Intrahepatic CholangiocarcinomaEarly Phase 1NCT06572189
The Safety and Efficacy of CD-801 in Patients With Advanced Intrahepatic Cholangiocarcinoma
A Early Phase 1 study testing Injection of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α) in people with Intrahepatic Cholangiocarcinoma, run by Shanghai Changzheng Hospital at 1 site in China.
Trial summary
The goal of this clinical trial is to learn about the safety of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α) in treating patients with advanced intrahepatic cholangiocarcinoma (ICC). It will also learn if CD-801 works to treat advanced ICC. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Intrahepatic Cholangiocarcinoma
- What is being tested?
- Injection of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteChina
- Study dates
- 28 Aug 2024to 31 Jul 2026
- 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.
- Histologically or cytologically confirmed intrahepatic cholangiocarcinoma patients.
- Patients with intrahepatic cholangiocarcinoma not suitable for surgical resection, liver transplantation, or ablation therapy, or those with post-surgical recurrence and/or metastasis.
- Patients not suitable for local or systemic treatment, or those who have progressed after at least one chemotherapy regimen containing gemcitabine/fluoropyrimidine/platinum, etc..
- Life expectancy of 12 weeks or more.
- Subjects must have an Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) of 0 to 2.
- 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.
- Subjects who had a voluntary agreement to provide written informed consent and the willingness and ability to comply with all aspects of the protocol.
Who cannot join
- Patients with any of the following criteria were excluded from participation in this study:
- 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.
- Inadequate renal function defined as creatinine >1.5 × 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.
- Poorly controlled hypertension, diabetes or other serious heart or lung diseases, or with serious dysfunction.
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- Patients who have received local or systemic anti-tumor treatments such as ablation, Transhepatic Arterial Chemotherapy and Embolization (TACE), local radiotherapy of the liver, immunotherapy, targeted therapy, etc., within 4 weeks, or chemotherapy, other trial drugs, or radiotherapy of metastatic lesions within 2 weeks, except for treatment regimens assessed as disease progression according to mRECIST or RECIST 1.1.
- Patients with uncurable brain metastasis.
- 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).
- 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.
- Uncontrolled active infection (eg, lung infections, or abdominal infections).
- 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.
- Hepatitis B virus DNA greater than 500 copies/mL, or hepatitis C virus RNA greater than 15 U/mL.
- Positive for human immunodeficiency virus (HIV).
- Allergic to contrast agents.
- Pregnant/lactating women, or women with the possibility of pregnancy.
- Any medical conditions which, in the opinion of the investigator, would preclude participation in this clinical trial.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: The Safety and Efficacy of Transarterial Injection of CD-801 in Patients With Advanced Intrahepatic Cholangiocarcinoma
The goal of this clinical trial is to learn about the safety of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α) in treating patients with advanced intrahepatic cholangiocarcinoma (ICC). It will also learn if CD-801 works to treat advanced ICC. The main questions it aims to answer are:
- What medical problems do participants have when injecting CD-801?
- Is CD-801 effective for ICC patients according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 or modified RECIST (mRECIST)
Read more
Participants will:
Receive administration of 100μg of CD-801 via hepatic arterial injection every 14 ± 3 days (the dosing interval will be adjusted based on the tolerability, safety, and therapeutic effect of the subjects), for one treatment cycle. Treatment will continue until the occurrence of disease progression, death, intolerable toxicity, voluntary withdrawal of informed consent, loss to follow-up, initiation of new antitumor treatment, or termination of the study by the investigator (whichever comes first), and completion of the final follow-up and assessment 14 days after the last administration.
