Alpha-1 Antitrypsin DeficiencyNCT02014415
Alpha-1 Antitrypsin Deficiency Adult Liver Study
An observational study of Alpha-1 Antitrypsin Deficiency, run by St. Louis University at 3 sites in United States.
Trial summary
The investigators hypothesize that there is liver injury (inflammation, fibrosis, cirrhosis) in adults with Alpha-1 Antitrypsin Deficiency (AATD), which is asymptomatic, under-recognized, and undiagnosed. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Alpha-1 Antitrypsin Deficiency
- What is being tested?
- Liver Biopsy (Biopsy Group Only)
- Type of treatment
- Surgery or procedure
- How many people
- 120 participants
- Where
- 3 sitesUnited States
- Study dates
- Dec 2013to Dec 2027
- How long will I take part?
- 7 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: Pi-ZZ AAT deficient adults with either no previous history of liver disease, moderate-severe liver disease or post liver transplant. Biologic family members who are also Pi-ZZ and ≥ 18 years of age, will be offered enrollment into the study. The purpose of including family members is to determine if the natural history of the liver disease is consistent in a given family.
Who may be able to join
- Adults (≥ 18 years of age), with Alpha-1 Antitrypsin Deficiency
- Documented evidence Pi-ZZ phenotype or genotype
- Both genders, all races and ethnic groups
- Willingness to be followed for up to 5 years
- Adults (≥ 18 years of age), with alpha-1-antitrypsin deficiency
- Documented evidence PI-ZZ phenotype or genotype
Show all 13Show fewer
- Documented evidence of portal hypertension or evidence of advanced liver disease defined by Child-Pugh Class B or C (score ≥ 7), or previous liver biopsy with an Ishak Fibrosis Score ≥ 4
- Both genders, all races and ethnic groups
- Willingness to be followed for up to 5 years
- Adults (≥ 18 years of age), with alpha-1-antitrypsin deficiency
- Pre-transplant documented evidence of PI-ZZ phenotype or genotype
- Documented evidence of liver transplantation
- Both genders, all races and ethnic groups
Who cannot join
- Evidence of advanced liver disease defined by Child-Pugh Class B or C (score ≥ 7)
- Known advanced lung disease defined as forced expiratory volume at one second (FEV1) < 40 % of Predicted
- History of Organ Transplantation
- Known congenital or metabolic liver disease (e.g.: Wilson's, glycogen storage, cystic fibrosis)or iron overload as evidenced by ≥ Grade 3 iron staining on a previous liver biopsy
- Evidence of chronic hepatitis B (marked by the presence of HBsAg in serum) or Hepatitis C (marked by the presence of anti-hepatitis C virus (HCV) or HCV RNA in serum)
- Vascular disorders of the liver (e.g.: cardiac sclerosis, acute or chronic Budd-Chiari, hepatoportal sclerosis, peliosis)
Show all 27Show fewer
- Known HIV positivity
- Diagnosis of malignancy within the last 5 years
- Active substance abuse, that in the opinion of the study investigator, would interfere with adherence to study requirements
- Concomitant severe underlying systemic illness or medical condition which in the opinion of the investigator, would make the patient unsuitable for the study or would interfere with completion of follow-up
- Inability to comply with the longitudinal follow-up as outlined in the protocol
- Failure of the participant to sign informed consent or Health Insurance Portability and Accountability Act (HIPAA) documents
- Known Severe Liver Disease Group:
- History of Organ Transplantation
- Known congenital or metabolic liver disease (e.g.: Wilson's, glycogen storage, cystic fibrosis) and iron overload as evidenced by ≥ Grade 3 iron staining on a previous liver biopsy
- Evidence of chronic hepatitis B (marked by the presence of HBsAg in serum) or Hepatitis C (marked by the presence of anti-HCV or HCV RNA in serum)
- Vascular disorders of the liver (e.g.: cardiac sclerosis, acute or chronic Budd-Chiari, hepatoportal sclerosis, peliosis)
- Known HIV positivity
- Diagnosis of malignancy within the last 5 years which in the opinion of the investigator, would make the patient's follow-up problematic or the results uninterpretable.
- Active substance abuse, that in the opinion of the study investigator, would interfere with adherence to study requirements
- Concomitant severe underlying systemic illness or medical condition which in the opinion of the investigator, would make the patient unsuitable for the study or would interfere with completion of follow-up
- Inability to comply with the longitudinal follow-up as outlined in the protocol
- Failure of the participant to sign informed consent or HIPAA documents.
