Liver FailurePhase 1/2NCT01218464
Safety and Efficacy of Human Mesenchymal Stem Cells for Treatment of Liver Failure
A Phase 1/2 study testing Conventional plus MSC treatment and Conventional plus pacebo treatment in people with Liver Failure and Mesenchymal Stem Cells, run by Beijing 302 Hospital at 1 site in China.
Trial summary
Liver failure (LF) is a dramatic clinical syndrome with massive necrosis of liver cells. and liver transplantation is the only available therapeutic option for patients suffering with this condition. However, lack of donors, surgical complications, rejection, and high cost are serious problems. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Liver Failure, Mesenchymal Stem Cells
- What is being tested?
- Conventional plus MSC treatment and Conventional plus pacebo treatment
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteChina
- Study dates
- Mar 2009to Mar 2014
- 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 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Aged 18-70 years
- Liver failure
- Negative pregnancy test (female patients in fertile age)
- Written consent
Who cannot join
- Hepatocellular carcinoma or other malignancies
- Severe problems in other vital organs(e.g.the heart,renal or lungs)
- Pregnant or lactating women
- Severe bacteria infection
- Anticipated with difficulty of follow-up observation
- Other candidates who are judged to be not applicable to this study by doctors
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Beijing 302 Hospital
Beijing, Beijing Municipality, 100039
Ming Shi, Professor, 86-10-63879735 ext. 2015.12, shiming302@sina.com
About this study
Official title: Phase Ⅰ/Ⅱ Study of Human Umbilical Cord Derived Mesenchymal Stem Cells (UC-MSCs) for Treatment of Liver Failure
Liver failure (LF) is a dramatic clinical syndrome with massive necrosis of liver cells. and liver transplantation is the only available therapeutic option for patients suffering with this condition. However, lack of donors, surgical complications, rejection, and high cost are serious problems. Previous study showed that bone marrow derived mesenchymal stem cells (BM-MSCs) replace hepatocytes in injured liver, and effectively rescue experimental liver failure and contribute to liver regeneration. In this study, the patients with LF will undergo administration of human umbilical cord mesenchymal stem cells (UC-MSCs) via peripheral vein transfusion to evaluate the safty and efficacy of UC-MSCs treatment for these patients.
Read the detailed description
Liver failure (LF) is a severe life-threatening condition, and is a dramatic clinical syndrome with massive necrosis of liver cells, and liver transplantation is the only available therapeutic option for patients suffering with this condition. However, lack of donors, surgical complications, rejection, and high cost are serious problems. Since current therapeutic options for LF that is usually with extremely poor prognosis are still limited, recent studies indicate that mesenchymal stem cells (MSCs), due to their function in immune modulation and liver-damage repair, are of great therapeutic potential for this disease. Previous study showed that bone marrow derived mesenchymal stem cells (BM-MSCs) replace hepatocytes in injured liver, and effectively rescue experimental liver failure and contribute to liver regeneration.The purpose of this study is to investigate the safety and initial efficacy of human umbilical cord MSC (UC-MSCs) treatment for patients with LF. In this study, MSCs were isolated from umbilical cord and generated in appropriate growth medium. 50 LF patients with LF received i.v. transfusion of 0.5-1.0×106 cells/kg of MSCs as the treated group and other 20 LF patients with LF were transfused with placebo without MSCs as control group. All 70 of them received the routine management for liver failure. During the 2-year follow up, the evaluation of safty and efficacy will be undergone to help to establish innovative cell-based therapies for the treatment of diseases.
- Conditions
- Liver Failure, Mesenchymal Stem Cells
- Being tested
- Conventional plus MSC treatment, Conventional plus pacebo treatment
- Keywords
- Liver Failure, Mesenchymal Stem Cells, Model for End-Stage Liver Disease, Ascite, Serum Albumin
- Medical subject headings
- Liver Failure
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Conventional plus MSC treatmentDrug
Participants received conventional treatment and taken i.v., once per 4 week, at a dose of 0.5*10E6 MSC/kg body for 12 weeks.
