Ignite Creation Date:
2024-05-19 @ 5:35 PM
Last Modification Date:
2024-10-26 @ 3:29 PM
Study NCT ID:
NCT06418659
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-05-17
First Post:
2024-05-13
Brief Title:
A Clinical Trial Evaluating the Safety and Efficacy of Intravenous CD-801 in Treating Advanced HCC Patients
Sponsor:
Shanghai Changzheng Hospital
Organization:
Shanghai Changzheng Hospital
Study Overview
Official Title:
A Clinical Trial Assessing the Safety and Efficacy of Intravenous CD-801 for the Treatment of Patients With Advanced Hepatocellular Carcinoma
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The goal of this investigator-initiated a single-arm open-label pilot study is to investigate the safety tolerability and efficacy of Intravenous CD-801 treatment in subjects with advanced hepatocellular carcinomaHCC
Condition of disease advanced hepatocellular carcinoma
Intervention CD-801 will be administered intravenously for the treatment of HCC The dosing regimen is planned for a second dose 14 3 days post-initial treatment followed by subsequent treatments every 28 7 days with adjustments made based on patient tolerance and therapeutic response The trial is structured in two phases dose escalation and dose expansion
Dose Escalation Phase
The study employs a i33 design to assess escalating CD-801 dosages 25 μg 50 μg and 100 μg Post-initial dose a 14-day DLT observation will evaluate tolerability and safety guiding dose adjustments or selection of the Recommended Dose RD for the expansion phase Cohorts may include up to 9 participants adjusted for safety
Dose Expansion Phase
The expansion phase will use the safe dosage and regimen from the escalation phase with treatments starting 14 3 days after the initial dose then every 28 7 days adjusted as needed It ends upon complete response disease progression toxicity withdrawal loss to follow-up new oncological treatments or investigator termination with a final assessment 14 days post-last dose The phase plans to enroll about 10 participants to further assess CD-801s safety tolerability and antitumor effects using mRECIST
Drug CD-801 a drug specifically designed to target liver cancer cells and facilitate the expression of HNF4α
Condition of disease advanced hepatocellular carcinoma
Intervention CD-801 will be administered intravenously for the treatment of HCC The dosing regimen is planned for a second dose 14 3 days post-initial treatment followed by subsequent treatments every 28 7 days with adjustments made based on patient tolerance and therapeutic response The trial is structured in two phases dose escalation and dose expansion
Dose Escalation Phase
The study employs a i33 design to assess escalating CD-801 dosages 25 μg 50 μg and 100 μg Post-initial dose a 14-day DLT observation will evaluate tolerability and safety guiding dose adjustments or selection of the Recommended Dose RD for the expansion phase Cohorts may include up to 9 participants adjusted for safety
Dose Expansion Phase
The expansion phase will use the safe dosage and regimen from the escalation phase with treatments starting 14 3 days after the initial dose then every 28 7 days adjusted as needed It ends upon complete response disease progression toxicity withdrawal loss to follow-up new oncological treatments or investigator termination with a final assessment 14 days post-last dose The phase plans to enroll about 10 participants to further assess CD-801s safety tolerability and antitumor effects using mRECIST
Drug CD-801 a drug specifically designed to target liver cancer cells and facilitate the expression of HNF4α
Detailed Description:
Hepatocellular carcinoma HCC is the most common form of liver cancer Recent advancements in understanding tumor biology and the tumor microenvironment along with the approval of systemic therapeutic agents including immunotherapy and vascular endothelial growth factor VEGF-targeted agents 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 our own 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 specifically designed to target liver cancer cells and facilitate the expression of HNF4α 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 Sprague-Dawley rats have demonstrated that a single intravenous injection of CD-801 injection at a dose of 150 μganimal is well-tolerated with no significant toxicity indicating good safety profiles
This trial structured in two phases dose escalation and dose expansion is a single-arm open-label exploratory clinical study aimed at evaluating the efficacy safety and tolerability of CD-801 administered intravenously through a peripheral vein in the treatment of advanced-stage HCC The treatment schedule is planned for a second dose 14 3 days post-initial treatment followed by subsequent treatments every 28 7 days with adjustments made based on patient tolerance and therapeutic response
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 our own 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 specifically designed to target liver cancer cells and facilitate the expression of HNF4α 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 Sprague-Dawley rats have demonstrated that a single intravenous injection of CD-801 injection at a dose of 150 μganimal is well-tolerated with no significant toxicity indicating good safety profiles
This trial structured in two phases dose escalation and dose expansion is a single-arm open-label exploratory clinical study aimed at evaluating the efficacy safety and tolerability of CD-801 administered intravenously through a peripheral vein in the treatment of advanced-stage HCC The treatment schedule is planned for a second dose 14 3 days post-initial treatment followed by subsequent treatments every 28 7 days with adjustments made based on patient tolerance and therapeutic response
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None