HCL001 Cell Injection for Decompensated Cirrhosis: A Phase I/II Trial

NCT07488546 | Enrolling By Invitation Phase 1

• 2 other identifiers: HI-HCL001CT-01CTR20253647
• Study Type: interventional
• Target: 39
• Locations: 1 country
• Sites: 1

Brief Summary

Three sequential dose cohorts are predefined for single administration: Cohort 1 (1.0 × 10⁶ cells/kg), Cohort 2 (2.0 × 10⁶ cells/kg), and Cohort 3 (4.0 × 10⁶ cells/kg). Escalation proceeds from Cohort 1 to Cohort 3 according to a "3 + 3" algorithm, with each subject receiving a single assigned dose. To ensure maximal subject safety, the first subject in every cohort must complete ≥ 14 days of post-dose observation within the DLT evaluation window before additional subjects in that cohort may be dosed. After the last subject in a cohort has completed the entire DLT observation period (from dosing through Day 29), the Safety Review Committee (SRC) will review the cumulative safety data and, by consensus, determine whether escalation to the first subject of the subsequent cohort may commence. Concurrent enrollment into two or more cohorts is prohibited. Upon completion of the Week 12 assessment in the last subject enrolled in Phase I, one or two dose levels will be selected for Phase II expansion based on the safety and preliminary efficacy data obtained during Phase I.

Conditions

Decompensated Cirrhosis

Keywords

Patients with decompensated cirrhosis

Outcome Measures

Primary Outcomes (2)

• Dose-limiting toxicity (DLT).

  Grade ≥ 3 non-hematologic toxicity

  Within 29 days after administration

• Types and incidence of treatment-related adverse events during the DLT observation period.

  Types and incidence of treatment-related adverse events during the DLT observation period.

  Within 29 days after administration

Secondary Outcomes (6)

• Secondary Endpoint

  From enrollment to the end of treatment at 48 weeks

• Secondary Endpoint

  From enrollment to the end of treatment at 48 weeks

• Secondary Endpoint

  baseline , Week 4, Week 12,Week 24, Week 48 after administration

• Secondary Endpoint

  baseline , Week 4, Week 12,Week 24, Week 48 after administration

• Secondary Endpoint

  baseline , Week 4, Week 12,Week 24, Week 48 after administration

Study Arms (3)

Phase I：Cohort 1 (1.0 × 10⁶ cells/kg)

EXPERIMENTAL

Three sequential dose cohorts are predefined for single administration: Cohort 1 (1.0 × 10⁶ cells/kg), Cohort 2 (2.0 × 10⁶ cells/kg), and Cohort 3 (4.0 × 10⁶ cells/kg). Escalation proceeds from Cohort 1 to Cohort 3 according to a "3 + 3" algorithm, with each subject receiving a single assigned dose. To ensure maximal subject safety, the first subject in every cohort must complete ≥ 14 days of post-dose observation within the DLT evaluation window before additional subjects in that cohort may be dosed. After the last subject in a cohort has completed the entire DLT observation period (from dosing through Day 29), the Safety Review Committee (SRC) will review the cumulative safety data and, by consensus, determine whether escalation to the first subject of the subsequent cohort may commence. Concurrent enrollment into two or more cohorts is prohibited.

Drug: Cohort 1 (1.0 × 10⁶ cells/kg)

Phase I：Cohort 2 (2.0 × 10⁶ cells/kg)

EXPERIMENTAL

Three sequential dose cohorts are predefined for single administration: Cohort 1 (1.0 × 10⁶ cells/kg), Cohort 2 (2.0 × 10⁶ cells/kg), and Cohort 3 (4.0 × 10⁶ cells/kg). Escalation proceeds from Cohort 1 to Cohort 3 according to a "3 + 3" algorithm, with each subject receiving a single assigned dose. To ensure maximal subject safety, the first subject in every cohort must complete ≥ 14 days of post-dose observation within the DLT evaluation window before additional subjects in that cohort may be dosed. After the last subject in a cohort has completed the entire DLT observation period (from dosing through Day 29), the Safety Review Committee (SRC) will review the cumulative safety data and, by consensus, determine whether escalation to the first subject of the subsequent cohort may commence. Concurrent enrollment into two or more cohorts is prohibited.

Drug: Cohort 2 (2.0 × 10⁶ cells/kg)

Phase I：Cohort 3 (4.0 × 10⁶ cells/kg)

EXPERIMENTAL

Three sequential dose cohorts are predefined for single administration: Cohort 1 (1.0 × 10⁶ cells/kg), Cohort 2 (2.0 × 10⁶ cells/kg), and Cohort 3 (4.0 × 10⁶ cells/kg). Escalation proceeds from Cohort 1 to Cohort 3 according to a "3 + 3" algorithm, with each subject receiving a single assigned dose. To ensure maximal subject safety, the first subject in every cohort must complete ≥ 14 days of post-dose observation within the DLT evaluation window before additional subjects in that cohort may be dosed. After the last subject in a cohort has completed the entire DLT observation period (from dosing through Day 29), the Safety Review Committee (SRC) will review the cumulative safety data and, by consensus, determine whether escalation to the first subject of the subsequent cohort may commence. Concurrent enrollment into two or more cohorts is prohibited.

