Patients Waiting for a Liver Transplant.LALTH-1NCT02852304
Assessment of the Prevalence of Lysosomal Acid Lipase Deficiency in Patients Waiting for a Liver Transplant.
An observational study of Patients Waiting for a Liver Transplant., run by Hospices Civils de Lyon at 1 site in France.
Trial summary
Lysosomal Acid Lipase (LAL) deficiency is a rare, autosomal recessive storage disease linked to decrease enzymatic activity of LAL, responsible for intracellular accumulation of cholesterol esters and triglycerides. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Patients Waiting for a Liver Transplant.
- How many people
- 100 participants
- Where
- 1 siteFrance
- Study dates
- Oct 2015to Mar 2018
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: The Assessment of the prevalence of Lysosomal Acid Lipase deficiency were performed from few blood drops absorbed onto blotting paper during a routine visit in patients waiting for a liver transplant. Patients will be included when they will be registered on the liver transplant waiting list. The enzymatic analysis onto blotting paper will be made during a routine visit in patients with cryptogenic cirrhosis, NASH (isolated or associated with other liver disease). Oral and written information will be given by the clinician. Only research team members will have access to patient data and their analysis. The patient data (age, gender, medical history, etiology of liver disease, liver function, lipid, glucose, radiological ...) will be collected and stored in an Excel file. The diagnosis analysis deficit LAL will be performed as described in the article by Hamilton.
Who may be able to join
- patients registered on the liver transplant waiting list.
- patients with cryptogenic cirrhosis, NASH
Who cannot join
- Patients without metabolic syndrome clinical, biological or radiological.
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Hépato-Gastro-Entérologie, Hôpital de la Croix Rousse, 103 gde rue de la Croix Rousse
Lyon, 69004
Sylvie Radenne, MD, (0)4 26 10 93 59 ext. +33, sylvie.radenne@chu-lyon.fr
Isabelle Delfour, (0)4 26 73 27 25, isabelle.delfour@chu-lyon.fr
About this study
Lysosomal Acid Lipase (LAL) deficiency is a rare, autosomal recessive storage disease linked to decrease enzymatic activity of LAL, responsible for intracellular accumulation of cholesterol esters and triglycerides.
The accumulation of lipid is in hepatocytes, Kupffer cells and macrophages leading to a fatty liver, hepatic fibrosis that can evolve up to cirrhosis.
Read more
LAL deficiency is responsible for significant morbidity and early mortality in children, adolescents and adults in connection with a multi visceral disease reaching the liver, gastrointestinal tract and the vascular endothelium. The disease is caused by homozygous or heterozygous mutations in the gene (LIPA chromosome 10q23.2-23.3) which is responsible for the synthesis of the LAL.
The disease can be diagnosed by enzymatic analysis using few drops of blood absorbed onto blotting paper .
Patients with this deficiency LAL, have no or reduced activity of this enzyme. Because of its rarity, the deficit in LAL is under diagnosed or is diagnosed in patients with liver biological disturbances and / or lipid profile disturbances, steatohepatitis-hepatitis (NASH), the steatosis (NAFLD), the cryptogenic cirrhosis or Wilson disease.
Inclusion period of 12 to 18 months (100 patients).
- Conditions
- Patients Waiting for a Liver Transplant.
- Keywords
- liver, transplant, Lysosomal Acid Lipase
- Medical subject headings
- Cholesterol Ester Storage Disease
What is being measured
The primary outcome is the main question the study is designed to answer.
Deficiency Lysosomal Acid Lipase prevalence in patients waiting for a liver transplant
Assessment of deficiency Lysosomal Acid Lipase prevalence in patients waiting for a liver transplant
Measured: During the routine visit (Day 1)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Aug 2016 (estimated)
- Study startOct 2015
- Primary completionOct 2017 (estimated)
- Study completionMar 2018 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Patients Waiting for a Liver Transplant.Studies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe sponsor's other registered studies.
