A Phase 1/2 Study of ADR-001 in Patients With Liver Cirrhosis
Trial Snapshot
- Phase
- Phase 1
- Status
- Completed
- Enrollment
- 21
- Locations
- 2
- Primary Endpoint
- Improvement rate of Child-Pugh score (Phase 2)
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 20 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Mesenchymal stem cell
Phase 1 Dose escalation : low Mid High Single administalation of ADR-001
Phase 2 The recommended dose of ADR-001
Intervention: Mesenchymal stem cell (Biological)
Outcomes
Primary Outcomes
Improvement rate of Child-Pugh score (Phase 2)
Time Frame: 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)
Time Frame: 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 Outcomes
- Change of liver function evaluated by Child-Pugh score (Phase 2)(24 weeks)
- Change of liver function evaluated by Child-Pugh score (Phase 1)(24 weeks)
- Safety profile of ADR-001 including the incidence of adverse events (Phase 2)(24 weeks)
- Improvement rate of Child-Pugh score (Phase 1)(24 weeks)
- Improvement rate of Child-Pugh grade (Phase 1)(24 weeks)
- Improvement rate of Child-Pugh grade (Phase 2)(24 weeks)