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

Randomized, Controlled, Open-Label, Multicenter Phase Ⅱ/Ⅲ Seamless Adaptive Design Registration Clinical Trial of Pracytarabine Versus Regorafenib for Advanced Hepatocellular Carcinoma After Failure of Targeted Drugs and Immune Checkpoint Inhibitors or Dual Immune Checkpoint Inhibitors Therapy

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.

Study Overview

• Status: Active, not recruiting
• Conditions: HCC
• Intervention / Treatment: Drug: Pracytarabine; Drug: regorafenib

• Study Type: Interventional
• Enrollment (Estimated): 60
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• China
  • Jiangsu
    • Nanjing, Jiangsu, China, 211100
      • Nanjing Tianyinshan Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Pracytarabine; Treatment with 1200 mg/m²/d Pracytarabine	Drug: Pracytarabine; Pracytarabine 1200 mg/m²/d, administered as a continuous intravenous infusion for 7 days (Days 1 to 7), followed by a 21-day treatment-free observation period; each treatment cycle is 4 weeks.
Active Comparator: regorafenib; Treatment with regorafenib 160 mg once daily	Drug: regorafenib; Regorafenib 160 mg, once daily, administered continuously for 3 weeks followed by 1 week of rest, i.e., each cycle is 4 weeks.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
9-month Overall Survival (OS) rate	9-month Overall Survival (OS) rate：The proportion of the total number of subjects who survived for 9 months from randomization.	Up to 12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Progression-free survival (PFS) assessed by investigator per RECIST v1.1	PFS is defined as the time from randomization till the first documented disease progression (Per RECIST v1.1 assessed by the investigator) or death due to any cause, whichever occurs first.	Up to 12 months

Collaborators and Investigators

• Sponsor: Xi'an Xintong Pharmaceutical Research Co.,Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): September 1, 2025
• Primary Completion (Estimated): February 28, 2027
• Study Completion (Estimated): April 30, 2027

Study Registration Dates

• First Submitted: December 9, 2025
• First Submitted That Met QC Criteria: December 9, 2025
• First Posted (Estimated): December 23, 2025

Study Record Updates

• Last Update Posted (Actual): December 31, 2025
• Last Update Submitted That Met QC Criteria: December 23, 2025
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: regorafenib
• Other Study ID Numbers: MB07133-2025-01

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No