Clinical trial NCT05331872: Umbilical Cord-derived Mesenchymal Stem Cell Infusion in the Management of Adult Liver Cirrhosis
Official title: A Phase I, Open-label Trial on the Safety and Efficacy of Human Umbilical Cord-derived Mesenchymal Stem Cell Administration in the Management of Adult Liver Cirrhosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Liver Cirrhosis
Lead sponsor: Vinmec Research Institute of Stem Cell and Gene Technology
Sponsor class: OTHER
First posted: 2022-04-18
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a phase I, open label, single arm trial using UC-MSCs to treat patients with liver cirrhosis that includes 20 patients. The primary outcome measure will be change in MELD score at 3, 6, and 12 months after UC-MSC transplantation from baseline. The safety is assessed by frequency and severity of the adverse event or serious adverse event associated with stem cell injection. This study could reconfirm the efficacy of stem cell transplantation for liver cirrhosis and would open a novel cell therapy for the treatment of adult liver cirrhosis.

Detailed description: Liver cirrhosis is defined as the histological development of regenerative nodules surrounded by fibrous bands and causes by many forms of liver diseases and conditions such as hepatitis and chronic alcoholism. Liver cirrhosis has become one of the major causes of morbidity and mortality with over one million people died due to cirrhosis in 2010 worldwide. Currently, the only curative treatment for end-stage liver disease is liver transplantation. However, the shortage in donor organ availability and the side-effect associated with long-term immunosuppression after transplant requires the development of new alternative therapies for liver cirrhosis. Recent clinical trials demonstrate that bone marrow-derived stem cells (BMDSCs) as well as umbilical cord-derive mesenchymal stem cells (UC-MSCs) improve liver function in patients with liver cirrhosis. In Vietnam, however, there are no clinical trials to date that investigate the potential of UC-MSCs for liver cirrhosis management. This is a phase I, open label, single arm trial using UC-MSCs to treat patients with liver cirrhosis that includes 20 patients. The primary outcome measure will be change in MELD score at 3, 6, and 12 months after UC-MSC transplantation from baseline. The safety is assessed by frequency and severity of the adverse event or serious adverse event associated with stem cell injection. This study could reconfirm the efficacy of stem cell transplantation for liver cirrhosis and would open a novel cell therapy for the treatment of adult liver cirrhosis

Eligibility criteria: Inclusion Criteria:

* Aged 18 to 70 years old.
* Patients diagnosed of liver cirrhosis: splenomegaly, elevated hepatic enzymes and/or esophageal varices detected by endoscopy, or liver biopsy demonstrated liver cirrhosis
* Be willing to complete the study and sign the consent form.

Exclusion Criteria:

* Younger than 18 or older than 70 years old
* Recent infection
* Patients with history of chronic diseases such as cancer, chronic hepatitis, chronic kidney disease.
* Any clinically significant blood coagulation disorder(s)

Primary outcomes:
measure: Number of Adverse events and serious adverse events (AEs or SAEs)
timeFrame: up to the 12-month period following the first UC-MSCs infusion
Secondary outcomes:
measure: Change of liver function through the Model for End Stage Liver Disease (MELD) score
timeFrame: 3 months, 6 months, 12 months after the UC-MSCs infusion
measure: Change in health-related quality of life using Chronic Liver Disease Questionnaire - (CLDQ)
timeFrame: 3 months, 6 months, 12 months after the UC-MSCs infusion
Other outcomes:
Locations:
city: Hanoi
country: Vietnam
facility: Vinmec Research Institute of Stem Cell and Gene Technology
Study officials:
affiliation: Vinmec Research Institute of Stem Cell and Gene Technology
name: Liem Nguyen, PHD
role: PRINCIPAL_INVESTIGATOR
References: