NCT03254758

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
21

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jul 2017

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 20, 2017

Completed
14 days until next milestone

First Submitted

Initial submission to the registry

August 3, 2017

Completed
15 days until next milestone

First Posted

Study publicly available on registry

August 18, 2017

Completed
5.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 13, 2023

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 13, 2023

Completed
Last Updated

July 6, 2023

Status Verified

July 1, 2023

Enrollment Period

5.7 years

First QC Date

August 3, 2017

Last Update Submit

July 4, 2023

Conditions

Decompensated Liver Cirrhosis

Outcome Measures

Primary Outcomes (2)

  • 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.

    24 weeks

  • Improvement rate of Child-Pugh score (Phase 2)

    Improvement rate of Child-Pugh score from the baseline will be evaluated.

    24 weeks

Secondary Outcomes (6)

  • Change of liver function evaluated by Child-Pugh score (Phase 1)

    24 weeks

  • Improvement rate of Child-Pugh score (Phase 1)

    24 weeks

  • Improvement rate of Child-Pugh grade (Phase 1)

    24 weeks

  • Change of liver function evaluated by Child-Pugh score (Phase 2)

    24 weeks

  • Improvement rate of Child-Pugh grade (Phase 2)

    24 weeks

  • +1 more secondary outcomes

Study Arms (1)

Mesenchymal stem cell

EXPERIMENTAL

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

Biological: Mesenchymal stem cell

Interventions

Mesenchymal stem cellBIOLOGICAL

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.

Mesenchymal stem cell

Eligibility Criteria

Age20 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Men and women ≥ 20 years of age
  • Chronic hepatitis C or nonalcoholic steatohepatitis(NASH)
  • Child-Pugh grade B liver cirrhosis
  • ECOG Performance Status ≤ 2

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Niigata University Medical & Dental Hospital

Niigata, 951-8510, Japan

Location

Nihon University Itabashi Hospital

Tokyo, 173-8610, Japan

Location

Study Officials

  • Shuji Terai, MD

    Niigata University Medical & Dental Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 3, 2017

First Posted

August 18, 2017

Study Start

July 20, 2017

Primary Completion

April 13, 2023

Study Completion

April 13, 2023

Last Updated

July 6, 2023

Record last verified: 2023-07

Locations

JP(2)