Conventional plus pacebo treatmentDrug
Participants received conventional treatment and taken i.v., once per 4 week, at 50 ml saline for 12 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Conventional plus MSC treatment | Experimental | Participants will receive conventional treatment plus a dose of MSC from day 0 through the week 12 study visit. Participants will then be followed until 2 years study visit Receives: Drug: Conventional plus MSC treatment |
| Conventional plus pacebo treatment | Experimental | Participants will receive conventional plus placebo treatment from day 0 through the week 12 study visit. Participants will then be followed until 2 years study visit Receives: Drug: Conventional plus pacebo treatment |
What is being measured
The primary outcome is the main question the study is designed to answer.
The levels of serum Total Protein and Albumin
Measured: 2 years after treatment
Secondary outcomes (8)
The levels of serum Total Bilirubin and Direct Bilirubin
Measured: 2 years after treatment
The levels of serum Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST) and Cholinesterase (CHE)
Measured: 2 years after treatment
The level of alpha-fetoprotein (AFP)
Measured: 2 years after treatment
The content of ascites
Measured: 2 years after treatment
Survival rate and time
Measured: 2 years after treatment
Body temperature, tetter and allergy
Measured: Between 0 to 24 hours after UC-MSCs transfusion
The levels of Prothrombin Activity (PA) and Prothrombin Time (PT)
Measured: 2 years after treatment
The score for Model for End-Stage Liver Disease
Measured: 2 years after treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Oct 2010 (estimated)
- Study startMar 2009
- Primary completionMar 2014 (estimated)
- Study completionMar 2014 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (5)Papers the sponsor has linked to this study.
- Other trials for Liver FailureStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing 302 Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Fu-Sheng Wang, Beijing 302 Hospital
- Investigators
- Fu-Sheng Wang, Professor, Principal investigator, Beijing 302 Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01218464
- Sponsor's protocol ID
- Beijing302-003
- Status last verified
- May 2013
- First submitted
- 8 Oct 2010
- Last updated
- 31 May 2013
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Kuo TK, Hung SP, Chuang CH, Chen CT, Shih YR, Fang SC, Yang VW, Lee OK. Stem cell therapy for liver disease: parameters governing the success of using bone marrow mesenchymal stem cells. Gastroenterology. 2008 Jun;134(7):2111-21, 2121.e1-3. doi: 10.1053/j.gastro.2008.03.015. Epub 2008 Mar 12.
PubMed 18455168Campard D, Lysy PA, Najimi M, Sokal EM. Native umbilical cord matrix stem cells express hepatic markers and differentiate into hepatocyte-like cells. Gastroenterology. 2008 Mar;134(3):833-48. doi: 10.1053/j.gastro.2007.12.024. Epub 2007 Dec 23.
PubMed 18243183Terai S, Ishikawa T, Omori K, Aoyama K, Marumoto Y, Urata Y, Yokoyama Y, Uchida K, Yamasaki T, Fujii Y, Okita K, Sakaida I. Improved liver function in patients with liver cirrhosis after autologous bone marrow cell infusion therapy. Stem Cells. 2006 Oct;24(10):2292-8. doi: 10.1634/stemcells.2005-0542. Epub 2006 Jun 15.
PubMed 16778155Mohamadnejad M, Alimoghaddam K, Mohyeddin-Bonab M, Bagheri M, Bashtar M, Ghanaati H, Baharvand H, Ghavamzadeh A, Malekzadeh R. Phase 1 trial of autologous bone marrow mesenchymal stem cell transplantation in patients with decompensated liver cirrhosis. Arch Iran Med. 2007 Oct;10(4):459-66.
