A Study of ADR-001 in Patients With Liver Cirrhosis
- Conditions
- Decompensated Liver Cirrhosis
- Interventions
- Biological: Mesenchymal stem cell
- Registration Number
- NCT03254758
- Lead Sponsor
- Rohto Pharmaceutical Co., Ltd.
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 21
- Men and women ≥ 20 years of age
- Chronic hepatitis C or nonalcoholic steatohepatitis(NASH)
- Child-Pugh grade B liver cirrhosis
- ECOG Performance Status ≤ 2
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Mesenchymal stem cell Mesenchymal stem cell Phase 1 Dose escalation : low Mid High Single administalation of ADR-001 Phase 2 The recommended dose of ADR-001
- Primary Outcome Measures
Name Time Method Improvement rate of Child-Pugh score (Phase 2) 24 weeks Improvement rate of Child-Pugh score from the baseline will be evaluated.
Safety profile of ADR-001 including the incidence of adverse events (Phase 1) 24 weeks 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.
- Secondary Outcome Measures
Name Time Method Change of liver function evaluated by Child-Pugh score (Phase 2) 24 weeks Change of liver function from the baseline will be evaluated by Child-Pugh score.
Change of liver function evaluated by Child-Pugh score (Phase 1) 24 weeks Change of liver function from the baseline will be evaluated by Child-Pugh score.
Safety profile of ADR-001 including the incidence of adverse events (Phase 2) 24 weeks 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 1) 24 weeks Improvement rate of Child-Pugh score from the baseline will be evaluated.
Improvement rate of Child-Pugh grade (Phase 1) 24 weeks Improvement rate of Child-Pugh grade from the baseline will be evaluated.
Improvement rate of Child-Pugh grade (Phase 2) 24 weeks Improvement rate of Child-Pugh grade from the baseline will be evaluated.
Trial Locations
- Locations (2)
Niigata University Medical & Dental Hospital
🇯🇵Niigata, Japan
Nihon University Itabashi Hospital
🇯🇵Tokyo, Japan