Back to resultsA Study of ADR-001 in Patients With Liver Cirrhosis
Primary Purpose
Decompensated Liver Cirrhosis
Status
Completed
Phase
Phase 1
Locations
Japan
Study Type
Interventional
Intervention
Mesenchymal stem cell
Sponsored by
About this trial
This is an interventional treatment trial for Decompensated Liver Cirrhosis
Eligibility Criteria
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
Sites / Locations
- Niigata University Medical & Dental Hospital
- Nihon University Itabashi Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Mesenchymal stem cell
Arm Description
Phase 1 Dose escalation : low Mid High Single administalation of ADR-001 Phase 2 The recommended dose of ADR-001
Outcomes
Primary Outcome Measures
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.
Improvement rate of Child-Pugh score (Phase 2)
Improvement rate of Child-Pugh score from the baseline will be evaluated.
Secondary Outcome Measures
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.
Improvement rate of Child-Pugh score (Phase 1)
Improvement rate of Child-Pugh score from the baseline will be evaluated.
Improvement rate of Child-Pugh grade (Phase 1)
Improvement rate of Child-Pugh grade from the baseline will be evaluated.
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.
Improvement rate of Child-Pugh grade (Phase 2)
Improvement rate of Child-Pugh grade from the baseline will be evaluated.
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.
Full Information
NCT ID
NCT03254758
First Posted
August 3, 2017
Last Updated
July 4, 2023
Sponsor
Rohto Pharmaceutical Co., Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT03254758
Brief Title
A Study of ADR-001 in Patients With Liver Cirrhosis
Official Title
A Phase 1/2 Study of ADR-001 in Patients With Liver Cirrhosis
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
July 20, 2017 (Actual)
Primary Completion Date
April 13, 2023 (Actual)
Study Completion Date
April 13, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Rohto Pharmaceutical Co., Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief 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.
Detailed 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Decompensated Liver Cirrhosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
21 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Mesenchymal stem cell
Arm Type
Experimental
Arm Description
Phase 1 Dose escalation : low Mid High Single administalation of ADR-001
Phase 2 The recommended dose of ADR-001
Intervention Type
Biological
Intervention Name(s)
Mesenchymal stem cell
Intervention Description
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.
Primary Outcome Measure Information:
Title
Safety profile of ADR-001 including the incidence of adverse events (Phase 1)
Description
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.
Time Frame
24 weeks
Title
Improvement rate of Child-Pugh score (Phase 2)
Description
Improvement rate of Child-Pugh score from the baseline will be evaluated.
Time Frame
24 weeks
Secondary Outcome Measure Information:
Title
Change of liver function evaluated by Child-Pugh score (Phase 1)
Description
Change of liver function from the baseline will be evaluated by Child-Pugh score.
Time Frame
24 weeks
Title
Improvement rate of Child-Pugh score (Phase 1)
Description
Improvement rate of Child-Pugh score from the baseline will be evaluated.
Time Frame
24 weeks
Title
Improvement rate of Child-Pugh grade (Phase 1)
Description
Improvement rate of Child-Pugh grade from the baseline will be evaluated.
Time Frame
24 weeks
Title
Change of liver function evaluated by Child-Pugh score (Phase 2)
Description
Change of liver function from the baseline will be evaluated by Child-Pugh score.
Time Frame
24 weeks
Title
Improvement rate of Child-Pugh grade (Phase 2)
Description
Improvement rate of Child-Pugh grade from the baseline will be evaluated.
Time Frame
24 weeks
Title
Safety profile of ADR-001 including the incidence of adverse events (Phase 2)
Description
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.
Time Frame
24 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
20 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Shuji Terai, MD
Organizational Affiliation
Niigata University Medical & Dental Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Niigata University Medical & Dental Hospital
City
Niigata
ZIP/Postal Code
951-8510
Country
Japan
Facility Name
Nihon University Itabashi Hospital
City
Tokyo
ZIP/Postal Code
173-8610
Country
Japan
12. IPD Sharing Statement
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A Study of ADR-001 in Patients With Liver Cirrhosis
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