Intestinal Failure-associated Liver DiseaseTHRIVE-1NCT05011370
Prevalence of Liver Disease in Patients Dependent on Parenteral Nutrition
An observational study of Intestinal Failure-associated Liver Disease, run by Protara Therapeutics at 15 sites in 4 countries.
Trial summary
This is a multi-center prospective cross-sectional observational study that will assess the prevalence of liver disease in patients dependent on parenteral nutrition (PN) for 4 or more days per week. Read more about this study
- Who is it for?
- Ages 12 to 80 years
- Conditions the study is for
- Intestinal Failure-associated Liver Disease
- How many people
- 80 participants
- Where
- 15 sitesin 4 countries
- Study dates
- 23 Aug 2021to 29 Jun 2023
- Phase
- Observational
- 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 12 to 80 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: This study will enroll male and female adults and adolescent participants 12 years and older who are dependent on PN (≥ 4 days a week) in the primary care clinic setting.
Who may be able to join
- The participant and/or their parent/Legally Authorized Representative is willing and able to provide signed informed consent or assent as appropriate
- Male or female adults 18 to 80 years of age, or adolescents 12 to 17 years of age
- Patients dependent on parenteral nutrition (PN) that receive PN for an average ≥ 4 days a week for 10 weeks or longer prior to screening to meet nutritional, caloric, fluid, and/or electrolyte needs
- The Investigator expects no changes in the lipid, dextrose, amino acid, or vitamin regimen to be medically necessary during the participant's participation in the study
- Willingness of participant to maintain his/her current habitual oral diet and fluids regimen for the study duration
Who cannot join
- Participants taking steatogenic medications for ≥12 weeks in the past 12 months (e.g., amiodarone, tamoxifen, methotrexate, tetracycline, glucocorticoids, anabolic steroids, over the usual dose of estrogen for hormone replacement therapy, and valproate); those taking any medicine (e.g., metformin, thiazolidinediones, ursodeoxycholic acid, pentoxifylline, S-adenosyl-L-Methionine, and betaine) that could affect the measurement of IFALD within 12 weeks prior to study entry
- Participants taking potential hepatotoxic medications that in the judgement of the Investigator is causing hepatic abnormalities
- Participants with a cardiac pacemaker, intravascular stents, other metallic devices, and claustrophobia which are contraindicated to magnetic resonance imaging
- Participants who took choline supplements or choline-containing multivitamins within 14 days of screening
- History of major organ transplant (e.g., heart, kidney, liver, etc.)
- For more information on eligibility criteria, please contact the sponsor.
Trial locations and contacts
15 sites in 4 countries.
15 sites in 4 countries
United States10 sites
The University of Chicago
Chicago, Illinois, 60637
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Duke University Medical Center
Durham, North Carolina, 27710
IHS Health
Kissimmee, Florida, 34741
Ronald Reagan Medical Center of UCLA
Los Angeles, California, 90095
UPMC Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224
Virginia Commonwealth University
Richmond, Virginia, 23298
Mayo Clinic
Rochester, Minnesota, 55905
Seattle Children's
Seattle, Washington, 98105
United Kingdom3 sites
St. Mark's Hospital
Harrow, England, HA1 3UJ
University College London Hospitals
London, England, NW1 2BU
Northern Care Alliance NHS Foundation
Salford, England
Italy1 site
Center for Chronic Intestinal Failure, St. Orsola Hospital Dept of Medical and Surgical Sciences, University of Bologna
Bologna
About this study
Official title: A Prospective Prevalence Study in Adolescent and Adult Patients Dependent on Parenteral Nutrition to Assess the Incidence of Intestinal Failure-Associated Liver Disease
This is a multi-center prospective cross-sectional observational study that will assess the prevalence of liver disease in patients dependent on parenteral nutrition (PN) for 4 or more days per week. Liver disease will be determined by the presence of choline deficiency, cholestasis (confirmed by elevated serum alkaline phosphatase (ALP) liver isoenzyme level), and steatosis (confirmed by magnetic resonance imaging-derived proton density fat fraction (MRI-PDFF).
The objective of this study is to investigate the presence/prevalence of liver disease in patients dependent on PN (≥4 days a week).
Read the detailed description
Eligible participants will provide blood samples at Visit 1 to determine choline deficiency, elevated serum ALP liver isoenzyme level and liver dysfunction, and complete one imaging study (MRI-PDFF) at Visit 1 to assess steatosis.
The purpose of this study is to understand and document critical epidemiological data related to choline deficiency and the incidence of liver disease in patients dependent on PN (≥ 4 days a week) and to better understand this patient population to help determine who might benefit from innovative treatments including IV Choline Chloride treatment.
- Keywords
- Intestinal failure-associated liver disease (IFALD), IFALD, Liver disease, Parenteral nutrition, Choline deficiency, Hepatic steatosis, Cholestasis, parenteral nutrition-associated liver disease
- Medical subject headings
- Liver Diseases, Hyperphagia, Choline Deficiency, Fatty Liver, Cholestasis
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of patients with choline deficiency
Choline deficiency is defined as plasma-free choline concentration less than 7 nmol/mL.
Measured: Day 1
Percentage of patients with choline deficiency, elevated serum ALP liver isoenzyme level and MRI-PDFF ≥8%
Choline deficiency is defined as plasma-free choline concentration less than 7 nmol/mL. Elevated serum ALP liver isoenzyme level is defined as >1.5x upper limit of normal. MRI-PDFF ≥8 %
Measured: Day 1
Secondary outcomes (3)
Percentage of patients with MRI-PDFF ≥8%
Assessment of liver tests all patients and patients with choline deficiency. Choline deficiency is defined as plasma-free choline concentration less than 7 nmol/mL;
Measured: Day 1
Percentage of patients with elevated serum ALP liver isoenzyme level (defined as ≥ 1.5x upper limit of normal)
Assessment of liver tests all patients and patients with choline deficiency. Choline deficiency is defined as plasma-free choline concentration less than 7 nmol/mL;
Measured: Day 1
Percentage of patients with abnormal liver tests including serum direct bilirubin, AST, ALT and GGT
Assessment of liver tests all patients and patients with choline deficiency. Choline deficiency is defined as plasma-free choline concentration less than 7 nmol/mL; AST = aspartate aminotransferase; ALT = alanine transaminase; GGT = gamma-glutamyl transferase.
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Aug 2021
- Study start23 Aug 2021
- Primary completion29 Jun 2023
- Study completion29 Jun 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Intestinal Failure-associated Liver DiseaseStudies 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
- 80 participants (actual)
Sponsor and investigators
- Lead sponsor
- Protara Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Chief Scientific Operations Officer, Study director, Protara Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05011370
- Sponsor's protocol ID
- TARA-001-502
- Acronym
- THRIVE-1
- Status last verified
- Sep 2023
- First submitted
- 11 Aug 2021
- Last updated
- 3 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective Prevalence Study in Adolescent and Adult Patients Dependent on Parenteral Nutrition to Assess the Incidence of Intestinal Failure-Associated Liver Disease
- ClinicalTrials.gov ID
- NCT05011370
- Acronym / sponsor ID
- THRIVE-1 / TARA-001-502
- Keywords
- Intestinal failure-associated liver disease (IFALD), IFALD, Liver disease, Parenteral nutrition, Choline deficiency, Hepatic steatosis, Cholestasis, parenteral nutrition-associated liver disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 80 participants (actual)
- Lead sponsor
- Protara TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 23 Aug 2021 (actual)
- Primary completion
- 29 Jun 2023 (actual)
- Study completion
- 29 Jun 2023 (actual)
- First posted
- 18 Aug 2021 (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 multi-center prospective cross-sectional observational study that will assess the prevalence of liver disease in patients dependent on parenteral nutrition (PN) for 4 or more days per week. Liver disease will be determined by the presence of choline deficiency, cholestasis (confirmed by elevated serum alkaline phosphatase (ALP) liver isoenzyme level), and steatosis (confirmed by magnetic resonance imaging-derived proton density fat fraction (MRI-PDFF).
