Hepatocellular CarcinomaPhase 1/2NCT05603572
KAT-101 in Subjects With Hepatocellular Carcinoma (HCC)
A Phase 1/2 study testing KAT-101 and KAT-201 in people with Hepatocellular Carcinoma and Fibrolamellar Carcinoma, run by Primocure Pharma at 1 site in South Korea.
Trial summary
NLP-KAT-101 is a Phase 1/2a dose escalation and expansion study to investigate the safety, tolerability, PK, and preliminary efficacy of oral + intratumoral (IT) KAT in subjects with HCC. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hepatocellular Carcinoma, Fibrolamellar Carcinoma
- What is being tested?
- KAT-101 and KAT-201
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteSouth Korea
- Study dates
- 1 Nov 2022to 26 Dec 2022
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Confirmed HCC not amenable to surgical resection or curative-intent locoregional ablative treatments and who are not eligible for liver transplantation.
- Systemic treatment-naive for unresectable locally advanced or metastatic HCC. In addition, have progressed on, refused or were intolerant to sorafenib, lenvatinib, or atezolizumab in combination with bevacizumab. A maximum of 2 prior lines of systemic therapy (including chemotherapy or targeted therapy, not including locoregional therapy) will be allowed.
- At least one measurable lesion based on RECIST 1.1
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2
- Adequate organ function
Who cannot join
- Prior to the first administration of the study treatment:
- Major surgery within 28 days
- Radiotherapy within 14 days including palliative radiation
- Use of steroids (except for topical agents) within 14 days
- Chemotherapy within 3 weeks (6 weeks for nitrosourea compounds)
- Prior treatment with biologic agents, including hormone therapy, within the last 3 weeks, or at least 5 half-lives, whichever is shorter
Show all 20Show fewer
- Tumor infiltration in the portal vein, hepatic veins or inferior vena cava that completely blocks circulation in liver
- Treatment with another investigational product within 4 weeks prior to screening or for which 5 half-lives have not elapsed, whichever is longer
- Uncontrolled central nervous system (CNS) metastasis
- Any clinically significant abnormal intestinal findings that may interfere with the investigational product
- Severe cardiac disorders or subjects with comorbidities of other serious internal disorders on investigator's judgment
- QTcF > 450 msec or congenital long QT syndrome
- Suspected serious infectious diseases, intestinal paralysis, bowel obstruction, interstitial pneumonia, or pulmonary fibrosis
- Serious underlying medical or psychiatric condition, dementia or altered mental status that would impair the ability to understand informed consent, contraindicate participation in the study or confound the results of the study
- Known human immunodeficiency virus (HIV) infection or chronic or active hepatitis B virus (HBV) hepatitis C virus (HCV). Subjects with HCV who have a documented cure (undetectable HCV ribonucleic acid (RNA) 24 weeks after the end of treatment) may be enrolled.
- Severe physical or mental trauma that results from injury or a wound(s).
- Any condition or non-removable device contraindicated for MRI examination
- Pregnant women or nursing mothers.
- Women of childbearing potential (WOCBP) who are unwilling to use a medically acceptable method of birth control during the study until 185 days after the last dose of study treatment
- Men with partners of childbearing potential who are unwilling to use condoms in combination with a second medically acceptable method of contraception during the study until 95 days after the last dose of study treatment.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Multi-center, Open-label, Phase 1/2a Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of KAT-101 in Subjects With HCC
NLP-KAT-101 is a Phase 1/2a dose escalation and expansion study to investigate the safety, tolerability, PK, and preliminary efficacy of oral + intratumoral (IT) KAT in subjects with HCC.
Read the detailed description
Phase 1 will identify the optimal dose for oral alone, IT alone and the recommended Phase 2 dose (RP2D) dose for oral + IT together. Once the RP2D is identified, additional subjects will be enrolled into Phase 2a (dose-expansion) to further investigate the efficacy and safety of oral + IT KAT at the RP2D.