- Post Liver Transplant Group
- Active substance abuse, that in the opinion of the study investigator, would interfere with adherence to study requirements
- Concomitant severe underlying systemic illness or medical condition which in the opinion of the investigator, would make the patient unsuitable for the study or would interfere with completion of study requirements
- Failure of the participant to sign informed consent or HIPAA documents.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Boston University School of Medicine
Boston, Massachusetts, 02118
University of California
San Diego, California, 92103
Saint Louis University
St Louis, Missouri, 63104
About this study
Official title: Alpha-1 Antitrypsin Deficiency Adult Clinical and Genetic Linkage Study
The investigators hypothesize that there is liver injury (inflammation, fibrosis, cirrhosis) in adults with Alpha-1 Antitrypsin Deficiency (AATD), which is asymptomatic, under-recognized, and undiagnosed. In addition, the investigators believe that the genetic and environmental factors that play an important role in the development of alpha-1 antitrypsin (AAT) liver disease, can be identified by comparing a cohort database of clinical disease information to linked biospecimen and DNA samples.
Read the detailed description
Alpha-1 Antitrypsin Deficiency (AATD) is a genetic disorder resulting in a low level of a protein called alpha-1 antitrypsin (AAT). This deficiency can cause life-threatening liver disease and/or lung disease at various ages. Some patients experience life-threatening liver disease in childhood or liver cancer as adults. There is no specific treatment for AAT related liver disease. Some patients develop emphysema as young adults, while some patients remain healthy throughout their lives. Differences in the environment or in other genes may explain such inconsistency in the disease.
The primary objective of this multi-center study is to assess the natural history of individuals with Pi-ZZ AAT deficiency, identify biomarkers for the progression of liver disease and construct a database capable of linking cohort data with repository biospecimens. The secondary objective is to analyze components of the demographic, social, and family history associated with more severe liver disease.
This study will examine the natural history of liver disease by recording participant's family history, medical history, current health, laboratory test results, and medical treatment(s). Participants may complete brief research questionnaires about their physical and mental health, diet, alcohol intake, and smoke, environmental and occupational (work) exposures.
At least 120 Pi-ZZ AAT deficient adults with no previous history of liver disease, moderate-severe liver disease, or post liver transplant, will be enrolled at one of three sites. Eligible subjects will participate in one of the following study arms:
- Liver Biopsy
- Known Severe Liver Disease - subjects not meeting Biopsy Group eligibility due to the presence of advanced liver disease
- Post Liver Transplant - subjects who have previously undergone a liver transplant
At the time of enrollment, each participant will be assigned a unique study identification (ID) number. All participant information recorded and samples collected for the study will be saved by this unique number. All blood, tissue and genetic samples collected will be sent to a secured repository for future retrieval and study. The process of coding data and samples lessens the chances of a breach in confidentiality.
The length of study participation, tests and activities performed specifically for research will be determined by the enrollment group. Subjects in the Biopsy and Known Severe Liver Disease groups participate in the study for 5 years (enrollment and four annual follow-up visits). Both groups undergo a physical exam, diagnostic abdominal ultrasound, pulmonary function testing and the collection of serum, plasma and blood for routine laboratory and genetic testing (Induced Pluripotent Stem Cells (iPS cells), microRNA and DNA). However, only the Biopsy Group participants undergo a liver biopsy and FibroScan at enrollment, and again in Year 5. The liver tissue samples will help the researchers learn what causes liver disease in some patients and how the liver disease progresses.
Subjects in the Post Liver Transplant group have a single study visit to record their history, complete questionnaires and perform pulmonary function testing. In addition, whole blood for DNA analysis will be collected from these participants.
Based on their study arm assignment, participants will receive copies of their diagnostic abdominal ultrasound, pulmonary function test, routine laboratory test and liver biopsy pathology results, to share with their primary care physician.
Upon completion of the 5-year study, two cross-sectional visits were added to the Biopsy Group and Known Severe Liver Disease Group. The cross-sectional visit will be completed by phone and review of medical records.
