Advanced Hepatocellular CarcinomaEarly Phase 1NCT06092112
A Clinical Trial for the Safety and Efficacy of CD-801 in Patients With Advanced Hepatocellular Carcinoma
A Early Phase 1 study testing CD-801 in people with Advanced Hepatocellular Carcinoma, run by Shanghai Changzheng Hospital at 1 site in China.
Trial 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 . Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Hepatocellular Carcinoma
- What is being tested?
- CD-801
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteChina
- Study dates
- 13 Oct 2023to 29 Apr 2024
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Males or females, aged 18 years or older.
- Subjects must have confirmed diagnosis of HCC according to the American Association for the Study of Liver Diseases criteria.
- Unresectable HCC.
- Subjects are unsuitable for local or systemic anti-tumor therapy or had tumor progression after receiving at least one kind of conventional treatment.
- According to mRECIST, subjects should be with at least 1 measurable target lesion.
- Life expectancy of 12 weeks or more.
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- Subjects must have an Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) of 0 to 2.
- Male 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.
- Subjects who have voluntary agreement to provide written informed consent and the willingness and ability to comply with all aspects of the protocol.
Who cannot join
- ALB < 28 g/L, or Bilirubin >5.0 mg/dL, or aspartate aminotransferase (AST), alkaline phosphatase (ALP), or alanine aminotransferase (ALT) >5×ULN.
- Inadequate renal function defined as creatinine >1.5 times the upper limit of normal (ULN) or calculated creatinine clearance < 40 mL/min.
- Absolute neutrophil count (ANC) < 1.0×109/L, or Platelets < 30×109/L, or Hemoglobin < 8.5 g/dL.
- International Normalized Ratio (INR) > 2.3.
- Subjects with a history of liver transplantation.
- Subjects with poorly controlled hypertension, diabetes or other serious heart or lung diseases, or with serious dysfunction.
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- Subjects with extrahepatic metastasis who had potential benefit from first-line systemic therapies.
- Subjects who had prior anticancer treatment with any locoregional therapies, antiangiogenic targeted therapies, immune checkpoint inhibitors or chemotherapy within 4 weeks (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.
- 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).
- Subjects with complication histories of liver cirrhosis or HCC such as gastrointestinal hemorrhage, overt hepatic encephalopathy, or uncontrollable ascites within 2 weeks prior to the first dose of study treatment.
- Uncontrolled active infection (eg, lung infections, or abdominal infections).
- Subjects with moderate to severe hepatic artery- portal vein fistula or hepatic artery - vein fistula which could not be avoided even through the artery super selection by DSA.
- 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 OS rate > 90%), such as adequately treated early gastric carcinoma, carcinoma in situ of the cervix, non-melanoma skin carcinoma, or localized prostate cancer.
- HBV DNA greater than 500 copies/mL, or HCV RNA greater than 15 U/mL.
- Subject is positive for Human Immunodeficiency Virus (HIV).
- Any subject who is allergic to MRI contrast agents.
- Pregnant/lactating women, or women who have the possibility of pregnancy.
- Participation in other investigational drug trials within 4 weeks prior to initiation of this study treatment.
- Any medical or other condition which, in the opinion of the investigator, would preclude participation in this clinical trial.
Trial locations and contacts
1 site in 1 country.
China1 site
Changzheng Hospital，Naval Medical University
Shanghai, Shanghai Municipality, 200003
About this study
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.
Read the 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
- Being tested
- CD-801
- Keywords
- Hepatocellular carcinoma, HNF4α
- Medical subject headings
- Carcinoma, Hepatocellular
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD-801Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| CD-801 treatment | Experimental | The subjects with advanced HCC will be treated by CD-801 through the hepatic artery injection. Receives: Drug: CD-801 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The objective response rate based on mRECIST
To assess the proportion of subjects who have best overall response of complete response or partial response at the time of data cutoff based on mRECIST
Measured: From the first study dose date until the date of documented complete response or partial response, assessed up to 24 months
Secondary outcomes (9)
Adverse events as assessed by CTCAE v5.0
To assess the incidence and severity of AE after CD-801 treatment in subjects with advanced hepatocellular carcinoma by CTCAE v5.0
Measured: Through study completion, an average of 2 years
The objective response rate based on RECIST v1.1
To assess the proportion of subjects who have best overall response of complete response or partial response at the time of data cutoff based on RECIST v1.1
Measured: From the first study dose date until the date of documented complete response or partial response, assessed up to 24 months
Duration of response based on mRECIST and RECIST v1.1
To assess the time from the first documentation of complete response or partial response to the date of first documentation of disease progression or death (whichever occurs first) based on mRECIST and RECIST v1.1
Measured: up to 24 months
Progression-free survival based on mRECIST and RECIST v1.1
To assess the time from the first study dose date to the date of first documentation of disease progression or death (whichever occurs first) based on mRECIST and RECIST v1.1
Measured: up to 24 months
Time to progression based on mRECIST and RECIST v1.1
To assess the time from the first study dose date to the date of first documentation of disease progression based on mRECIST and RECIST v1.1
Measured: up to 24 months
Time to response based on mRECIST and RECIST v1.1
To assess the time from the date of first study dose to the date of first documentation of complete response or partial response based on mRECIST and RECIST v1.1
Measured: up to 24 months
Disease control rate based on mRECIST and RECIST v1.1
To assess the proportion of subjects who have best overall response of complete response or partial response or stable disease (minimum duration from C1D1 to stable disease ≥5 weeks) based on mRECIST and RECIST v1.1
Measured: up to 24 months
Clinical benefit rate based on mRECIST and RECIST v1.1
To assess the proportion of subjects who have best overall response of complete response or partial response or durable stable disease (duration of stable disease ≥ 23 weeks) based on mRECIST and RECIST v1.1
Measured: up to 24 months
Overall Survival
To assess the time from the first study dose date until date of death from any cause . Subjects who are lost to follow-up and the subjects who are alive at the date of data cutoff will be censored at the date the subject was last known alive or the cut-off date, whichever comes earlier.
