Radiological and biochemical effects in children, adolescents and young adults under supplementation with the dietary supplement choline on cystic fibrosis-associated liver disease.

Not Applicable

Recruiting

• Conditions: Cystic fibrosis associated liver disease; E84.9; Cystic fibrosis, unspecified

• Registration Number: DRKS00032870
• Lead Sponsor: niversitätsklinik Tübingen

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-10-16
• Last Update Posted: 19 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 50

Inclusion Criteria

Patients aged 6 years and older up to the age of 25 years 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

- 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).

Study & Design

• Study Type: interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Primary outcome criterion: <br>Improvement of CF-associated liver disease (steatosis, transaminase elevation):<br>- Decrease in liver fat content (on MRI [visit 4] or ultrasound [visits 3 and 4]).<br>- Decrease in liver volume (in MRI [Rounds 4]).<br>- Improvement of hepatocellular liver elevations (GOT, GPT, gGT, AP [Visit 4])

Secondary Outcome Measures

Name	Time	Method
Secondary outcome criteria:<br>- Comparability of ultrasonography (ATI) and magnetic resonance imaging for quantification of liver fat [Visite 2 and 4].<br>- Normalization of plasma concentration of choline [Visit 2 to Visit 4].