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Being tested
- Liver Biopsy (Biopsy Group Only)
- Keywords
- Alpha-1, AAT Deficiency, AATD, Liver, Fibrosis, Liver Transplant, Cirrhosis
- Medical subject headings
- alpha 1-Antitrypsin Deficiency, Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Liver Biopsy (Biopsy Group Only)Procedure or surgery
A percutaneous (needle) liver biopsy will be performed on subjects in the Liver Biopsy Group, in Year 1 and Year 5 of the study.
| Study group | Role | What they receive |
|---|---|---|
| Liver Biopsy | Participants will provide liver tissue specimens collected at the time of liver biopsy, to determine the rate of progression of liver injury in adults with Pi-ZZ Alpha-1 Antitrypsin Deficiency. Receives: Procedure: Liver Biopsy (Biopsy Group Only) | |
| Known Severe Liver Disease | Participants will provide samples of serum, plasma, and DNA at defined time points to determine what genetic and environmental modifiers and biomarkers are associated with severe clinical liver disease, such cirrhosis, portal hypertension and liver failure in adults with Pi-ZZ Alpha-1 Antitrypsin Deficiency. | |
| Post Liver Transplant | Participants will provide a DNA sample to determine what genetic and environmental modifiers are associated with the need for liver transplantation in adults with Pi-ZZ Alpha-1 Antitrypsin Deficiency. |
What is being measured
The primary outcome is the main question the study is designed to answer.
The risk and rate of histologic liver injury progression, as measured by liver biopsy, over a 5-year period.
Measured: Liver biopsy performed in Year 1 and Year 5
Secondary outcomes (10)
Calculated Model for End-stage Liver Disease score (MELD)
Measured: Calculated at baseline and annually through year 5
Liver synthetic dysfunction defined by international normalized ratio (INR) > 1.3 or serum albumin < 3.2 gm/dL
Measured: Measured at baseline and annually through year 5
Presence of ascites (or treatment for ascites)
Measured: Assessed at baseline and annually through year 5
Development of complications of portal hypertension (e.g., variceal hemorrhage)
Measured: Assessed at baseline and annually through year 5
Jaundice (total serum bilirubin >2.0 mg/dl)
Measured: Measured at baseline and annually through year 5
Liver transplantation
Measured: Assessed annually through year 5
Listing for liver transplantation
Measured: Assessed at baseline and annually through year 5
Health related quality of life
Measured: Measured at baseline and annually through year 5
FEV1 % of Predicted
Measured: Collected at baseline and annually through year 5
Death
Measured: Collected annually through year 5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Dec 2013 (estimated)
- Study startDec 2013
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Alpha-1 Antitrypsin DeficiencyStudies 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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 120 participants (estimated)
- Target duration
- 7 Years
Sponsor and investigators
- Lead sponsor
- St. Louis University
- Sponsor type
- University, hospital or other
- Collaborators
- Alpha-1 Foundation, University of California, Boston University, University College, London, University of Massachusetts, Worcester
- Responsible party
- Principal investigator, Jeffrey Teckman M.D., St. Louis University
- Investigators
- Jeffrey Teckman, MD, Study chair, St. Louis UniversityAndrew A Wilson, MD, Study director, Alpha-1 FoundationDavid A. Brenner, MD, Principal investigator, The University of California, San DiegoAndrew A Wilson, MD, Principal investigator, Boston University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02014415
- Sponsor's protocol ID
- A1F-SLU-7113
- Other IDs
- Alpha-1 Foundation, 22609
- Status last verified
- Apr 2026
- First submitted
- 12 Dec 2013
- Last updated
- 27 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes: Based on study group assignment, participants will be given a copy of their test results as follows: Liver Biopsy Group- liver biopsy pathology, diagnostic abdominal ultrasound, pulmonary function testing, and routine clinical laboratory testing. Known Severe Liver Disease Group- diagnostic abdominal ultrasound, pulmonary function testing, and routine clinical laboratory testing. Post Liver Transplant Group- pulmonary function testing.
Publications
Suri A, Zhang Z, Neuschwander-Tetri B, Lomas DA, Heyer-Chauhan N, Burling K, Loomba R, Brenner DA, Nagy R, Wilson A, Carpenter D, Blomenkamp K, Teckman J. Fibrosis, biomarkers and liver biopsy in AAT deficiency and relation to liver Z protein polymer accumulation. Liver Int. 2024 Dec;44(12):3204-3213. doi: 10.1111/liv.16094. Epub 2024 Sep 12.
PubMed 39263815
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Alpha-1 Antitrypsin Deficiency Adult Clinical and Genetic Linkage Study
- ClinicalTrials.gov ID
- NCT02014415
- Acronym / sponsor ID
- A1F-SLU-7113
- Conditions
- Alpha-1 Antitrypsin Deficiency
- Keywords
- Alpha-1, AAT Deficiency, AATD, Liver, Fibrosis, Liver Transplant, Cirrhosis
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 120 participants (estimated)
- Lead sponsor
- St. Louis UniversityUniversity, hospital or other
- Collaborators
- Alpha-1 Foundation, University of California, Boston University, University College, London, University of Massachusetts, Worcester
- Study start
- Dec 2013
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Dec 2027 (estimated)
- First posted
- 18 Dec 2013 (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.
