A Study of ADR-001 in Patients With Liver Cirrhosis

ROHTO Pharmaceutical

Status and phase: Completed; Phase 2; Phase 1

Conditions: Decompensated Liver Cirrhosis

Treatments: Biological: Mesenchymal stem cell

Study type: Interventional

Funder types: Industry

Identifiers

NCT03254758

ADR-001-01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 21 patients

Sex: All

Ages: 20+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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21 participants in 1 patient group

Mesenchymal stem cell

Experimental group

Description:

Phase 1 Dose escalation : low Mid High Single administalation of ADR-001 Phase 2 The recommended dose of ADR-001

Treatment:

Biological: Mesenchymal stem cell