- Conditions
- Hepatocellular Carcinoma, Fibrolamellar Carcinoma
- Being tested
- KAT-101, KAT-201
- Medical subject headings
- Carcinoma, Hepatocellular, Fibrolamellar hepatocellular carcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KAT-101Drug
oral dosage form
KAT-201Drug
IT dosage form
| Study group | Role | What they receive |
|---|---|---|
| Oral experimental arm | Experimental | Oral administration (KAT-101) taken once per day for 4 consecutive days out of 7 (4 days on / 3 days off weekly). Each cycle is 28 days. Treatment will continue for up to 12 cycles until progressive disease (PD), unacceptable toxicity, or any reason for discontinuing its administration, whichever occurs first. Receives: Drug: KAT-101 |
| IT experimental arm | Experimental | IT administration (KAT-201) will be injected via percutaneous IT injection with ultrasound and/or computed tomography (CT) guidance once a week (on Day 1 weekly). Each cycle is 28 days. Treatment will continue for up to 2 cycles until PD, unacceptable toxicity, or any reason for discontinuing its administration, whichever occurs first. Receives: Drug: KAT-201 |
| Oral + IT experimental arm | Experimental | Once optimal oral and IT dose are determined, oral + IT will be administered as follows: oral administration (KAT-101) will be taken once per day for 4 consecutive days out of 7 (4 days on / 3 days off weekly). Treatment will continue for up to 12 cycles (each cycle 28 days). IT administration (KAT-201) will be injected via percutaneous IT injection with ultrasound and/or CT guidance once a week (on Day 1 weekly). Treatment will continue for up to 2 cycles (each cycle 28 days) until PD, unacceptable toxicity, or any reason for discontinuing its administration, whichever occurs first. Receives: Drug: KAT-101; Drug: KAT-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To determine the Recommended Phase 2 Dose (RP2D) for oral + IT administration
RP2D is defined as the dose at which dose escalation (oral + IT) ceases
Measured: 24 months
Secondary outcomes (6)
To evaluate the safety and tolerability of KAT (oral, IT, and oral + IT) in subjects with HCC
Measured as the number of AEs per CTCAE and changes in laboratory values compared to baseline.
Measured: 54 months
To evaluate the preliminary anti-tumor activity of KAT for oral + IT administration
Overall response rate (ORR) is defined as the proportion of subjects with a best overall response (BOR) by Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) and/or mRECIST; disease control rate (DCR), defined as the proportion of subjects with a BOR of CR, PR, or stable disease (SD); duration of response (DOR), defined as the length of time from the time of response (CR or PR) to the time of PD or death; progression-free survival (PFS), defined as the time from the date of treatment initiation to PD or death; overall survival (OS), defined as the length of time from the date of treatment initiation to death from any cause.
Measured: 54 months
To assess maximum concentration (Cmax) of KAT (oral and oral + IT)
Blood samples will be drawn to determine Cmax of KAT
Measured: 54 months
To assess median time to the maximum drug concentration (Tmax) of KAT (oral and oral + IT)
Blood samples will be drawn to determine Tmax of KAT
Measured: 54 months
To assess half lives (T1/2) of KAT (oral and oral + IT)
Blood samples will be drawn to determine T1/2 of KAT
Measured: 54 months
To assess area under the curve (AUC) of KAT (oral and oral + IT)
Blood samples will be drawn to determine AUC of KAT
Measured: 54 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Nov 2022
- Study start1 Nov 2022
- Primary completion26 Dec 2022
- Study completion26 Dec 2022
Why it stopped: discontinued enrollment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hepatocellular CarcinomaStudies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- 1. Oral administration (KAT-101) 2. IT administration (KAT-201) 3. Oral administration (KAT-101) + IT administration (KAT-201)
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Primocure Pharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05603572
- Sponsor's protocol ID
- NLP-KAT-101
- Status last verified
- Oct 2026
- First submitted
- 23 Oct 2022
- Last updated
- 27 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open-label, Phase 1/2a Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of KAT-101 in Subjects With HCC
- ClinicalTrials.gov ID
- NCT05603572
- Acronym / sponsor ID
- NLP-KAT-101
- Conditions
- Hepatocellular Carcinoma, Fibrolamellar Carcinoma
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: discontinued enrollment
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Primocure PharmaIndustry (pharma, biotech, devices)
- Study start
- 1 Nov 2022 (actual)
- Primary completion
- 26 Dec 2022 (actual)
- Study completion
- 26 Dec 2022 (actual)
- First posted
- 2 Nov 2022 (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.
NLP-KAT-101 is a Phase 1/2a dose escalation and expansion study to investigate the safety, tolerability, PK, and preliminary efficacy of oral + intratumoral (IT) KAT in subjects with HCC.
