Examination of the Lipid Metabolism of the Liver After Choline Substitution in Cystic Fibrosis

Pilot study to investigate the effect of choline chloride in cystic fibrosis patients with liver steatosis by comparing their status before and after the intervention

Study Overview

• Status: Completed
• Conditions: Cystic Fibrosis Liver Disease
• Intervention / Treatment: Drug: Choline Chloride

Detailed Description

Within this single arm pilot study patients with cystic fibrosis and liver steatosis receive choline chloride (2x0.5 gram three times a day) as a food supply for 88 days. D9-choline metabolism is measured before and after the intervention phase. Hypothesis of the study is, that supplying choline augments the secretion of phosphatidyl choline and lipoprotein (VLDL) from the liver and therefore results in a measurable reduction of the triglyceride storage of the liver.

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Not Applicable

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (ADULT, OLDER_ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Inclusion Criteria:

• Age > 18 years
• male
• Cystic fibrosis is verified
• pancreatic insufficiency
• Decline of plasma phosphatidylcholine and steatosis of the liver
• informed consent is given

Exclusion Criteria:

• cirrhosis of the liver
• Hyperreactivity to choline containing food
• allergic bronchopulmonary aspergillosis or other acute exacerbations of pulmonary infections
• FEV1 < 40%
• smoker
• chronic alcohol consume
• clinically relevant diseases (for example thyroid dysfunction, disturbance of lipid metabolism, renal diseases, significant left or right ventricular functional restriction) which will change the lipid metabolism of the liver
• Implants or other reasons which make magnetic resonance examinations impossible

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
OTHER: Choline Chloride; Patients receive choline chloride (1g Choline) three times a day for 12.6 weeks as a food supply	Drug: Choline Chloride

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Measurement of D9-Methyl-Choline Kinetic	The substitution of choline as a food supplement will augment the phosphatidylcholine and lipoprotein (VLDL) secretion of the liver into the blood and will therefore result in a reduction of the triglyceride retention in the liver	3 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Lipid storage of the liver	Comparison of steatosis of the liver (measured by magnetic resonance spectroscopy or sonography) before to after the choline substitution	3 months
Augmentation of creatinine concentration within the calf muscle	Augmentation of creatine concentration within the calf muscle due to the choline substitution measured by magnetic resonance spectroscopy	3 months
FVC	Comparison of FVC before to after the choline substitution	3 months
FEV1	Comparison of FEV1 before to after the choline substitution	3 months
MEF25	Comparison of MEF25 before to after the choline substitution	3 months
MMEF	Comparison of MMEF before to after the choline substitution	3 months

Collaborators and Investigators

• Sponsor: University Hospital Tuebingen
• Investigators: Principal Investigator: Joachim Riethmüller, PD, Klinik für Kinder- und Jugendmedizin Tübingen

Study record dates

Study Major Dates

• Study Start (ACTUAL): November 6, 2014
• Primary Completion (ACTUAL): April 24, 2016
• Study Completion (ACTUAL): February 8, 2017

Study Registration Dates

• First Submitted: October 4, 2017
• First Submitted That Met QC Criteria: October 16, 2017
• First Posted (ACTUAL): October 17, 2017

Study Record Updates

• Last Update Posted (ACTUAL): November 30, 2017
• Last Update Submitted That Met QC Criteria: November 29, 2017
• Last Verified: October 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Pathologic Processes; Respiratory Tract Diseases; Lung Diseases; Infant, Newborn, Diseases; Genetic Diseases, Inborn; Pancreatic Diseases; Liver Diseases; Fibrosis; Cystic Fibrosis; Molecular Mechanisms of Pharmacological Action; Antimetabolites; Gastrointestinal Agents; Hypolipidemic Agents; Lipid Regulating Agents; Nootropic Agents; Lipotropic Agents; Choline
• Other Study ID Numbers: Phos-CF II V1.2

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No