Measured: Throughout the entire course of treatment until the end of the follow-up period, an average of 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Oct 2023
- Study start13 Oct 2023
- Primary completion29 Apr 2024
- Study completion29 Apr 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Hepatocellular CarcinomaStudies of the same condition, open and under way.
- More trials run by Shanghai Changzheng HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- The subjects with advanced HCC will be treated by CD-801 through the hepatic artery injection
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shanghai Changzheng Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Wei-Fen Xie, Shanghai Changzheng Hospital
- Investigators
- Wei-Fen Xie, M.D., Principal investigator, Shanghai Changzheng Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06092112
- Sponsor's protocol ID
- CZXH-HCC-2023-IIT-EX
- Status last verified
- Apr 2025
- First submitted
- 7 Oct 2023
- Last updated
- 24 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Trial for the Safety and Efficacy of CD-801 in Patients With Advanced Hepatocellular Carcinoma
- ClinicalTrials.gov ID
- NCT06092112
- Acronym / sponsor ID
- CZXH-HCC-2023-IIT-EX
- Conditions
- Advanced Hepatocellular Carcinoma
- Keywords
- Hepatocellular carcinoma, HNF4α
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Shanghai Changzheng HospitalUniversity, hospital or other
- Study start
- 13 Oct 2023 (actual)
- Primary completion
- 29 Apr 2024 (actual)
- Study completion
- 29 Apr 2024 (actual)
- First posted
- 23 Oct 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- The subjects with advanced HCC will be treated by CD-801 through the hepatic artery injection
| Arm | Type | Receives |
|---|---|---|
| CD-801 treatment | Experimental | Drug: CD-801The subjects with advanced HCC will be treated by CD-801 through the hepatic artery injection. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Males or females, aged 18 years or older.
- Subjects must have confirmed diagnosis of HCC according to the American Association for the Study of Liver Diseases criteria.
- Unresectable HCC.
- Subjects are unsuitable for local or systemic anti-tumor therapy or had tumor progression after receiving at least one kind of conventional treatment.
- According to mRECIST, subjects should be with at least 1 measurable target lesion.
Key exclusion criteria (19 listed)
- ALB < 28 g/L, or Bilirubin >5.0 mg/dL, or aspartate aminotransferase (AST), alkaline phosphatase (ALP), or alanine aminotransferase (ALT) >5×ULN.
- Inadequate renal function defined as creatinine >1.5 times the upper limit of normal (ULN) or calculated creatinine clearance < 40 mL/min.
- Absolute neutrophil count (ANC) < 1.0×109/L, or Platelets < 30×109/L, or Hemoglobin < 8.5 g/dL.
- International Normalized Ratio (INR) > 2.3.
- Subjects with a history of liver transplantation.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CD-801The 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. | Drug | CD-801 treatment |
Primary outcome
The objective response rate based on mRECIST
Time frame: From the first study dose date until the date of documented complete response or partial response, assessed up to 24 months
To assess the proportion of subjects who have best overall response of complete response or partial response at the time of data cutoff based on mRECIST
9 secondary outcomes
Adverse events as assessed by CTCAE v5.0
Time frame: Through study completion, an average of 2 years
The objective response rate based on RECIST v1.1
Time frame: From the first study dose date until the date of documented complete response or partial response, assessed up to 24 months
Duration of response based on mRECIST and RECIST v1.1
Time frame: up to 24 months
Progression-free survival based on mRECIST and RECIST v1.1
Time frame: up to 24 months
Time to progression based on mRECIST and RECIST v1.1
Time frame: up to 24 months
Time to response based on mRECIST and RECIST v1.1
Time frame: up to 24 months
Disease control rate based on mRECIST and RECIST v1.1
Time frame: up to 24 months
Clinical benefit rate based on mRECIST and RECIST v1.1
Time frame: up to 24 months
Overall Survival
Time frame: Throughout the entire course of treatment until the end of the follow-up period, an average of 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2025.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wei-Fen Xie, M.D. principal investigator
Shanghai Changzheng Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing CD-801 in people with Advanced Hepatocellular Carcinoma, run by Shanghai Changzheng Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 19 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The objective response rate based on mRECIST (time frame: From the first study dose date until the date of documented complete response or partial response, assessed up to 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2025.