Detailed description
Phase 1 will identify the optimal dose for oral alone, IT alone and the recommended Phase 2 dose (RP2D) dose for oral + IT together. Once the RP2D is identified, additional subjects will be enrolled into Phase 2a (dose-expansion) to further investigate the efficacy and safety of oral + IT KAT at the RP2D.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- 1. Oral administration (KAT-101) 2. IT administration (KAT-201) 3. Oral administration (KAT-101) + IT administration (KAT-201)
| Arm | Type | Receives |
|---|---|---|
| Oral experimental arm | Experimental | Drug: KAT-101Oral administration (KAT-101) taken once per day for 4 consecutive days out of 7 (4 days on / 3 days off weekly). Each cycle is 28 days. Treatment will continue for up to 12 cycles until progressive disease (PD), unacceptable toxicity, or any reason for discontinuing its administration, whichever occurs first. |
| IT experimental arm | Experimental | Drug: KAT-201IT administration (KAT-201) will be injected via percutaneous IT injection with ultrasound and/or computed tomography (CT) guidance once a week (on Day 1 weekly). Each cycle is 28 days. Treatment will continue for up to 2 cycles until PD, unacceptable toxicity, or any reason for discontinuing its administration, whichever occurs first. |
| Oral + IT experimental arm | Experimental | Drug: KAT-101, Drug: KAT-201Once optimal oral and IT dose are determined, oral + IT will be administered as follows: oral administration (KAT-101) will be taken once per day for 4 consecutive days out of 7 (4 days on / 3 days off weekly). Treatment will continue for up to 12 cycles (each cycle 28 days). IT administration (KAT-201) will be injected via percutaneous IT injection with ultrasound and/or CT guidance once a week (on Day 1 weekly). Treatment will continue for up to 2 cycles (each cycle 28 days) until PD, unacceptable toxicity, or any reason for discontinuing its administration, whichever occurs first. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Confirmed HCC not amenable to surgical resection or curative-intent locoregional ablative treatments and who are not eligible for liver transplantation.
- Systemic treatment-naive for unresectable locally advanced or metastatic HCC. In addition, have progressed on, refused or were intolerant to sorafenib, lenvatinib, or atezolizumab in combination with bevacizumab. A maximum of 2 prior lines of systemic therapy (including chemotherapy or targeted therapy, not including locoregional therapy) will be allowed.
- At least one measurable lesion based on RECIST 1.1
- Eastern Cooperative Oncology Group (ECOG) performance status 0-2
- Adequate organ function
Key exclusion criteria (20 listed)
- Prior to the first administration of the study treatment:
- Any clinically significant abnormal intestinal findings that may interfere with the investigational product
- Severe cardiac disorders or subjects with comorbidities of other serious internal disorders on investigator's judgment
- QTcF > 450 msec or congenital long QT syndrome
- Suspected serious infectious diseases, intestinal paralysis, bowel obstruction, interstitial pneumonia, or pulmonary fibrosis
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 |
|---|---|---|
| KAT-101oral dosage form | Drug | Oral + IT experimental arm, Oral experimental arm |
| KAT-201IT dosage form | Drug | IT experimental arm, Oral + IT experimental arm |
Primary outcome
To determine the Recommended Phase 2 Dose (RP2D) for oral + IT administration
Time frame: 24 months
RP2D is defined as the dose at which dose escalation (oral + IT) ceases
6 secondary outcomes
To evaluate the safety and tolerability of KAT (oral, IT, and oral + IT) in subjects with HCC
Time frame: 54 months
To evaluate the preliminary anti-tumor activity of KAT for oral + IT administration
Time frame: 54 months
To assess maximum concentration (Cmax) of KAT (oral and oral + IT)
Time frame: 54 months
To assess median time to the maximum drug concentration (Tmax) of KAT (oral and oral + IT)
Time frame: 54 months
To assess half lives (T1/2) of KAT (oral and oral + IT)
Time frame: 54 months
To assess area under the curve (AUC) of KAT (oral and oral + IT)
Time frame: 54 months
Results and safety
The study was stopped early (reason given: discontinued enrollment). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Oct 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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing KAT-101 and KAT-201 in people with Hepatocellular Carcinoma and Fibrolamellar Carcinoma, run by Primocure Pharma at 1 site 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 5 inclusion and 20 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: stopped early, as of the sponsor's last update on 27 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: discontinued enrollment). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To determine the Recommended Phase 2 Dose (RP2D) for oral + IT administration (time frame: 24 months). 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 Oct 2026.