PubMed 17903050Kharaziha P, Hellstrom PM, Noorinayer B, Farzaneh F, Aghajani K, Jafari F, Telkabadi M, Atashi A, Honardoost M, Zali MR, Soleimani M. Improvement of liver function in liver cirrhosis patients after autologous mesenchymal stem cell injection: a phase I-II clinical trial. Eur J Gastroenterol Hepatol. 2009 Oct;21(10):1199-205. doi: 10.1097/MEG.0b013e32832a1f6c.
PubMed 19455046
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase Ⅰ/Ⅱ Study of Human Umbilical Cord Derived Mesenchymal Stem Cells (UC-MSCs) for Treatment of Liver Failure
- ClinicalTrials.gov ID
- NCT01218464
- Acronym / sponsor ID
- Beijing302-003
- Conditions
- Liver Failure, Mesenchymal Stem Cells
- Keywords
- Liver Failure, Mesenchymal Stem Cells, Model for End-Stage Liver Disease, Ascite, Serum Albumin
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Beijing 302 HospitalUniversity, hospital or other
- Study start
- Mar 2009
- Primary completion
- Mar 2014 (estimated)
- Study completion
- Mar 2014 (estimated)
- First posted
- 11 Oct 2010 (estimated)
- 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.
Liver failure (LF) is a dramatic clinical syndrome with massive necrosis of liver cells. and liver transplantation is the only available therapeutic option for patients suffering with this condition. However, lack of donors, surgical complications, rejection, and high cost are serious problems. Previous study showed that bone marrow derived mesenchymal stem cells (BM-MSCs) replace hepatocytes in injured liver, and effectively rescue experimental liver failure and contribute to liver regeneration. In this study, the patients with LF will undergo administration of human umbilical cord mesenchymal stem cells (UC-MSCs) via peripheral vein transfusion to evaluate the safty and efficacy of UC-MSCs treatment for these patients.
Detailed description
Liver failure (LF) is a severe life-threatening condition, and is a dramatic clinical syndrome with massive necrosis of liver cells, and liver transplantation is the only available therapeutic option for patients suffering with this condition. However, lack of donors, surgical complications, rejection, and high cost are serious problems. Since current therapeutic options for LF that is usually with extremely poor prognosis are still limited, recent studies indicate that mesenchymal stem cells (MSCs), due to their function in immune modulation and liver-damage repair, are of great therapeutic potential for this disease. Previous study showed that bone marrow derived mesenchymal stem cells (BM-MSCs) replace hepatocytes in injured liver, and effectively rescue experimental liver failure and contribute to liver regeneration.The purpose of this study is to investigate the safety and initial efficacy of human umbilical cord MSC (UC-MSCs) treatment for patients with LF. In this study, MSCs were isolated from umbilical cord and generated in appropriate growth medium. 50 LF patients with LF received i.v. transfusion of 0.5-1.0×106 cells/kg of MSCs as the treated group and other 20 LF patients with LF were transfused with placebo without MSCs as control group. All 70 of them received the routine management for liver failure. During the 2-year follow up, the evaluation of safty and efficacy will be undergone to help to establish innovative cell-based therapies for the treatment of diseases.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, care provider)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Conventional plus MSC treatment | Experimental | Drug: Conventional plus MSC treatmentParticipants will receive conventional treatment plus a dose of MSC from day 0 through the week 12 study visit. Participants will then be followed until 2 years study visit |
| Conventional plus pacebo treatment | Experimental | Drug: Conventional plus pacebo treatmentParticipants will receive conventional plus placebo treatment from day 0 through the week 12 study visit. Participants will then be followed until 2 years study visit |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Aged 18-70 years
- Liver failure
- Negative pregnancy test (female patients in fertile age)
- Written consent
Key exclusion criteria (6 listed)
- Hepatocellular carcinoma or other malignancies
- Severe problems in other vital organs(e.g.the heart,renal or lungs)
- Pregnant or lactating women
- Severe bacteria infection
- Anticipated with difficulty of follow-up observation
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 |
|---|---|---|
| Conventional plus MSC treatmentParticipants received conventional treatment and taken i.v., once per 4 week, at a dose of 0.5*10E6 MSC/kg body for 12 weeks. | Drug | Conventional plus MSC treatment |
| Conventional plus pacebo treatmentParticipants received conventional treatment and taken i.v., once per 4 week, at 50 ml saline for 12 weeks. | Drug | Conventional plus pacebo treatment |
Primary outcome
The levels of serum Total Protein and Albumin
Time frame: 2 years after treatment
8 secondary outcomes
The levels of serum Total Bilirubin and Direct Bilirubin
Time frame: 2 years after treatment
The levels of serum Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST) and Cholinesterase (CHE)
Time frame: 2 years after treatment
The level of alpha-fetoprotein (AFP)
Time frame: 2 years after treatment
The content of ascites
Time frame: 2 years after treatment
Survival rate and time
Time frame: 2 years after treatment
Body temperature, tetter and allergy
Time frame: Between 0 to 24 hours after UC-MSCs transfusion
The levels of Prothrombin Activity (PA) and Prothrombin Time (PT)
Time frame: 2 years after treatment
The score for Model for End-Stage Liver Disease
Time frame: 2 years after treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 May 2013.
