Clinical trial NCT03254758: 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
Overall status: COMPLETED
Phase: PHASE1
PHASE2
Conditions: Decompensated Liver Cirrhosis
Lead sponsor: Rohto Pharmaceutical Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2017-08-18
Enrollment: 21
Sex: ALL
Minimum age (years): 20
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Safety profile of ADR-001 including the incidence of adverse events (Phase 1)
timeFrame: 24 weeks
measure: Improvement rate of Child-Pugh score (Phase 2)
timeFrame: 24 weeks
Secondary outcomes:
measure: Change of liver function evaluated by Child-Pugh score (Phase 1)
timeFrame: 24 weeks
measure: Improvement rate of Child-Pugh score (Phase 1)
timeFrame: 24 weeks
measure: Improvement rate of Child-Pugh grade (Phase 1)
timeFrame: 24 weeks
measure: Change of liver function evaluated by Child-Pugh score (Phase 2)
timeFrame: 24 weeks
measure: Improvement rate of Child-Pugh grade (Phase 2)
timeFrame: 24 weeks
measure: Safety profile of ADR-001 including the incidence of adverse events (Phase 2)
timeFrame: 24 weeks
Other outcomes:
Locations:
city: Niigata
country: Japan
facility: Niigata University Medical & Dental Hospital
city: Tokyo
country: Japan
facility: Nihon University Itabashi Hospital
Study officials:
affiliation: Niigata University Medical & Dental Hospital
name: Shuji Terai, MD
role: PRINCIPAL_INVESTIGATOR
References: