Liver Post-transplant Patients Clinical Trial
— LALTH-2Official title:
Assessement of the Prevalence of Lysosomal Acid Lipase Deficiency in Liver Post-transplant Patients
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).
| Status | Recruiting |
| Enrollment | 100 |
| Est. completion date | June 2018 |
| Est. primary completion date | December 2017 |
| Accepts healthy volunteers | No |
| Gender | Both |
| Age group | 18 Years and older |
| Eligibility |
Inclusion Criteria: - liver post-transplant patient - patients with cryptogenic cirrhosis, NASH Exclusion Criteria: - Patients without metabolic syndrome clinical, biological or radiological. |
Observational Model: Cohort, Time Perspective: Prospective
| Country | Name | City | State |
|---|---|---|---|
| France | Hépato-Gastro-Entérologie, Hôpital de la Croix Rousse, 103 gde rue de la Croix Rousse | Lyon |
| Lead Sponsor | Collaborator |
|---|---|
| Hospices Civils de Lyon |
France,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Prevalence of Lysosomal Acid Lipase deficiency in liver post-transplant patients | Assessment of the prevalence of Lysosomal Acid Lipase deficiency in liver post-transplant patients | During the routine visit (Day 1) | No |