A Study of ADR-001 in Patients With Liver Cirrhosis

Decompensated Liver Cirrhosis Clinical Trial

Official title:

A Phase 1/2 Study of ADR-001 in Patients With Liver Cirrhosis

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03254758
• Other study ID #: ADR-001-01
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: July 20, 2017
• Est. completion date: April 13, 2023

Study information

• Verified date: July 2023
• Source: Rohto Pharmaceutical Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a first-in-human Phase1/2 study of ADR-001, adipose-derived mesenchymal stem cells (AD-MSCs). The safety and preliminary efficacy are evaluated in Phase 1 in patients with liver cirrhosis caused by Hepatitis C or Nonalcoholic Steatohepatitis and a recommended Phase 2 dose is determined by the evaluation. The exploratory efficacy and safety are investigated against the same target population in Phase 2.

Description:

Patients with decompensated liver cirrhosis (Child-Pugh score; Grade B) caused by Hepatitis C or Nonalcoholic Steatohepatitis are enrolled to the study. In Phase 1, one of 3 doses of AD-MSCs is administered by 1 hour single intravenous infusion. Patients are hospitalized for 1 week and a recommended dose for Phase 2 is determined by the evaluation of the safety and efficacy. In Phase 2, patients with the same disease criteria are enrolled and dosed to investigate the exploratory efficacy and safety.

The safety and efficacy are evaluated until 24 weeks after dosing both in Phase 1 and Phase 2.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 21
• Est. completion date: April 13, 2023
• Est. primary completion date: April 13, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 20 Years and older

Eligibility

Inclusion Criteria:

• Men and women = 20 years of age

• Chronic hepatitis C or nonalcoholic steatohepatitis(NASH)

• Child-Pugh grade B liver cirrhosis

• ECOG Performance Status = 2

Exclusion Criteria:

• Liver cirrhosis patients other than hepatitis C or NASH

• Malignant neoplasm (except hepatocellular carcinoma patients without recurrence more than 2 years)

• History of venous thrombosis or pulmonary embolism

• Serum creatinine = 2 mg/dL or T-Bil = 5.0 mg/dL

• Infection with hepatitis B, HIV, ATLV-1 or parvovirus B19

• Patients experienced transplantation or cell therapy

• Pregnancy or positive on pregnancy test

• Complications of significant heart disease, kidney disorder, or respiratory disease

• Drug or alcohol abuse

Related Conditions & MeSH terms

• Decompensated Liver Cirrhosis
• Fibrosis
• Liver Cirrhosis

Intervention

Biological:
• Mesenchymal stem cell
Phase1 The dose of AD-MSCs are escalated from low to mid and high step by step. Each administration is one time via intravenous infusion for one hour. Phase2 The recommended dose of ADR-001 is administrated once a week 4 times. The administration route and time is same method with Phase 1.

Locations

Country	Name	City	State
Japan	Niigata University Medical & Dental Hospital	Niigata
Japan	Nihon University Itabashi Hospital	Tokyo

Sponsors (1)

Lead Sponsor	Collaborator
Rohto Pharmaceutical Co., Ltd.

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety profile of ADR-001 including the incidence of adverse events (Phase 1)	Safety will be evaluated based on the medical review of adverse event reports and the results of clinical laboratory tests, vital sign, and physical examinations.	24 weeks
Primary	Improvement rate of Child-Pugh score (Phase 2)	Improvement rate of Child-Pugh score from the baseline will be evaluated.	24 weeks
Secondary	Change of liver function evaluated by Child-Pugh score (Phase 1)	Change of liver function from the baseline will be evaluated by Child-Pugh score.	24 weeks
Secondary	Improvement rate of Child-Pugh score (Phase 1)	Improvement rate of Child-Pugh score from the baseline will be evaluated.	24 weeks
Secondary	Improvement rate of Child-Pugh grade (Phase 1)	Improvement rate of Child-Pugh grade from the baseline will be evaluated.	24 weeks
Secondary	Change of liver function evaluated by Child-Pugh score (Phase 2)	Change of liver function from the baseline will be evaluated by Child-Pugh score.	24 weeks
Secondary	Improvement rate of Child-Pugh grade (Phase 2)	Improvement rate of Child-Pugh grade from the baseline will be evaluated.	24 weeks
Secondary	Safety profile of ADR-001 including the incidence of adverse events (Phase 2)	Safety will be evaluated based on the medical review of adverse event reports and the results of clinical laboratory tests, vital sign, and physical examinations.	24 weeks