- 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
- 100 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Sylvie Radenne, MD, Principal investigator, Hospices Civils de Lyon
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02852304
- Sponsor's protocol ID
- 69HCL16_0428
- Acronym
- LALTH-1
- Status last verified
- Jul 2016
- First submitted
- 28 Jul 2016
- Last updated
- 2 Aug 2016
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of the Prevalence of Lysosomal Acid Lipase Deficiency in Patients Waiting for a Liver Transplant.
- ClinicalTrials.gov ID
- NCT02852304
- Acronym / sponsor ID
- LALTH-1 / 69HCL16_0428
- Conditions
- Patients Waiting for a Liver Transplant.
- Keywords
- liver, transplant, Lysosomal Acid Lipase
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 100 participants (estimated)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- Oct 2015
- Primary completion
- Oct 2017 (estimated)
- Study completion
- Mar 2018 (estimated)
- First posted
- 2 Aug 2016 (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.
Lysosomal Acid Lipase (LAL) deficiency is a rare, autosomal recessive storage disease linked to decrease enzymatic activity of LAL, responsible for intracellular accumulation of cholesterol esters and triglycerides.
The accumulation of lipid is in hepatocytes, Kupffer cells and macrophages leading to a fatty liver, hepatic fibrosis that can evolve up to cirrhosis.
LAL deficiency is responsible for significant morbidity and early mortality in children, adolescents and adults in connection with a multi visceral disease reaching the liver, gastrointestinal tract and the vascular endothelium. The disease is caused by homozygous or heterozygous mutations in the gene (LIPA chromosome 10q23.2-23.3) which is responsible for the synthesis of the LAL.
The disease can be diagnosed by enzymatic analysis using few drops of blood absorbed onto blotting paper .
Patients with this deficiency LAL, have no or reduced activity of this enzyme. Because of its rarity, the deficit in LAL is under diagnosed or is diagnosed in patients with liver biological disturbances and / or lipid profile disturbances, steatohepatitis-hepatitis (NASH), the steatosis (NAFLD), the cryptogenic cirrhosis or Wilson disease.
Inclusion period of 12 to 18 months (100 patients).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- The Assessment of the prevalence of Lysosomal Acid Lipase deficiency were performed from few blood drops absorbed onto blotting paper during a routine visit in patients waiting for a liver transplant. Patients will be included when they will be registered on the liver transplant waiting list. The enzymatic analysis onto blotting paper will be made during a routine visit in patients with cryptogenic cirrhosis, NASH (isolated or associated with other liver disease). Oral and written information will be given by the clinician. Only research team members will have access to patient data and their analysis. The patient data (age, gender, medical history, etiology of liver disease, liver function, lipid, glucose, radiological ...) will be collected and stored in an Excel file. The diagnosis analysis deficit LAL will be performed as described in the article by Hamilton.
Key inclusion criteria (2 listed)
- patients registered on the liver transplant waiting list.
- patients with cryptogenic cirrhosis, NASH
Key exclusion criteria (1 listed)
- Patients without metabolic syndrome clinical, biological or radiological.
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
Deficiency Lysosomal Acid Lipase prevalence in patients waiting for a liver transplant
Time frame: During the routine visit (Day 1)
Assessment of deficiency Lysosomal Acid Lipase prevalence in patients waiting for a liver transplant
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Aug 2016.
Related trials for Patients Waiting for a Liver Transplant.
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Cirrhosis, Registered on Transplant List
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.
Sylvie Radenne, MD principal investigator
Hospices Civils de Lyon
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Patients Waiting for a Liver Transplant., run by Hospices Civils de Lyon at 1 site in France.
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 2 inclusion and 1 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 2 Aug 2016. 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 France. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Sylvie Radenne, MD ((0)4 26 10 93 59 ext. +33, sylvie.radenne@chu-lyon.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Deficiency Lysosomal Acid Lipase prevalence in patients waiting for a liver transplant (time frame: During the routine visit (Day 1)). 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 2 Aug 2016.