Drug: Cohort 3 (4.0 × 10⁶ cells/kg)

Interventions

Cohort 1 (1.0 × 10⁶ cells/kg) DRUG

Three sequential dose cohorts are predefined for single administration: Cohort 1 (1.0 × 10⁶ cells/kg), Cohort 2 (2.0 × 10⁶ cells/kg), and Cohort 3 (4.0 × 10⁶ cells/kg). Escalation proceeds from Cohort 1 to Cohort 3 according to a "3 + 3" algorithm, with each subject receiving a single assigned dose. To ensure maximal subject safety, the first subject in every cohort must complete ≥ 14 days of post-dose observation within the DLT evaluation window before additional subjects in that cohort may be dosed. After the last subject in a cohort has completed the entire DLT observation period (from dosing through Day 29), the Safety Review Committee (SRC) will review the cumulative safety data and, by consensus, determine whether escalation to the first subject of the subsequent cohort may commence. Concurrent enrollment into two or more cohorts is prohibited.

Phase I：Cohort 1 (1.0 × 10⁶ cells/kg)

Cohort 2 (2.0 × 10⁶ cells/kg) DRUG

Three sequential dose cohorts are predefined for single administration: Cohort 1 (1.0 × 10⁶ cells/kg), Cohort 2 (2.0 × 10⁶ cells/kg), and Cohort 3 (4.0 × 10⁶ cells/kg). Escalation proceeds from Cohort 1 to Cohort 3 according to a "3 + 3" algorithm, with each subject receiving a single assigned dose. To ensure maximal subject safety, the first subject in every cohort must complete ≥ 14 days of post-dose observation within the DLT evaluation window before additional subjects in that cohort may be dosed. After the last subject in a cohort has completed the entire DLT observation period (from dosing through Day 29), the Safety Review Committee (SRC) will review the cumulative safety data and, by consensus, determine whether escalation to the first subject of the subsequent cohort may commence. Concurrent enrollment into two or more cohorts is prohibited.

Phase I：Cohort 2 (2.0 × 10⁶ cells/kg)

Cohort 3 (4.0 × 10⁶ cells/kg) DRUG

Three sequential dose cohorts are predefined for single administration: Cohort 1 (1.0 × 10⁶ cells/kg), Cohort 2 (2.0 × 10⁶ cells/kg), and Cohort 3 (4.0 × 10⁶ cells/kg). Escalation proceeds from Cohort 1 to Cohort 3 according to a "3 + 3" algorithm, with each subject receiving a single assigned dose. To ensure maximal subject safety, the first subject in every cohort must complete ≥ 14 days of post-dose observation within the DLT evaluation window before additional subjects in that cohort may be dosed. After the last subject in a cohort has completed the entire DLT observation period (from dosing through Day 29), the Safety Review Committee (SRC) will review the cumulative safety data and, by consensus, determine whether escalation to the first subject of the subsequent cohort may commence. Concurrent enrollment into two or more cohorts is prohibited.

Phase I：Cohort 3 (4.0 × 10⁶ cells/kg)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age ≥ 18 years at the time of informed consent; sex unrestricted.
• Diagnosis of decompensated cirrhosis established according to the Chinese Guidelines for the Diagnosis and Treatment of Liver Cirrhosis (2019 Edition) and attributable to: hepatitis B, hepatitis C, alcohol, autoimmune disease, metabolic-associated fatty liver disease, or other less common aetiologies when deemed appropriate by the investigator.
• Karnofsky Performance Status (KPS) ≥ 60 (see Appendix 6).
• Presence of a vascular access route judged safe for selective catheterisation of the proper hepatic artery.
• For subjects with hepatitis B or C cirrhosis: HBV-DNA ≤ 50 IU/mL and HCV-RNA below the limit of quantification at screening; for alcohol-related cirrhosis: documented abstinence for ≥ 6 months.
• Ability to understand the study procedures and willingness to comply; written informed consent obtained from the subject or legally authorised representative.

You may not qualify if:

• Known hypersensitivity to HCL001 Cell Injection or any of its excipients.
• Child-Pugh score ≥ 13 (see Appendix 1 for scoring and classification).
• Cavernous transformation of the portal vein.
• Serum total bilirubin \> 10 × the upper limit of normal (ULN).
• Hepatocellular carcinoma or any other concurrent malignancy.
• Inability to cooperate or unwillingness to comply with protocol requirements.
• Bleeding diathesis (e.g., haemophilia) or platelet count \< 40 × 10⁹/L despite platelet-raising therapy.
• Use of anticoagulant or antiplatelet agents within 7 days prior to dosing.
• Grade ≥ 3 hepatic encephalopathy within 3 months before dosing (grading criteria in Appendix 2).
• Previous transjugular intrahepatic portosystemic shunt (TIPS) or requirement/planned TIPS during the study.
• History of venous thrombosis, including portal vein thrombosis (PVT) Grade ≥ 2 (grading criteria in Appendix 9), deemed unsuitable by the investigator.
• Severe dysfunction of vital organs (heart, lung, brain, kidney):
• Pulmonary: severe emphysema, pulmonary embolism, or any pulmonary disorder significantly impairing pulmonary function.
• Cardiac history meeting any of the following:
• Decompensated heart failure (NYHA Class III-IV);

Sponsors & Collaborators

• Shanghai Huicun Medical Technology Co., Ltd.: lead

Study Sites (1)

Shanghai Jiao Tong University School of Medicine Affiliated Renji Hospita

Shanghai, Shanghai Municipality, China

Location

Study Officials

• Qiang Xia

  RenJi Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 24, 2025
• First Posted: March 23, 2026
• Study Start: September 23, 2025
• Primary Completion (Estimated): April 30, 2027
• Study Completion (Estimated): December 30, 2028
• Last Updated: March 23, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)