NCT06092112

A Clinical Trial for the Safety and Efficacy of CD-801 in Patients With Advanced Hepatocellular Carcinoma

• Status: Completed
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Shanghai Changzheng Hospital · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this investigator-initiated, a single-arm, open-label, pilot study is to investigate the safety, tolerability, and efficacy of CD-801 treatment in subjects with advanced hepatocellular carcinoma. Condition of disease: advanced hepatocellular carcinoma . Intervention：treatment with 100μg CD-801 through the hepatic artery at two-week intervals. The dosing interval will be adjusted based on subject tolerability, safety, and efficacy. For example, it may be adjusted to administer the medication once every three weeks or four weeks. Drug: CD-801, a drug designed specifically to enhance the expression of HNF4α and selectively target liver cancer cells.

Detailed description

Hepatocellular carcinoma (HCC) is the most common form of liver cancer. Recent advancements in understanding tumor biology and the tumor microenvironment have dramatically transformed the treatment landscape for advanced stage HCC. However, the survival for advanced HCC patients still remains unsatisfactory. Differentiation therapy in oncology is defined as a therapeutic strategy that reactivates endogenous differentiation programs and reverts malignant phenotypes. Its hallmark success is the treatment of acute promyelocytic leukemia (APL) by the combination of all-trans retinoic acid (ATRA) and arsenic. Unfortunately, this approach has achieved limited success in solid tumors. Hepatocyte nuclear factor 4α (HNF4α) is a transcription factor (TF) belonging to the nuclear receptor family. HNF4α is highly enriched in mature hepatocytes and serves as a master regulator of hepatocyte differentiation and hepatic metabolism. Previous studies, including the investigators' and others, have demonstrated that the reduced expression of HNF4α plays a critical role in hepatocarcinogenesis. Restoring HNF4α expression induces the differentiation of HCC cells into mature hepatocytes and has shown significant therapeutic effects in various animal models of HCC. In this study, the investigators developed CD-801, a drug designed specifically to enhance the expression of HNF4α and selectively target liver cancer cells, for the treatment of HCC patients. Preclinical studies have shown that CD-801 effectively inhibits the growth of subcutaneous and orthotopic liver tumors in mice. Acute toxicity tests in SD rats have demonstrated that a single intravenous injection of CD-801 injection at a dose of 150 μg/animal is well-tolerated, with no significant toxicity, indicating good safety profiles. Furthermore, in the dose escalation phase of the clinical trial which the investigators have completed, CD-801(25, 50 and 100 μg) was found to be well-tolerated. None of the patients experienced dose-limiting toxicities (DLTs) during the DLT phase. This trial is a single-arm, open-label, exploratory clinical study aimed at further evaluating the efficacy, safety, and tolerability of 100 μg CD-801 administered through the hepatic artery in the treatment of advanced-stage HCC at two-week intervals (The dosing interval will be adjusted based on subject tolerability, safety, and efficacy. For example, it may be adjusted to administer the medication once every three weeks or four weeks).

Conditions

• Advanced Hepatocellular Carcinoma

Interventions

Type	Name	Description
DRUG	CD-801	The subjects with advanced HCC will be treated by 100 μg CD-801 through the hepatic artery injection at two-week intervals. The dosing interval will be adjusted based on subject tolerability, safety, and efficacy. For example, it may be adjusted to administer the medication once every three weeks or four weeks.

Timeline

• Start date: 2023-10-13
• Primary completion: 2024-04-29
• Completion: 2024-04-29
• First posted: 2023-10-23
• Last updated: 2025-04-24

Locations

1 site across 1 country: China