The investigators hypothesize that there is liver injury (inflammation, fibrosis, cirrhosis) in adults with Alpha-1 Antitrypsin Deficiency (AATD), which is asymptomatic, under-recognized, and undiagnosed. In addition, the investigators believe that the genetic and environmental factors that play an important role in the development of alpha-1 antitrypsin (AAT) liver disease, can be identified by comparing a cohort database of clinical disease information to linked biospecimen and DNA samples.
Detailed description
Alpha-1 Antitrypsin Deficiency (AATD) is a genetic disorder resulting in a low level of a protein called alpha-1 antitrypsin (AAT). This deficiency can cause life-threatening liver disease and/or lung disease at various ages. Some patients experience life-threatening liver disease in childhood or liver cancer as adults. There is no specific treatment for AAT related liver disease. Some patients develop emphysema as young adults, while some patients remain healthy throughout their lives. Differences in the environment or in other genes may explain such inconsistency in the disease.
The primary objective of this multi-center study is to assess the natural history of individuals with Pi-ZZ AAT deficiency, identify biomarkers for the progression of liver disease and construct a database capable of linking cohort data with repository biospecimens. The secondary objective is to analyze components of the demographic, social, and family history associated with more severe liver disease.
This study will examine the natural history of liver disease by recording participant's family history, medical history, current health, laboratory test results, and medical treatment(s). Participants may complete brief research questionnaires about their physical and mental health, diet, alcohol intake, and smoke, environmental and occupational (work) exposures.
At least 120 Pi-ZZ AAT deficient adults with no previous history of liver disease, moderate-severe liver disease, or post liver transplant, will be enrolled at one of three sites. Eligible subjects will participate in one of the following study arms:
- Liver Biopsy
- Known Severe Liver Disease - subjects not meeting Biopsy Group eligibility due to the presence of advanced liver disease
- Post Liver Transplant - subjects who have previously undergone a liver transplant
At the time of enrollment, each participant will be assigned a unique study identification (ID) number. All participant information recorded and samples collected for the study will be saved by this unique number. All blood, tissue and genetic samples collected will be sent to a secured repository for future retrieval and study. The process of coding data and samples lessens the chances of a breach in confidentiality.
The length of study participation, tests and activities performed specifically for research will be determined by the enrollment group. Subjects in the Biopsy and Known Severe Liver Disease groups participate in the study for 5 years (enrollment and four annual follow-up visits). Both groups undergo a physical exam, diagnostic abdominal ultrasound, pulmonary function testing and the collection of serum, plasma and blood for routine laboratory and genetic testing (Induced Pluripotent Stem Cells (iPS cells), microRNA and DNA). However, only the Biopsy Group participants undergo a liver biopsy and FibroScan at enrollment, and again in Year 5. The liver tissue samples will help the researchers learn what causes liver disease in some patients and how the liver disease progresses.
Subjects in the Post Liver Transplant group have a single study visit to record their history, complete questionnaires and perform pulmonary function testing. In addition, whole blood for DNA analysis will be collected from these participants.
Based on their study arm assignment, participants will receive copies of their diagnostic abdominal ultrasound, pulmonary function test, routine laboratory test and liver biopsy pathology results, to share with their primary care physician.
Upon completion of the 5-year study, two cross-sectional visits were added to the Biopsy Group and Known Severe Liver Disease Group. The cross-sectional visit will be completed by phone and review of medical records.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 7 Years
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Liver Biopsy | – | Procedure: Liver Biopsy (Biopsy Group Only)Participants will provide liver tissue specimens collected at the time of liver biopsy, to determine the rate of progression of liver injury in adults with Pi-ZZ Alpha-1 Antitrypsin Deficiency. |
| Known Severe Liver Disease | – | –Participants will provide samples of serum, plasma, and DNA at defined time points to determine what genetic and environmental modifiers and biomarkers are associated with severe clinical liver disease, such cirrhosis, portal hypertension and liver failure in adults with Pi-ZZ Alpha-1 Antitrypsin Deficiency. |
| Post Liver Transplant | – | –Participants will provide a DNA sample to determine what genetic and environmental modifiers are associated with the need for liver transplantation in adults with Pi-ZZ Alpha-1 Antitrypsin Deficiency. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Pi-ZZ AAT deficient adults with either no previous history of liver disease, moderate-severe liver disease or post liver transplant. Biologic family members who are also Pi-ZZ and ≥ 18 years of age, will be offered enrollment into the study. The purpose of including family members is to determine if the natural history of the liver disease is consistent in a given family.