- Background: PubMed 18455168Kuo TK, Hung SP, Chuang CH, Chen CT, Shih YR, Fang SC, Yang VW, Lee OK. Stem cell therapy for liver disease: parameters governing the success of using bone marrow mesenchymal stem cells. Gastroenterology. 2008 Jun;134(7):2111-21, 2121.e1-3. doi: 10.1053/j.gastro.2008.03.015. Epub 2008 Mar 12.
- Background: PubMed 18243183Campard D, Lysy PA, Najimi M, Sokal EM. Native umbilical cord matrix stem cells express hepatic markers and differentiate into hepatocyte-like cells. Gastroenterology. 2008 Mar;134(3):833-48. doi: 10.1053/j.gastro.2007.12.024. Epub 2007 Dec 23.
- Background: PubMed 16778155Terai S, Ishikawa T, Omori K, Aoyama K, Marumoto Y, Urata Y, Yokoyama Y, Uchida K, Yamasaki T, Fujii Y, Okita K, Sakaida I. Improved liver function in patients with liver cirrhosis after autologous bone marrow cell infusion therapy. Stem Cells. 2006 Oct;24(10):2292-8. doi: 10.1634/stemcells.2005-0542. Epub 2006 Jun 15.
- Results publication: PubMed 17903050Mohamadnejad M, Alimoghaddam K, Mohyeddin-Bonab M, Bagheri M, Bashtar M, Ghanaati H, Baharvand H, Ghavamzadeh A, Malekzadeh R. Phase 1 trial of autologous bone marrow mesenchymal stem cell transplantation in patients with decompensated liver cirrhosis. Arch Iran Med. 2007 Oct;10(4):459-66.
- Results publication: PubMed 19455046Kharaziha P, Hellstrom PM, Noorinayer B, Farzaneh F, Aghajani K, Jafari F, Telkabadi M, Atashi A, Honardoost M, Zali MR, Soleimani M. Improvement of liver function in liver cirrhosis patients after autologous mesenchymal stem cell injection: a phase I-II clinical trial. Eur J Gastroenterol Hepatol. 2009 Oct;21(10):1199-205. doi: 10.1097/MEG.0b013e32832a1f6c.
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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.
Fu-Sheng Wang, Professor principal investigator
Beijing 302 Hospital
1 site 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 Conventional plus MSC treatment and Conventional plus pacebo treatment in people with Liver Failure and Mesenchymal Stem Cells, run by Beijing 302 Hospital at 1 site in China.
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 6 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: status not confirmed, as of the sponsor's last update on 31 May 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fu-Sheng Wang, Professor (86-10-63879735 ext. 2015.12, fswang@public.bta.net.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The levels of serum Total Protein and Albumin (time frame: 2 years after treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01218464, which also lists 5 linked publications.
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 31 May 2013.