Cystic fibrosis associated liver disease E84.9
Conditions
Interventions
Group 1: Group 1: CF patients with CFALD and proven choline deficiency substituted with dietary choline supplement.
The study includes 5 study visits.
Visit 1 (Day 0): Screening with medical history a
Sponsors
Universitätsklinik Tübingen
Eligibility
Sex/Gender
All
Age
6 Years to No maximum
Inclusion criteria
Inclusion criteria: - Patients aged 6 years and older with a diagnosis of cystic fibrosis. - Detection of the CFTR genotype - Exocrine pancreatic insufficiency - Presence of CF-associated hepatopathy (steatosis, elevation of hepatocellular enzymes, liver parenchymal changes in the sense of fibrosis). - Laboratory evidence of at least once decreased plasma concentration of choline (<8.1µmol/l) with recommendation for supplementation with a choline-containing food supplement. - Written informed consent and agreement to the data protection declaration
Exclusion criteria
Exclusion criteria: - Lack of informed consent - Patients who want to implement the recommendation for choline supplementation in any case, i.e., cannot be randomized - Chronic alcohol consumption - Concomitant liver disease apart from CFALD, such as infectious or congenital or autoimmune liver disease, Meulengracht disease, or Wilson disease - Irreversible cirrhotic liver remodeling - Patients in whom there is no indication for blood sampling outside of the trial - Critically ill patients who cannot be expected to undergo additional sonography or MRI examinations or who have a contraindication to MRI examinations - Patients who require sedation/anesthesia to undergo sonography or MRI examination. - Participation in another interventional study or planned start of another interventional study within the study period planned here, for example with a disease modifier (modulator).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary outcome criterion: Improvement of CF-associated liver disease (steatosis, transaminase elevation): - Decrease in liver fat content (on MRI [visit 4] or ultrasound [visits 3 and 4]). - Decrease in liver volume (in MRI [Rounds 4]). - Improvement of hepatocellular liver elevations (GOT, GPT, gGT, AP [Visit 4]) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary outcome criteria: - Comparability of ultrasonography (ATI) and magnetic resonance imaging for quantification of liver fat [Visite 2 and 4]. - Normalization of plasma concentration of choline [Visit 2 to Visit 4]. | — |
Countries
Germany
Contacts
Public ContactJohannes Hilberath
Universitätsklinik Tübingen
Outcome results
None listed