The Safety and Efficacy of CD-801 in Patients With Advanced Intrahepatic Cholangiocarcinoma
NCT Number: NCT06572189
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: EARLY_PHASE1
Conditions: Intrahepatic Cholangiocarcinoma
Interventions: GENETIC: Injection of CD-801, a lipid nanoparticle-encapsulated self-replicating RNA encoding hepatocyte nuclear factor 4α (HNF4α)
Sponsor: Shanghai Changzheng Hospital
Sex: ALL
Age: ADULT, OLDER_ADULT
Enrollment: 20
Study Design: Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-08-28
Completion Date: 2026-07-31
Locations: Wen-Ping Xu, Shanghai, Shanghai, 200003, China

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. The main questions it aims to answer are:

1. What medical problems do participants have when injecting CD-801?
2. 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.

Primary Outcome Measures: 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., up to 24 months
Secondary Outcome Measures: Objective Response Rate, The proportion of subjects achieving Complete Response (CR) and Partial Response (PR)., 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., 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., 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., 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)., 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., 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)., up to 24 months|Overall Survival, The time from the start of treatment to death from any cause., 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), up to 24 months|Patient Reported Outcome-2, the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire BIL21 (QLQ-BIL21), up to 24 months|Patient Reported Outcome-3, The Generic Euroquol Five Dimension Five Level (EQ-5D-5L) Questionnaire, 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., up to 24 months