KAT-101 in Subjects With Hepatocellular Carcinoma (HCC)

NCT05603572 | Suspended Phase 1 DMC FDA Drug

• 1 other identifier: NLP-KAT-101
• Study Type: interventional
• Target: 148
• Locations: 1 country
• Sites: 3

Brief 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.

Conditions

Hepatocellular Carcinoma; Fibrolamellar Carcinoma

Outcome Measures

Primary Outcomes (1)

• 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

  24 months

Secondary Outcomes (6)

• To evaluate the safety and tolerability of KAT (oral, IT, and oral + IT) in subjects with HCC

  54 months

• To evaluate the preliminary anti-tumor activity of KAT for oral + IT administration

  54 months

• To assess maximum concentration (Cmax) of KAT (oral and oral + IT)

  54 months

• To assess median time to the maximum drug concentration (Tmax) of KAT (oral and oral + IT)

  54 months

• To assess half lives (T1/2) of KAT (oral and oral + IT)

  54 months

Study Arms (3)

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.

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.

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.

Drug: KAT-101; Drug: KAT-201

Interventions

KAT-101 DRUG

oral dosage form

Oral + IT experimental arm; Oral experimental arm

KAT-201 DRUG

IT dosage form

IT experimental arm; Oral + IT experimental arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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.

Sponsors & Collaborators

• Primocure Pharma: lead

Study Sites (3)

Kyungpook National University Hospital

Daegu, South Korea

Location

Samsung Medical Center

Seoul, South Korea

Location

Seoul National University Hospital

Seoul, South Korea

Location

MeSH Terms

Conditions

Carcinoma, Hepatocellular; Fibrolamellar hepatocellular carcinoma

Condition Hierarchy (Ancestors)

Adenocarcinoma; Carcinoma; Neoplasms, Glandular and Epithelial; Neoplasms by Histologic Type; Neoplasms; Liver Neoplasms; Digestive System Neoplasms; Neoplasms by Site; Digestive System Diseases; Liver Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: 1. Oral administration (KAT-101) 2. IT administration (KAT-201) 3. Oral administration (KAT-101) + IT administration (KAT-201)

Study Record Dates

• First Submitted: October 23, 2022
• First Posted: November 2, 2022
• Study Start: November 1, 2022
• Primary Completion (Estimated): November 1, 2027
• Study Completion (Estimated): November 1, 2027
• Last Updated: June 5, 2025

Record last verified: 2025-06

Data Sharing

• IPD Sharing: Will not share

Locations

KR (3)