A Phase 1/2 interventional study of Standard background therapy and Placebo in Primary Biliary Cholangitis, sponsored by Beijing 302 Hospital. Not yet recruiting at 1 site in China. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-08-31.
Sponsored by Beijing 302 Hospital · Phase 1/2, Interventional, and Treatment
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.
This is a phase I/II clinical trial. The phase I component uses a conventional "3+3" dose-escalation design with three dose levels: low (1.0×10⁸ cells), medium (1.5×10⁸ cells), and high (2.0×10⁸ cells). Each dose cohort enrolls 3-6 evaluable participants, for a total of 18-36 evaluable subjects. Phase Ia is a single-dose stage, with dose-limiting toxicity (DLT) assessed up to 7 days after the infusion. Phase Ib is a multiple-dose stage, in which participants receive one intravenous infusion weekly for 3 consecutive weeks, and DLT is evaluated up to 28 days after the first dose. The aim is to determine the maximum tolerated dose (MTD) and/or the recommended phase II dose (RP2D).
The phase II component plans to enroll 50 patients with primary biliary cholangitis, who will be randomized in a 2:2:1 ratio to receive either dose level 1 (1.5×10⁸ cells), dose level 2 (2.0×10⁸ cells), or placebo control, with the final dose to be confirmed based on the results of phase I. The treatment regimen consists of one intravenous infusion weekly for 3 consecutive weeks. The primary objective of phase II is to evaluate preliminary efficacy.
214 studies on the registry are indexed under Liver Cirrhosis, Biliary; 48 are open to participants now.
This study's planned enrollment of 68 is above the median of 60 across 151 interventional studies indexed under Liver Cirrhosis, Biliary.
Browse Liver Cirrhosis, Biliary studies →Beijing 302 Hospital is the lead sponsor of 88 studies on the registry; 31 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Diagnosis of PBC per the 2025 PBC Guideline of the National Health Commission of China, meeting at least 2 of the following 3 criteria:
Exclusion Criteria:
Use of prohibited medications within specified :
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
INR, PT and aPTT (clotting tests)
Blood tests of how long blood takes to clot. The INR is the prothrombin time (PT) put on a common scale; about 1 is usual for someone not taking a blood thinner.
Criteria often make an exception for people on a blood thinner such as warfarin, whose INR is raised on purpose.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants receive placebo infusions via peripheral intravenous infusion once weekly for 3 consecutive weeks (at Week 0, Week 1, and Week 2), in addition to background therapy as per inclusion criteria (i.e., continued stable UDCA for patients on prior UDCA, or no UDCA for those intolerant to UDCA).
Drug: Standard background therapy · Drug: Placebo
Participants receive human umbilical cord-derived mesenchymal stromal cells (hUC-MSC) at a planned dose of 1.5×10⁸ cells per infusion (final dose to be confirmed based on the maximum tolerated dose / recommended phase II dose determined from phase I results) via peripheral intravenous infusion once weekly for 3 consecutive weeks (at Week 0, Week 1, and Week 2), in addition to background therapy as per inclusion criteria (continued stable UDCA for prior users, or no UDCA for intolerant patients).
Drug: Standard background therapy · Biological: hUC-MSC Dose Level 1
Participants receive hUC-MSC at a planned dose of 2.0×10⁸ cells per infusion (final dose to be confirmed based on the MTD/RP2D from phase I) via peripheral intravenous infusion once weekly for 3 consecutive weeks (at Week 0, Week 1, and Week 2), in addition to background therapy as per inclusion criteria (continued stable UDCA for prior users, or no UDCA for intolerant patients).
Drug: Standard background therapy · Biological: hUC-MSC Dose Level 2
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.
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).
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).
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).
Incidence of Dose-Limiting Toxicity (DLT) in Phase I
Occurrence of DLT during the DLT observation period (single-dose cohort: 7 days after infusion; multiple-dose cohort: 28 days after first infusion), graded by CTCAE v6.0.
Time frame: Up to Day 7 (single-dose) or up to Day 28 (multiple-dose).
Incidence of Treatment-Emergent Adverse Events and Serious Adverse Events (Phase I)
Safety and tolerability assessed by monitoring TEAEs, SAEs, and clinically significant changes in vital signs, physical examination, laboratory parameters, and 12-lead ECG.
Time frame: Up to Day 7 (single-dose) or up to Day 28 (multiple-dose).
