A Phase 1/2 interventional study of Mesenchymal stem cell in Decompensated Liver Cirrhosis, sponsored by Rohto Pharmaceutical Co., Ltd.. Completed at 2 sites in Japan. Open to participants aged 20 Years and older. Per ClinicalTrials.gov, last updated 2023-07-06.
Sponsored by Rohto Pharmaceutical Co., Ltd. · Phase 1/2, Interventional, and Treatment
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.
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.
Exclusion Criteria:
Phase 1 Dose escalation : low Mid High Single administalation of ADR-001 Phase 2 The recommended dose of ADR-001
Biological: Mesenchymal stem cell
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.
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.
Time frame: 24 weeks
Improvement rate of Child-Pugh score (Phase 2)
Improvement rate of Child-Pugh score from the baseline will be evaluated.
Time frame: 24 weeks
Change of liver function evaluated by Child-Pugh score (Phase 1)
Change of liver function from the baseline will be evaluated by Child-Pugh score.
Time frame: 24 weeks
Improvement rate of Child-Pugh score (Phase 1)
Improvement rate of Child-Pugh score from the baseline will be evaluated.
Time frame: 24 weeks
Improvement rate of Child-Pugh grade (Phase 1)
Improvement rate of Child-Pugh grade from the baseline will be evaluated.
Time frame: 24 weeks
Change of liver function evaluated by Child-Pugh score (Phase 2)
Change of liver function from the baseline will be evaluated by Child-Pugh score.
Time frame: 24 weeks
Improvement rate of Child-Pugh grade (Phase 2)
Improvement rate of Child-Pugh grade from the baseline will be evaluated.
Time frame: 24 weeks
Safety profile of ADR-001 including the incidence of adverse events (Phase 2)
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.
Time frame: 24 weeks
This study is completed, as verified in Jul 2023. You cannot join it, but the record below documents what was studied.
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Rohto Pharmaceutical Co., Ltd.