Decompensated Liver CirrhosisPhase 1/2NCT03254758
A Study of ADR-001 in Patients With Liver Cirrhosis
A Phase 1/2 study testing Mesenchymal stem cell in people with Decompensated Liver Cirrhosis, run by Rohto Pharmaceutical Co., Ltd. at 2 sites in Japan.
Trial 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. Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Decompensated Liver Cirrhosis
- What is being tested?
- Mesenchymal stem cell
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 2 sitesJapan
- Study dates
- 20 Jul 2017to 13 Apr 2023
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Men and women ≥ 20 years of age
- Chronic hepatitis C or nonalcoholic steatohepatitis(NASH)
- Child-Pugh grade B liver cirrhosis
- ECOG Performance Status ≤ 2
Who cannot join
- 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
Show all 9Show fewer
- Pregnancy or positive on pregnancy test
- Complications of significant heart disease, kidney disorder, or respiratory disease
- Drug or alcohol abuse
Trial locations and contacts
2 sites in 1 country.
Japan2 sites
Niigata University Medical & Dental Hospital
Niigata, 951-8510
Nihon University Itabashi Hospital
Tokyo, 173-8610
About this study
Official title: A Phase 1/2 Study of ADR-001 in Patients With Liver Cirrhosis
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.
Read the 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.
- Conditions
- Decompensated Liver Cirrhosis
- Being tested
- Mesenchymal stem cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| Mesenchymal stem cell | Experimental | Phase 1 Dose escalation : low Mid High Single administalation of ADR-001
Phase 2 The recommended dose of ADR-001 Receives: Biological: Mesenchymal stem cell |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 24 weeks
Improvement rate of Child-Pugh score (Phase 2)
Improvement rate of Child-Pugh score from the baseline will be evaluated.
Measured: 24 weeks
Secondary outcomes (6)
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.
Measured: 24 weeks
Improvement rate of Child-Pugh score (Phase 1)
Improvement rate of Child-Pugh score from the baseline will be evaluated.
Measured: 24 weeks
Improvement rate of Child-Pugh grade (Phase 1)
Improvement rate of Child-Pugh grade from the baseline will be evaluated.
Measured: 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.
Measured: 24 weeks
Improvement rate of Child-Pugh grade (Phase 2)
Improvement rate of Child-Pugh grade from the baseline will be evaluated.
Measured: 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.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2017
- Study start20 Jul 2017
- Primary completion13 Apr 2023
- Study completion13 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Decompensated Liver CirrhosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rohto Pharmaceutical Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Shuji Terai, MD, Principal investigator, Niigata University Medical & Dental Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03254758
- Sponsor's protocol ID
- ADR-001-01
- Status last verified
- Jul 2023
- First submitted
- 3 Aug 2017
- Last updated
- 6 Jul 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2 Study of ADR-001 in Patients With Liver Cirrhosis
- ClinicalTrials.gov ID
- NCT03254758
- Acronym / sponsor ID
- ADR-001-01
- Conditions
- Decompensated Liver Cirrhosis
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Rohto Pharmaceutical Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 20 Jul 2017 (actual)
- Primary completion
- 13 Apr 2023 (actual)
- Study completion
- 13 Apr 2023 (actual)
- First posted
- 18 Aug 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Mesenchymal stem cell | Experimental | Biological: Mesenchymal stem cellPhase 1 Dose escalation : low Mid High Single administalation of ADR-001 Phase 2 The recommended dose of ADR-001 |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Men and women ≥ 20 years of age
- Chronic hepatitis C or nonalcoholic steatohepatitis(NASH)
- Child-Pugh grade B liver cirrhosis
- ECOG Performance Status ≤ 2
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mesenchymal stem cellPhase1 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. | Biological | Mesenchymal stem cell |
Primary outcome
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.
Improvement rate of Child-Pugh score (Phase 2)
Time frame: 24 weeks
Improvement rate of Child-Pugh score from the baseline will be evaluated.
6 secondary outcomes
Change of liver function evaluated by Child-Pugh score (Phase 1)
Time frame: 24 weeks
Improvement rate of Child-Pugh score (Phase 1)
Time frame: 24 weeks
Improvement rate of Child-Pugh grade (Phase 1)
Time frame: 24 weeks
Change of liver function evaluated by Child-Pugh score (Phase 2)
Time frame: 24 weeks
Improvement rate of Child-Pugh grade (Phase 2)
Time frame: 24 weeks
Safety profile of ADR-001 including the incidence of adverse events (Phase 2)
Time frame: 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Shuji Terai, MD principal investigator
Niigata University Medical & Dental Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Mesenchymal stem cell in people with Decompensated Liver Cirrhosis, run by Rohto Pharmaceutical Co., Ltd. at 2 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety profile of ADR-001 including the incidence of adverse events (Phase 1) (time frame: 24 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jul 2023.