Efficacy and Safety of Pracytarabine Versus Regorafenib in the Treatment of Patients With Hepatocellular Carcinoma

Xi'an Xintong Pharmaceutical Research

Status and phase: Active, not recruiting; Phase 3; Phase 2

Conditions: HCC

Treatments: Drug: Pracytarabine; Drug: regorafenib

Study type: Interventional

Funder types: Other

Identifiers

NCT07300488

MB07133-2025-01

Details and patient eligibility

About

This is a Phase 2/3 study evaluating the efficacy and safety of Pracytarabine versus regorafenib in patients with advanced hepatocellular carcinoma (HCC) who have experienced treatment failure with standard systemic therapies involving targeted drugs and immune checkpoint inhibitors, or dual immune checkpoint inhibitors.

Enrollment: 60 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Aged 18-75 years, regardless of gender.
2. Diagnosed with HCC either by histopathological and/or cytological examination meeting pathological diagnostic criteria, or in line with the clinical diagnostic criteria of the aforementioned guidelines.
3. Unresectable or metastatic HCC, and have experienced treatment failure with targeted drugs and immune checkpoint inhibitors.
4. Child-Pugh liver function score: Class A/B (≤7 points).
5. Expected survival time ≥ 3 months.
6. ECOG performance status 0 or 1.
7. Barcelona Clinic Liver Cancer (BCLC) stage B or C.
8. No severe involvement of the portal vein, and no invasion of the hepatic vein, superior vena cava, or inferior vena cava.

Exclusion criteria

1. Known fibrolamellar HCC, sarcomatoid HCC, or mixed cholangiocarcinoma and HCC.
2. Previous history of liver or other organ transplantation.
3. Has participated in another clinical study within 4 weeks prior to the first dose.
4. Has a known history of, or any evidence of CNS metastases.
5. Clinically symptomatic or recurrently drained pleural effusion, pericardial effusion, or ascites.
6. History of bleeding event due to esophageal and/or gastric varices within 3 months prior to the first dose of study treatment.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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60 participants in 2 patient groups

Pracytarabine

Experimental group

Description:

Treatment with 1200 mg/m²/d Pracytarabine

Treatment:

Drug: Pracytarabine

regorafenib

Active Comparator group

Description:

Treatment with regorafenib 160 mg once daily

Treatment:

Drug: regorafenib