Alcoholic Liver CirrhosisPhase 1NCT03838250
Study to Evaluate Hepatic Artery Injection of Autologous Human Bone Marrow-Derived MSCs in Patients With Alcoholic LC
A Phase 1 study testing Cellgram™ (Bone marrow-derived MSCs) in people with Alcoholic Liver Cirrhosis, run by Pharmicell Co., Ltd. at 1 site in United States.
Trial summary
This study is to evaluate the safety and efficacy of a single dose of Cellgram™ delivered via hepatic artery in patients with decompensated alcoholic liver cirrhosis. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Alcoholic Liver Cirrhosis
- What is being tested?
- Cellgram™ (Bone marrow-derived MSCs)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Jun 2019to Jun 2021
- Phase
- Phase 1
- 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
- Alcoholic liver cirrhosis as diagnosed by clinical, biochemical, radiological, or histological evidence.
- Male or female, 18 to 70 years of age, inclusive.
- Child-Pugh class B (7 to 9 points)
- Capable, in the Investigators opinion, of undergoing hepatic artery catheterization.
- No consumption of alcohol or other potentially hepatotoxic substances considered clinically relevant in the opinion of the Investigator, within 6 months prior to screening and throughout the study.
- Provision of informed consent by the patient (or their legal representative) to participate in the clinical study.
- Able, in the Investigator's opinion, to comply with the requirements of the protocol (including the follow-up period).
- Females of childbearing potential must test negative on standard urine pregnancy test and must be willing to practice appropriate contraceptive methods for the duration of the study. Highly effective methods of birth control include hormonal birth control, intrauterine devices (IUDs), or any double-barrier method (sponges, female condoms) used by the woman in addition to contraception used by their male partner such as vasectomy or condom supplemented with spermicide.
Who cannot join
- Current diagnosis of malignant hematologic disease (e.g., acute myeloid leukemia, acute lymphoblastic leukemia, non-Hodgkin's lymphoma, Hodgkin's lymphoma, multiple myeloma).
- Etiology other than alcohol for underlying liver cirrhosis.
- Baseline creatinine >1.7 mg/dL and/or estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m2
- Clinical history of a solid cancer within 5 years prior to screening or current diagnosis of a solid cancer (including hepatocellular carcinoma assessed by ultrasonography and elevated AFP level) and currently receiving cancer treatment.
- Continuous use of a clinically relevant amount of steroids or antibiotics within 1 month prior to screening. Clinical relevance will be determined by the Investigator.
- Model for End-Stage Liver Disease score >20.
Show all 19Show fewer
- International normalized ratio >3.0 and/or platelet counts <30,000/mm3
- Major operation within 3 months prior to screening.
- Presence of extrahepatic biliary stricture.
- Participant has undergone transjugular intrahepatic portosystemic shunt.
- Active hepatic artery or portal vein thrombosis.
- Presence of advanced hepatic encephalopathy Stages 3-4 (West Haven criteria) at the time of screening.
- Active variceal bleeding during the last 6 months before screening.
- Severe cardiac, renal, or respiratory failure.
- Positive serological test results for human immunodeficiency virus (HIV), HCV, hepatitis B surface antigen (HBsAg) and/or syphilis.
- Patient is pregnant or breast feeding, or planning to become pregnant while enrolled in the study, up to the EOS visit.
- Positive urine pregnancy test at Screening.
- Drug abuse within the past 2 years (as confirmed by patient disclosure or a urine drug screen conducted at Screening).
- Participation in an interventional clinical study within 30 days prior to screening.
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
University of Utah
Salt Lake City, Utah, 84132
Juan Gallegos-Orozco, Ph.D, 801-581-7878, juan.gallegos@hsc.utah.edu
About this study
Official title: A Phase I, Open-Label, Single-Dose Study to Evaluate the Safety and Efficacy of Hepatic Artery Injection of Autologous Human Bone Marrow-Derived Mesenchymal Stem Cells (Cellgram™) in Patients With Alcoholic Liver Cirrhosis
This study is to evaluate the safety and efficacy of a single dose of Cellgram™ delivered via hepatic artery in patients with decompensated alcoholic liver cirrhosis.
