NCT07488546

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

A Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, and Preliminary Efficacy of HCL001 Cell Injection in Patients With Decompensated Cirrhosis

• Status: Enrolling By Invitation
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 39 (estimated)
• Sponsor: Shanghai Huicun Medical Technology Co., Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Detailed description

A single SRC will be convened during the Phase I dose-escalation stage to conduct dynamic safety oversight. The SRC will comprise investigators, the Sponsor, and/or medically qualified personnel designated by the Sponsor's delegate (contract research organization \[CRO\]). Detailed SRC procedures are provided in a separate SRC charter. * Begin with three subjects in the lowest cohort. * If zero of three subjects experience a DLT during the observation period, escalate to the next cohort. * If one of three subjects experiences a DLT, enroll an additional three subjects at the same dose level. * If ≤ 1/6 total subjects in the expanded cohort experience a DLT, escalate to the next cohort. * If \> 1/6 experience a DLT, dose escalation is terminated. * If ≥ 2 of the initial three subjects experience a DLT, escalation is stopped; the preceding dose level is declared the maximum tolerated dose (MTD). * If \> 1 subject experiences a DLT at any dose level, no further escalation will occur per protocol, and the preceding cohort will be designated the MTD. * If no DLT is observed at the highest predefined dose (4 × 10⁶ cells/kg), the investigator and the Sponsor will jointly determine whether exploration of higher doses is warranted. 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. Eligible subjects will be randomized to an investigational arm or a control arm. * If only one investigational dose is selected, the randomization ratio will be investigational arm : control arm = 2 : 1. * If two investigational doses are selected, the randomization ratio will be low-dose group : high-dose group : control group = 2 : 2 : 1. Each investigational cohort will enroll 20-30 subjects, and the control cohort will enroll 10-15 subjects; the control group will receive standard-of-care treatment only. The total anticipated enrollment for Phase II is 30-75 subjects.

Conditions

• Decompensated Cirrhosis

Interventions

Type	Name	Description
DRUG	Cohort 1 (1.0 × 10⁶ cells/kg)	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)	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)	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.

Timeline

• Start date: 2025-09-23
• Primary completion: 2027-04-30
• Completion: 2028-12-30
• First posted: 2026-03-23
• Last updated: 2026-03-23

Locations

1 site across 1 country: China