Maximum Tolerated Dose (MTD) of hUC-MSC in PBC Patients (Phase I)
Determination of MTD based on DLT occurrence in the 3+3 dose-escalation phase I; MTD is the highest dose with ≤1/6 participants experiencing DLT.
Time frame: At completion of phase I dose escalation (after DLT evaluation).
Composite Biochemical Response at Week 12 (Phase II)
Proportion of participants achieving all three: ALP \< 1.67×ULN, ALP reduction ≥15% from baseline, and total bilirubin ≤ ULN at Week 12.
Time frame: Week 12
Proportion with ALP <1.67×ULN or ALP Reduction ≥15% or TB ≤ULN at Week 12 (Phase I)
Exploratory efficacy endpoint for phase I - participants meeting at least one of the three criteria at Week 12.
Time frame: Week 12
Change from Baseline in Alkaline Phosphatase (ALP) (Phase I)
Absolute and percentage changes from baseline at each specified visit.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96
Change from Baseline in Total Bilirubin (TB) (Phase I)
Absolute and percentage changes from baseline at each specified visit.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96
Change from Baseline in Direct Bilirubin (DBIL) (Phase I)
Absolute and percentage changes from baseline at each specified visit.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96
Change from Baseline in Alanine Aminotransferase (ALT) (Phase I)
Absolute and percentage changes from baseline.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Aspartate Aminotransferase (AST) (Phase I)
Absolute and percentage changes from baseline.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Gamma-Glutamyl Transferase (GGT) (Phase I)
Absolute and percentage changes from baseline.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Primary Biliary Cholangitis-40 (PBC-40) Total Score (Phase I)
Scale range: 40-200; higher scores indicate worse disease-specific quality of life.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Pruritus Numerical Rating Scale (NRS) Score (Phase I)
Scale range: 0-10; higher scores indicate worse itch severity.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Itchy Quality of Life (ItchyQoL) Total Score (Phase I)
Scale range: 22-110; higher scores indicate worse itch-related quality of life.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Alkaline Phosphatase (ALP) (Phase II)
Absolute and percentage changes from baseline at each specified visit.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Total Bilirubin (TB) (Phase II)
Absolute and percentage changes from baseline at each specified visit.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Direct Bilirubin (DBIL) (Phase II)
Absolute and percentage changes from baseline at each specified visit.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Alanine Aminotransferase (ALT) (Phase II)
Absolute and percentage changes from baseline.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Aspartate Aminotransferase (AST) (Phase II)
Absolute and percentage changes from baseline.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Gamma-Glutamyl Transferase (GGT) (Phase II)
Absolute and percentage changes from baseline.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Primary Biliary Cholangitis-40 (PBC-40) Total Score (Phase II)
Scale range: 40-200; higher scores indicate worse disease-specific quality of life.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96
Change from Baseline in Pruritus Numerical Rating Scale (NRS) Score (Phase II)
Scale range: 0-10; higher scores indicate worse itch severity.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96
Change from Baseline in Itchy Quality of Life (ItchyQoL) Total Score (Phase II)
Scale range: 22-110; higher scores indicate worse itch-related quality of life.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96
Change from Baseline in Serum Total Bile Acids (TBA) (Phase II)
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in 7α-Hydroxy-4-cholesten-3-one (C4) (Phase II)
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Fibroblast Growth Factor 19 (FGF-19) (Phase II)
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48, 72, 96.
Change from Baseline in Liver Stiffness by Transient Elastography (Phase II)
Change in liver stiffness measurement (kPa) at Week 12.
Time frame: Baseline and Week 12.
Change from Baseline in Immunoglobulins (IgG, IgM) - (Phase I)
Exploratory immune function endpoint.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48.
Change from Baseline in Lymphocyte Subsets (Phase II)
Exploratory immune markers including CD3⁺, CD4⁺, CD8⁺, CD4⁺/CD8⁺ ratio.
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48.
Change from Baseline in Cytokines (Phase II)
Exploratory cytokine marker including Interleukin-6 (IL-6), Interleukin-10 (IL-10), Tumor Necrosis Factor-α (TNF-α), and Interferon-γ (IFN-γ).
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48.
Change from Baseline in C-Reactive Protein (CRP) (Phase II)
Time frame: Baseline, Day 3, Week 1, 2, 4, 12, 24, 48.
Plan to share: Yes — After approval from the steering committee and the Human Genetic ResourcesAdministration of China, this trial data can be shared with qualifying researchers whosubmit a proposal with a valuable research question. A contract should be signed.
This study is not yet recruiting, as verified in May 2026. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Beijing 302 Hospital