptamer-drug conjugate, Hepatocellular carcinoma (HCC), Advanced solid tumors, Metastatic, Dose Escalation and Expansion
- Medical subject headings
- Neoplasms, Carcinoma, Hepatocellular, Carcinoma, Non-Small-Cell Lung, Liver Neoplasms, Simpson-Golabi-Behmel syndrome, Neoplasm Metastasis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| AST-201 | Experimental | Participants receive AST-201 administered intravenously according to the study dosing schedule. Receives: Drug: AST-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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].
Measured: 4 weeks
Secondary outcomes (11)
Pharmacokinetics (PK): Cmax
Maximum Plasma Concentration (Cmax)
Measured: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): Tmax
Time to Reach Maximum Plasma Concentration (Tmax)
Measured: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): AUC
Area Under the Curve (AUC)
Measured: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): Cl
Clearance Rate
Measured: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): t1/2
Half-Life (t1/2)
Measured: Cycle 1, Days 1-2 (cycle is 28 days)
Objective Response Rate (ORR)
Objective Response Rate (ORR) is defined as the proportion of subjects with the best overall response (BOR) assessed as complete response (CR) and partial response (PR). ORR assessed by the Investigator and evaluated according to RECIST 1.1 criteria.
Measured: Baseline through the end of each 28-day cycle, up to 6 months.
Disease Control Rate (DCR)
Disease Control Rate (DCR) is defined as the proportion of subjects with the BOR assessed as CR, PR, or stable disease (SD). DCR assessed by the Investigator and evaluated according to RECIST 1.1 criteria.
Measured: Baseline through the end of each 28-day cycle, up to 6 months.
Duration of Response (DOR)
Duration of Response (DOR) is defined as the period from the initial assessment date confirming CR or PR to disease progression (PD) or death. DOR assessed by the Investigator and evaluated according to RECIST 1.1 criteria.
Measured: Baseline through the end of each 28-day cycle, up to 6 months.
Time to Progression (TTP)
Time to Progression (TTP) is defined as the period from the initial administration of the investigational product (IP) to disease progression (PD). TTP assessed by the Investigator and evaluated according to RECIST 1.1 criteria.
Measured: Baseline through the end of each 28-day cycle, up to 6 months.
Progression-Free Survival (PFS)
Progression-Free Survival (PFS) is defined as the period from the initial administration of the IP to disease progression (PD) or death. PFS assessed by the Investigator and evaluated according to RECIST 1.1 criteria.
Measured: Baseline through the end of each 28-day cycle, up to 6 months.
Overall Survival(OS)
Overall Survival(OS) is defined as the period from the initial administration of the IP to death.
Measured: Baseline through the end of each 28-day cycle, up to 6 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Nov 2024
- Study start11 Mar 2025
- Primary completionDec 2027 (estimated)
- Study completionMay 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for NeoplasmsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Aptamer Sciences, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- CHA University, National Cancer Center, Korea, Samsung Medical Center, Severance Hospital
- Responsible party
- Sponsor
- Investigators
- David Lee, Study director, Aptamer Sciences, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06687941
- Sponsor's protocol ID
- AST-201-01
- Status last verified
- Mar 2026
- First submitted
- 5 Nov 2024
- Last updated
- 27 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06687941
- Acronym / sponsor ID
- AST-201-01
- Keywords
- Glypican-3 (GPC3), GPC3-positive advanced solid tumors, AST-201, Aptamer-drug conjugate, Hepatocellular carcinoma (HCC), Advanced solid tumors, Metastatic, Dose Escalation and Expansion
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Aptamer Sciences, Inc.Industry (pharma, biotech, devices)
- Collaborators
- CHA University, National Cancer Center, Korea, Samsung Medical Center, Severance Hospital
- Study start
- 11 Mar 2025 (actual)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- May 2028 (estimated)
- First posted
- 14 Nov 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| AST-201 | Experimental | Drug: AST-201Participants receive AST-201 administered intravenously according to the study dosing schedule. |
Who may qualify
- Ages
- 19 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AST-201AST-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. | Drug | AST-201 |
Primary outcome
Dose Limiting Toxicity (DLT)
Time frame: 4 weeks
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].
11 secondary outcomes
Pharmacokinetics (PK): Cmax
Time frame: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): Tmax
Time frame: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): AUC
Time frame: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): Cl
Time frame: Cycle 1, Days 1-2 (cycle is 28 days)
Pharmacokinetics (PK): t1/2
Time frame: Cycle 1, Days 1-2 (cycle is 28 days)
Objective Response Rate (ORR)
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
Disease Control Rate (DCR)
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
Duration of Response (DOR)
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
Time to Progression (TTP)
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
Progression-Free Survival (PFS)
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
Overall Survival(OS)
Time frame: Baseline through the end of each 28-day cycle, up to 6 months.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
David Lee study director
Aptamer Sciences, Inc.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing AST-201 in people with Neoplasms and Carcinoma, Hepatocellular, run by Aptamer Sciences, Inc. at 4 sites in South Korea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 27 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in South Korea. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Aptamer Sciences Inc. (+82-70-5067-4275, kjkim@aptsci.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Dose Limiting Toxicity (DLT) (time frame: 4 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Mar 2026.