Key inclusion criteria (13 listed)
- Adults (≥ 18 years of age), with Alpha-1 Antitrypsin Deficiency
- Documented evidence Pi-ZZ phenotype or genotype
- Both genders, all races and ethnic groups
- Willingness to be followed for up to 5 years
- Adults (≥ 18 years of age), with alpha-1-antitrypsin deficiency
Key exclusion criteria (27 listed)
- Evidence of advanced liver disease defined by Child-Pugh Class B or C (score ≥ 7)
- Known advanced lung disease defined as forced expiratory volume at one second (FEV1) < 40 % of Predicted
- History of Organ Transplantation
- Known congenital or metabolic liver disease (e.g.: Wilson's, glycogen storage, cystic fibrosis)or iron overload as evidenced by ≥ Grade 3 iron staining on a previous liver biopsy
- Evidence of chronic hepatitis B (marked by the presence of HBsAg in serum) or Hepatitis C (marked by the presence of anti-hepatitis C virus (HCV) or HCV RNA in serum)
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 |
|---|---|---|
| Liver Biopsy (Biopsy Group Only)A percutaneous (needle) liver biopsy will be performed on subjects in the Liver Biopsy Group, in Year 1 and Year 5 of the study. | Procedure or surgery | Liver Biopsy |
Primary outcome
The risk and rate of histologic liver injury progression, as measured by liver biopsy, over a 5-year period.
Time frame: Liver biopsy performed in Year 1 and Year 5
10 secondary outcomes
Calculated Model for End-stage Liver Disease score (MELD)
Time frame: Calculated at baseline and annually through year 5
Liver synthetic dysfunction defined by international normalized ratio (INR) > 1.3 or serum albumin < 3.2 gm/dL
Time frame: Measured at baseline and annually through year 5
Presence of ascites (or treatment for ascites)
Time frame: Assessed at baseline and annually through year 5
Development of complications of portal hypertension (e.g., variceal hemorrhage)
Time frame: Assessed at baseline and annually through year 5
Jaundice (total serum bilirubin >2.0 mg/dl)
Time frame: Measured at baseline and annually through year 5
Liver transplantation
Time frame: Assessed annually through year 5
Listing for liver transplantation
Time frame: Assessed at baseline and annually through year 5
Health related quality of life
Time frame: Measured at baseline and annually through year 5
FEV1 % of Predicted
Time frame: Collected at baseline and annually through year 5
Death
Time frame: Collected annually through year 5
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2026.
- Derived from this study: PubMed 39263815Suri A, Zhang Z, Neuschwander-Tetri B, Lomas DA, Heyer-Chauhan N, Burling K, Loomba R, Brenner DA, Nagy R, Wilson A, Carpenter D, Blomenkamp K, Teckman J. Fibrosis, biomarkers and liver biopsy in AAT deficiency and relation to liver Z protein polymer accumulation. Liver Int. 2024 Dec;44(12):3204-3213. doi: 10.1111/liv.16094. Epub 2024 Sep 12.
- Individual participant dataSharing planned: Yes. Based on study group assignment, participants will be given a copy of their test results as follows: Liver Biopsy Group- liver biopsy pathology, diagnostic abdominal ultrasound, pulmonary function testing, and routine clinical laboratory testing. Known Severe Liver Disease Group- diagnostic abdominal ultrasound, pulmonary function testing, and routine clinical laboratory testing. Post Liver Transplant Group- pulmonary function testing.
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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.
Jeffrey Teckman, MD study chair
St. Louis University
Andrew A Wilson, MD study director
Alpha-1 Foundation
David A. Brenner, MD principal investigator
The University of California, San Diego
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Alpha-1 Antitrypsin Deficiency, run by St. Louis University at 3 sites 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 13 inclusion and 27 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: under way, not looking for participants, as of the sponsor's last update on 27 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
The risk and rate of histologic liver injury progression, as measured by liver biopsy, over a 5-year period. (time frame: Liver biopsy performed in Year 1 and Year 5). 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 NCT02014415, which also lists 1 linked publication.
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 27 Apr 2026.