Evaluating CD-801 for treating advanced liver cancer

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

This study is testing a new treatment called CD-801 to see if it can safely help people with advanced liver cancer feel better and improve their outcomes.

Quick facts

• Phase: EARLY_PHASE1
• Study type: Interventional
• Enrollment: 37 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Shanghai Changzheng Hospital (other)
• Drugs / interventions: chemotherapy, immunotherapy
• Locations: 1 site (Shanghai, Shanghai Municipality)
• Trial ID: NCT06418659 on ClinicalTrials.gov

What this trial studies

This pilot study investigates the safety, tolerability, and efficacy of intravenous CD-801 in patients with advanced hepatocellular carcinoma (HCC). It employs a single-arm, open-label design with a dose escalation phase followed by a dose expansion phase, where participants receive escalating doses of CD-801. The study aims to restore the expression of hepatocyte nuclear factor 4α (HNF4α) to induce differentiation in HCC cells, potentially improving treatment outcomes for this challenging condition.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 and older with unresectable advanced HCC who have not benefited from conventional therapies.

Not a fit: Patients with resectable HCC or those who are eligible for other systemic therapies may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced HCC who have limited treatment alternatives.

How similar studies have performed: While differentiation therapy has shown success in treating acute promyelocytic leukemia, its application in solid tumors like HCC is still largely untested.

Eligibility criteria

Inclusion Criteria:

1. Males or females, aged 18 years or older.
2. Subjects must have confirmed diagnosis of HCC with any of the following criteria according to the American Association for the Study of Liver Diseases criteria.
3. Unresectable HCC.
4. Subjects were not eligible for locoregional or systemic therapies, or had disease progression, or would not benefit after at least one of the conventional therapies.
5. According to mRECIST, subjects should be with at least 1 measurable target lesion.
6. Life expectancy of 12 weeks or more.
7. Subjects must have an Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) of 0 to 2.
8. Males with fertility and females of childbearing potential are willing to use a highly effective method of contraception for the entire study period and for 6 months after study drug discontinuation. Females of childbearing age, including premenopausal females and within 2 years after menopause, must have a negative serum pregnancy test result within 7 days prior to the first dose of study treatment.
9. Subjects who had a voluntary agreement to provide written informed consent and the willingness and ability to comply with all aspects of the protocol.

Exclusion Criteria:

1. Inadequate liver function：Albumin (ALB) \< 25 g/L, or total bilirubin \> 5 × the upper limit of normal (ULN), or aspartate aminotransferase (AST), alkaline phosphatase (ALP), or alanine aminotransferase (ALT) \>10 × ULN.
2. Inadequate renal function defined as creatinine \>1.5 × ULN or calculated creatinine clearance \< 40 mL/min.
3. Absolute neutrophil count (ANC) \< 1.0×109/L, or Platelets \< 30×109/L, or Hemoglobin \< 8.5 g/dL.
4. International Normalized Ratio (INR) \> 2.3.
5. Subjects with a history of liver transplantation.
6. Subjects with poorly controlled hypertension, diabetes or other serious heart or lung diseases, or with serious dysfunction.
7. Subjects with extrahepatic metastasis who had not received first-line systemic therapies (excluding those who are not eligible for systemic therapies) or who were receiving effective systemic therapy currently.
8. Subjects who had prior anticancer treatment with any locoregional therapies, antiangiogenic targeted therapies, immune checkpoint inhibitors or chemotherapy (within 4 weeks, or within 2 weeks in case of sorafenib), radiotherapy (within 3 weeks), or active traditional Chinese medicine (within 2 weeks) before the first dose of study treatment, except for the treatments after which the disease still progressed according to mRECIST.
9. All toxicities related to prior locoregional or systemic anti-tumor treatments are still grade 2 or more (except for hair loss and other events that have been judged tolerable by researchers).
10. Subjects with complication histories of liver cirrhosis or HCC such as gastrointestinal hemorrhage, overt hepatic encephalopathy, or refractory ascites within 2 weeks prior to the first dose of study treatment.
11. Uncontrolled active infection (eg, lung infections, or abdominal infections).
12. History of malignancy other than HCC within 5 years prior to screening, with the exception of malignancies with a negligible risk of metastasis or death (e.g., 5-year overall survival rate \> 90%), such as adequately treated early gastric carcinoma, carcinoma in situ of the cervix, non-melanoma skin carcinoma, or localized prostate cancer.
13. HBV DNA greater than 500 copies/mL, or HCV RNA greater than 15 U/mL.
14. Subject is positive for Human Immunodeficiency Virus (HIV).
15. Any subject who is allergic to MRI contrast agents.
16. Pregnant/lactating women, or women who have the possibility of pregnancy.
17. Participation in other investigational drug trials within 4 weeks prior to initiation of this study treatment.
18. Any medical or other condition which, in the opinion of the investigator, would preclude participation in this clinical trial.

Where this trial is running

Shanghai, Shanghai Municipality

• Changzheng Hospital — Shanghai, Shanghai Municipality, China (RECRUITING)

Study contacts

• Principal investigator: Wei-Fen Xie, M.D. — Shanghai Changzheng Hospital
• Study coordinator: CHUAN YIN, M.D.

Conditions: Advanced Hepatocellular Carcinoma, Hepatocellular carcinoma, Hepatocyte nuclear factor 4α