- Conditions
- Intrahepatic Cholangiocarcinoma
- Being tested
- Injection of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α)
- Medical subject headings
- Cholangiocarcinoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Injection of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α)Genetic
Receive administration of 100μg of CD-801 via hepatic arterial injection every 14 ± 3 days (the dosing interval will be adjusted based on the tolerability, safety, and therapeutic effect of the subjects), for one treatment cycle. Treatment will continue until the occurrence of disease progression, death, intolerable toxicity, voluntary withdrawal of informed consent, loss to follow-up, initiation of new antitumor treatment, or termination of the study by the investigator (whichever comes first), and completion of the final follow-up and assessment 14 days after the last administration.
| Study group | Role | What they receive |
|---|---|---|
| CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α | Experimental | Patients will receive administration of 100μg of CD-801 via hepatic arterial injection every 14 ± 3 days Receives: Genetic: Injection of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
The incidence, timing, and severity of adverse events, serious adverse events, and adverse events leading to treatment discontinuation according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 5.0.
Measured: up to 24 months
Secondary outcomes (12)
Objective Response Rate
The proportion of subjects achieving Complete Response (CR) and Partial Response (PR).
Measured: up to 24 months
Duration Of Response
The time from the first documentation of response (either Partial Response or Complete Response) after the start of treatment to the first documentation of disease progression or death due to Intrahepatic Cholangiocarcinoma (ICC), whichever occurs first.
Measured: up to 24 months
Progression-Free Survival
The time from the start of treatment to the first occurrence of disease progression or death due to Hepatocellular Carcinoma (HCC), whichever comes first.
The time from the initiation of treatment to the first documentation of disease progression or death due to Intrahepatic Cholangiocarcinoma (ICC), whichever occurs first.
Measured: From date of the start of treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 months
Time To Progression
The time from the start of treatment to the first recorded documentation of disease progression, excluding death.
Measured: up to 24 months
Time To Response
The time from the commencement of treatment to the first documentation of response achieving either Partial Response (PR) or Complete Response (CR).
Measured: up to 24 months
Disease Control Rate
The proportion of subjects achieving response with either Partial Response (PR) or Complete Response (CR), or maintaining stable disease (SD with duration of ≥ 5 weeks) after treatment.
Measured: up to 24 months
Clinical Benefit Rate
The proportion of subjects who achieve either a Partial Response (PR) or Complete Response (CR) following treatment, or maintain durable stable disease (SD with a duration of at least 23 weeks).
Measured: up to 24 months
Overall Survival
The time from the start of treatment to death from any cause.
Measured: From date of the start of treatment until the date of death from any cause, assessed up to 36 months
Patient Reported Outcome-1
the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 (EORTC QLQ-C30)
Measured: up to 24 months
Patient Reported Outcome-2
the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire BIL21 (QLQ-BIL21)
Measured: up to 24 months
Patient Reported Outcome-3
The Generic Euroquol Five Dimension Five Level (EQ-5D-5L) Questionnaire
Measured: up to 24 months
The changes in tumor markers after treatment.
The changes in tumor markers such as CA19-9, Carcinoembryonic Antigen (CEA), and Alpha-fetoprotein (AFP) after treatment.
Measured: up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Aug 2024
- Study start28 Aug 2024 (estimated)
- Primary completion31 Jul 2026 (estimated)
- Study completion31 Jul 2026 (estimated)
Why it stopped: Based on the results of another intravenous injection therapy CD-801 for the treatment of hepatocellular carcinoma, we have decided to change the administration method and therefore withdraw this trial protocol.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Intrahepatic CholangiocarcinomaStudies 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
- Masking
- None (open label)
- Enrolment
- 0 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
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06572189
- Sponsor's protocol ID
- 2024SL015
- Status last verified
- Jun 2025
- First submitted
- 6 Aug 2024
- Last updated
- 29 Jun 2025
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jun 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Safety and Efficacy of Transarterial Injection of CD-801 in Patients With Advanced Intrahepatic Cholangiocarcinoma
- ClinicalTrials.gov ID
- NCT06572189
- Acronym / sponsor ID
- 2024SL015
- Conditions
- Intrahepatic Cholangiocarcinoma
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Based on the results of another intravenous injection therapy CD-801 for the treatment of hepatocellular carcinoma, we have decided to change the administration method and therefore withdraw this trial protocol.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Shanghai Changzheng HospitalUniversity, hospital or other
- Study start
- 28 Aug 2024 (estimated)
- Primary completion
- 31 Jul 2026 (estimated)
- Study completion
- 31 Jul 2026 (estimated)
- First posted
- 27 Aug 2024 (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 clinical trial is to learn about the safety of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α) in treating patients with advanced intrahepatic cholangiocarcinoma (ICC). It will also learn if CD-801 works to treat advanced ICC. The main questions it aims to answer are:
- What medical problems do participants have when injecting CD-801?