Read the detailed description
After providing written informed consent, subjects will return to the study center for further evaluation and to have their Bone marrow harvested by an experienced hematologist or interventional radiologist.
Within approximately 1 month (30 ± 7 days) after Bone marrow aspiration, study participants will be admitted to the study center on Day 1.
At the study center, the participant will undergo hepatic artery catheterization by an interventional radiologist who will inject a single dose of Cellgram™. Participants will remain as in-patients and be observed for 24 hours post-infusion. Following discharge, participants will periodically return to the study center for study assessment visits over a period of 1 year.
When a suitable candidate is identified by the Investigator, the Investigator or designated healthcare professional will ask the patient about his/her willingness to be included in the clinical study. Following this, patients will be allowed sufficient time, in their own opinion, to consider study entry, and will be offered the opportunity to ask any further questions prior to signing the informed consent form.
- Conditions
- Alcoholic Liver Cirrhosis
- Being tested
- Cellgram™ (Bone marrow-derived MSCs)
- Keywords
- Alcoholic, Liver, Cirrhosis, Liver Disease
- Medical subject headings
- Liver Cirrhosis, Alcoholic, Fibrosis, Liver Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cellgram™ (Bone marrow-derived MSCs)Biological
Approximately 15 to 30 mL of bone marrow is aspirated from the posterior iliac crest of patients under local anesthesia. Approximately 30 days (±7 days) after BM aspiration, the participant will return to the study center for admission and for the infusion Cellgram™ (Bone marrow-derived MSCs).
| Study group | Role | What they receive |
|---|---|---|
| Cellgram™ (Bone marrow-derived MSCs) | Experimental | Infusion Cellgram™(Bone marrow-derived MSCs). Single dose administration of approximately 5 x 10^7 cells/10 mL (range: 4.5 x 10^7 to 5.5 x 10^7 cells/10 mL) via the hepatic artery. Receives: Biological: Cellgram™ (Bone marrow-derived MSCs) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Serious Adverse Events
An SAE suggests a significant hazard, contraindication, side-effect, or precaution. With respect to human clinical experience, this includes any event that:
- Results in death.
- Is life-threatening.*
- Requires in-patient hospitalization or prolongation of existing hospitalization.
- Results in persistent or significant disability/incapacity.
- Is a congenital anomaly/birth defect.
- Other medically important condition
- Life-threatening in the definition of a SAE or adverse reaction refers to an event in which the patient was at risk of death at the time of event; it does not refer to an event, which hypothetically might have caused death if it were more severe.
Measured: 12 months
Secondary outcomes (8)
Number of patients with Hepatocellular carcinoma (primary liver cancer) development
assessed via ultrasonography and alpha fetoprotein [AFP] analysis
Measured: 12 months
Incidence of Adverse Events
Incidence of Adverse Events as assessed by vital signs, physical examination, safety laboratory tests, and patient reporting
Measured: 12 months
Liver stiffness measurement
with transient elastography (i.e., FibroScan®)
Measured: 12 months
How well the Liver is functioning
by tests including: serum levels of aspartate aminotransferase (AST), alanine aminotransferase (ALT), bilirubin, albumin, gamma glutamyl transpeptidase (GGT) and creatinine.
Units of Measure: AST will be report in U/L. ALT will be report in U/L. GGT will be report in U/L. Bilirubin will be report in mg/dL. creatinine will be report in mg/dL. Albumin will be report in g/dL
Measured: 12 months
Chronic liver disease as assessed by the Child-Pugh score
The Child-Pugh Score will be used to determine the prognosis, required strength of treatment and the necessity of liver transplantation. The score employs 5 clinical measures of liver disease (total bilirubin, serum albumin, INR, ascites and hepatic encephalopathy). Each parameter is scored 1 to 3, with 3 indicating the most severe derangement.
Measured: 12 months
Model for End-Stage Liver Disease (MELD) Score
MELD uses the patient's values for serum bilirubin, serum creatinine, and the INR for PT to predict survival. It is calculated according to the following formula defined by Kamath et al (2001):
MELD = 3.78×ln[serum bilirubin (mg/dL)] + 11.2×ln[INR] + 9.57×ln[serum creatinine (mg/dL)] + 6.43
Measured: 12 months
Overall survival
defined as the time from infusion until death from any cause during the study period.