The objective of this study is to investigate the presence/prevalence of liver disease in patients dependent on PN (≥4 days a week).
Detailed description
Eligible participants will provide blood samples at Visit 1 to determine choline deficiency, elevated serum ALP liver isoenzyme level and liver dysfunction, and complete one imaging study (MRI-PDFF) at Visit 1 to assess steatosis.
The purpose of this study is to understand and document critical epidemiological data related to choline deficiency and the incidence of liver disease in patients dependent on PN (≥ 4 days a week) and to better understand this patient population to help determine who might benefit from innovative treatments including IV Choline Chloride treatment.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 12 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- This study will enroll male and female adults and adolescent participants 12 years and older who are dependent on PN (≥ 4 days a week) in the primary care clinic setting.
Key inclusion criteria (5 listed)
- The participant and/or their parent/Legally Authorized Representative is willing and able to provide signed informed consent or assent as appropriate
- Male or female adults 18 to 80 years of age, or adolescents 12 to 17 years of age
- Patients dependent on parenteral nutrition (PN) that receive PN for an average ≥ 4 days a week for 10 weeks or longer prior to screening to meet nutritional, caloric, fluid, and/or electrolyte needs
- The Investigator expects no changes in the lipid, dextrose, amino acid, or vitamin regimen to be medically necessary during the participant's participation in the study
- Willingness of participant to maintain his/her current habitual oral diet and fluids regimen for the study duration
Key exclusion criteria (6 listed)
- Participants taking steatogenic medications for ≥12 weeks in the past 12 months (e.g., amiodarone, tamoxifen, methotrexate, tetracycline, glucocorticoids, anabolic steroids, over the usual dose of estrogen for hormone replacement therapy, and valproate); those taking any medicine (e.g., metformin, thiazolidinediones, ursodeoxycholic acid, pentoxifylline, S-adenosyl-L-Methionine, and betaine) that could affect the measurement of IFALD within 12 weeks prior to study entry
- Participants taking potential hepatotoxic medications that in the judgement of the Investigator is causing hepatic abnormalities
- Participants with a cardiac pacemaker, intravascular stents, other metallic devices, and claustrophobia which are contraindicated to magnetic resonance imaging
- Participants who took choline supplements or choline-containing multivitamins within 14 days of screening
- History of major organ transplant (e.g., heart, kidney, liver, etc.)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Percentage of patients with choline deficiency
Time frame: Day 1
Choline deficiency is defined as plasma-free choline concentration less than 7 nmol/mL.
Percentage of patients with choline deficiency, elevated serum ALP liver isoenzyme level and MRI-PDFF ≥8%
Time frame: Day 1
Choline deficiency is defined as plasma-free choline concentration less than 7 nmol/mL. Elevated serum ALP liver isoenzyme level is defined as >1.5x upper limit of normal. MRI-PDFF ≥8 %
3 secondary outcomes
Percentage of patients with MRI-PDFF ≥8%
Time frame: Day 1
Percentage of patients with elevated serum ALP liver isoenzyme level (defined as ≥ 1.5x upper limit of normal)
Time frame: Day 1
Percentage of patients with abnormal liver tests including serum direct bilirubin, AST, ALT and GGT
Time frame: Day 1
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 3 Oct 2023.
- Individual participant dataSharing planned: No
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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.
Chief Scientific Operations Officer study director
Protara Therapeutics
15 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Intestinal Failure-associated Liver Disease, run by Protara Therapeutics at 15 sites in 4 countries.
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 5 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: finished, as of the sponsor's last update on 3 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in United States, United Kingdom, Denmark, Italy. 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?
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?
Percentage of patients with choline deficiency (time frame: Day 1). 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 3 Oct 2023.