- Is CD-801 effective for ICC patients according to Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1 or modified RECIST (mRECIST)
Participants will:
Receive administration of 100μg of CD-801 via hepatic arterial injection every 14 ± 3 days (the dosing interval will be adjusted based on the tolerability, safety, and therapeutic effect of the subjects), for one treatment cycle. Treatment will continue until the occurrence of disease progression, death, intolerable toxicity, voluntary withdrawal of informed consent, loss to follow-up, initiation of new antitumor treatment, or termination of the study by the investigator (whichever comes first), and completion of the final follow-up and assessment 14 days after the last administration.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α | Experimental | Genetic: Injection of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α)Patients will receive administration of 100μg of CD-801 via hepatic arterial injection every 14 ± 3 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Males or females, aged 18 years or older.
- Histologically or cytologically confirmed intrahepatic cholangiocarcinoma patients.
- Patients with intrahepatic cholangiocarcinoma not suitable for surgical resection, liver transplantation, or ablation therapy, or those with post-surgical recurrence and/or metastasis.
- Patients not suitable for local or systemic treatment, or those who have progressed after at least one chemotherapy regimen containing gemcitabine/fluoropyrimidine/platinum, etc..
- Life expectancy of 12 weeks or more.
Key exclusion criteria (17 listed)
- Patients with any of the following criteria were excluded from participation in this study:
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 |
|---|---|---|
| Injection of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α)Receive administration of 100μg of CD-801 via hepatic arterial injection every 14 ± 3 days (the dosing interval will be adjusted based on the tolerability, safety, and therapeutic effect of the subjects), for one treatment cycle. Treatment will continue until the occurrence of disease progression, death, intolerable toxicity, voluntary withdrawal of informed consent, loss to follow-up, initiation of new antitumor treatment, or termination of the study by the investigator (whichever comes first), and completion of the final follow-up and assessment 14 days after the last administration. | Genetic | CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α |
Primary outcome
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability]
Time frame: up to 24 months
The incidence, timing, and severity of adverse events, serious adverse events, and adverse events leading to treatment discontinuation according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) Version 5.0.
12 secondary outcomes
Objective Response Rate
Time frame: up to 24 months
Duration Of Response
Time frame: up to 24 months
Progression-Free Survival
Time frame: From date of the start of treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 36 months
Time To Progression
Time frame: up to 24 months
Time To Response
Time frame: up to 24 months
Disease Control Rate
Time frame: up to 24 months
Clinical Benefit Rate
Time frame: up to 24 months
Overall Survival
Time frame: From date of the start of treatment until the date of death from any cause, assessed up to 36 months
Patient Reported Outcome-1
Time frame: up to 24 months
Patient Reported Outcome-2
Time frame: up to 24 months
Patient Reported Outcome-3
Time frame: up to 24 months
The changes in tumor markers after treatment.
Time frame: up to 24 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jun 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.
The record lists no contact. Use the official record to reach the sponsor.
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 Injection of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α) in people with Intrahepatic Cholangiocarcinoma, 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 8 inclusion and 17 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: withdrawn, as of the sponsor's last update on 29 Jun 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 was withdrawn before enrolling participants, so there are no results.
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?
Incidence of Treatment-Emergent Adverse Events [Safety and Tolerability] (time frame: 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 29 Jun 2025.