Measured: 12 months
Quality of life as assessed by 36-Item Short Form Survey (SF-36) Questionnaire
The Short Form-36 Quality Of Life questionnaire will be recorded for each patient. The 8 subscales of the SF-36 (Physical Functioning, Role Physical, Bodily Pain, General Health, Vitality, Social Functioning, Role Emotional, Mental Health) and 2 summary component measures (Physical Component Summary, Mental Component Summary) will be calculated and summarized.
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Feb 2019
- Study start20 Jun 2019
- Primary completionMar 2021 (estimated)
- Study completionJun 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Alcoholic 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A single-dose injection of autologous bone marrow-derived mesenchymal stem cells (Cellgram™) will be administered to the patient by an experienced interventional radiologist.
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Pharmicell Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Juan Gallegos-Orozco, Ph.D, Principal investigator, University of Utah
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03838250
- Sponsor's protocol ID
- PMC-P-08
- Status last verified
- Oct 2020
- First submitted
- 7 Jan 2019
- Last updated
- 22 Oct 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 22 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Open-Label, Single-Dose Study to Evaluate the Safety and Efficacy of Hepatic Artery Injection of Autologous Human Bone Marrow-Derived Mesenchymal Stem Cells (Cellgram™) in Patients With Alcoholic Liver Cirrhosis
- ClinicalTrials.gov ID
- NCT03838250
- Acronym / sponsor ID
- PMC-P-08
- Conditions
- Alcoholic Liver Cirrhosis
- Keywords
- Alcoholic, Liver, Cirrhosis, Liver Disease
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Pharmicell Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 20 Jun 2019 (actual)
- Primary completion
- Mar 2021 (estimated)
- Study completion
- Jun 2021 (estimated)
- First posted
- 12 Feb 2019 (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 study is to evaluate the safety and efficacy of a single dose of Cellgram™ delivered via hepatic artery in patients with decompensated alcoholic liver cirrhosis.
Detailed description
After providing written informed consent, subjects will return to the study center for further evaluation and to have their Bone marrow harvested by an experienced hematologist or interventional radiologist.
Within approximately 1 month (30 ± 7 days) after Bone marrow aspiration, study participants will be admitted to the study center on Day 1.
At the study center, the participant will undergo hepatic artery catheterization by an interventional radiologist who will inject a single dose of Cellgram™. Participants will remain as in-patients and be observed for 24 hours post-infusion. Following discharge, participants will periodically return to the study center for study assessment visits over a period of 1 year.
When a suitable candidate is identified by the Investigator, the Investigator or designated healthcare professional will ask the patient about his/her willingness to be included in the clinical study. Following this, patients will be allowed sufficient time, in their own opinion, to consider study entry, and will be offered the opportunity to ask any further questions prior to signing the informed consent form.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A single-dose injection of autologous bone marrow-derived mesenchymal stem cells (Cellgram™) will be administered to the patient by an experienced interventional radiologist.
| Arm | Type | Receives |
|---|---|---|
| Cellgram™ (Bone marrow-derived MSCs) | Experimental | Biological: Cellgram™ (Bone marrow-derived MSCs)Infusion Cellgram™(Bone marrow-derived MSCs). Single dose administration of approximately 5 x 10^7 cells/10 mL (range: 4.5 x 10^7 to 5.5 x 10^7 cells/10 mL) via the hepatic artery. |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Alcoholic liver cirrhosis as diagnosed by clinical, biochemical, radiological, or histological evidence.
- Male or female, 18 to 70 years of age, inclusive.
- Child-Pugh class B (7 to 9 points)
- Capable, in the Investigators opinion, of undergoing hepatic artery catheterization.
- No consumption of alcohol or other potentially hepatotoxic substances considered clinically relevant in the opinion of the Investigator, within 6 months prior to screening and throughout the study.
