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Phase I/II Trial of Human Umbilical Cord Mesenchymal Stromal Cells for UDCA-Refractory Primary Biliary Cholangitis

NCT07795151 · Status: NOT_YET_RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 68

Last updated 2026-08-31

No results posted yet for this study

Summary

This phase I/II clinical trial evaluates human umbilical cord-derived mesenchymal stromal cell (hUC-MSC) injection in patients with primary biliary cholangitis (PBC). The phase I component uses a 3+3 dose-escalation design with separate single-dose and multiple-dose stages to assess safety and tolerability, establish the maximum tolerated dose and recommended phase II dose, while monitoring adverse events, vital signs, laboratory parameters, and immunogenicity, and to explore preliminary efficacy signals. The phase II component is a randomized, double-blind, placebo-controlled trial with the primary endpoint of composite response of alkaline phosphatase and bilirubin at 12 weeks to evaluate efficacy, alongside continuous safety surveillance. Systematic measurements of liver function, cholestasis, immune markers, quality of life, and pruritus scores are incorporated to investigate mechanisms and potential biomarkers, aiming to generate robust clinical evidence that supports future development and clinical translation of hUC-MSC therapy for PBC.

Conditions

Interventions

DRUG

Standard background therapy

UDCA capsule 250 mg, orally, 13-15 mg/kg/day, taken with a small amount of water. This background therapy is administered only to participants who have been on a stable dose of UDCA for at least 6 months prior to enrollment (and stable for ≥3 months before screening). Participants who are intolerant to UDCA (and have not used UDCA for ≥3 months before enrollment) do not receive UDCA during the study.

DRUG

Placebo

Matching placebo solution (e.g., 5% human serum albumin in 0.9% saline) administered via peripheral intravenous infusion at Week 0, Week 1, and Week 2 (once weekly for 3 infusions).

BIOLOGICAL

hUC-MSC Dose Level 1

Human umbilical cord-derived mesenchymal stromal cells, planned at 1.5×10⁸ cells per infusion (final dose subject to confirmation based on phase I MTD/RP2D). Administered via peripheral intravenous infusion at Week 0, Week 1, and Week 2 (once weekly for 3 infusions).

BIOLOGICAL

hUC-MSC Dose Level 2

Human umbilical cord-derived mesenchymal stromal cells, planned at 2.0×10⁸ cells per infusion (final dose subject to confirmation based on phase I MTD/RP2D). Administered via peripheral intravenous infusion at Week 0, Week 1, and Week 2 (once weekly for 3 infusions).

Sponsors & Collaborators

  • Beijing 302 Hospital

    lead OTHER

Principal Investigators

  • Fu-Sheng Wang, MD, PhD · The Fifth Medical Center of PLAGeneral Hospital

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-08-31
Primary Completion
2029-08-31
Completion
2030-08-31

Countries

  • China

Study Locations

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Entities

Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07795151 on ClinicalTrials.gov