Key exclusion criteria (19 listed)
- Current diagnosis of malignant hematologic disease (e.g., acute myeloid leukemia, acute lymphoblastic leukemia, non-Hodgkin's lymphoma, Hodgkin's lymphoma, multiple myeloma).
- Etiology other than alcohol for underlying liver cirrhosis.
- Baseline creatinine >1.7 mg/dL and/or estimated glomerular filtration rate (eGFR) <30 mL/min/1.73 m2
- Clinical history of a solid cancer within 5 years prior to screening or current diagnosis of a solid cancer (including hepatocellular carcinoma assessed by ultrasonography and elevated AFP level) and currently receiving cancer treatment.
- Continuous use of a clinically relevant amount of steroids or antibiotics within 1 month prior to screening. Clinical relevance will be determined by the Investigator.
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 |
|---|---|---|
| Cellgram™ (Bone marrow-derived MSCs)Approximately 15 to 30 mL of bone marrow is aspirated from the posterior iliac crest of patients under local anesthesia. Approximately 30 days (±7 days) after BM aspiration, the participant will return to the study center for admission and for the infusion Cellgram™ (Bone marrow-derived MSCs). | Biological | Cellgram™ (Bone marrow-derived MSCs) |
Primary outcome
Incidence of Serious Adverse Events
Time frame: 12 months
An SAE suggests a significant hazard, contraindication, side-effect, or precaution. With respect to human clinical experience, this includes any event that: * Results in death. * Is life-threatening.* * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity. * Is a congenital anomaly/birth defect. * Other medically important condition * Life-threatening in the definition of a SAE or adverse reaction refers to an event in which the patient was at risk of death at the time of event; it does not refer to an event, which hypothetically might have caused death if it were more severe.
8 secondary outcomes
Number of patients with Hepatocellular carcinoma (primary liver cancer) development
Time frame: 12 months
Incidence of Adverse Events
Time frame: 12 months
Liver stiffness measurement
Time frame: 12 months
How well the Liver is functioning
Time frame: 12 months
Chronic liver disease as assessed by the Child-Pugh score
Time frame: 12 months
Model for End-Stage Liver Disease (MELD) Score
Time frame: 12 months
Overall survival
Time frame: 12 months
Quality of life as assessed by 36-Item Short Form Survey (SF-36) Questionnaire
Time frame: 12 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Oct 2020.
- Individual participant dataSharing planned: Undecided
Related trials for Alcoholic Liver Cirrhosis
RecruitingPhase 3
Trial to Evaluate ALN-CIDEB in Adults With Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD)
Metabolic Dysfunction-Associated Steatotic Liver Disease, MASLD
RecruitingPhase 2
A Study of Efimosfermin Alfa in Participants With Biopsy-confirmed Cirrhosis (Compensated) Due to MASH
Metabolic Dysfunction-Associated Steatohepatitis, Non-alcoholic Fatty Liver Disease
RecruitingPhase 3
LIVERAGE™: A Study to Test Whether Survodutide Helps People With a Liver Disease Called NASH/MASH Who Have Moderate or Advanced Liver Fibrosis
Metabolic Dysfunction Associated Steatohepatitis (MASH), Liver Fibrosis
RecruitingPhase 3
LIVERAGE™ - Cirrhosis: A Study to Test Whether Survodutide Helps People With a Liver Disease Called NASH/MASH Who Have Cirrhosis
Metabolic Dysfunction Associated Steatohepatitis
Not yet recruitingObservational study
Inflammatory and Neuroimmune Guided Investigation of Underlying Fatigue in Patients With Cirrhosis
Cirrhosis Alcoholic, Fatigue Symptom
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.
Juan Gallegos-Orozco, Ph.D principal investigator
University of Utah
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cellgram™ (Bone marrow-derived MSCs) in people with Alcoholic Liver Cirrhosis, run by Pharmicell Co., Ltd. at 1 site in United States.
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 8 inclusion and 19 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 22 Oct 2020. 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 United States. 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 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.
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 JIYEOUN JEONG, CCRP, Bachelor (82-02-3496-0134, jyjeong@pharmicell.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Serious Adverse Events (time frame: 12 months). 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 22 Oct 2020.