Cystic Fibrosis Liver DiseaseNCT03312140
Examination of the Lipid Metabolism of the Liver After Choline Substitution in Cystic Fibrosis
A study testing Choline Chloride in people with Cystic Fibrosis Liver Disease, run by University Hospital Tuebingen.
Trial summary
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 Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Cystic Fibrosis Liver Disease
- What is being tested?
- Choline Chloride
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- No sites listed yet
- Study dates
- 6 Nov 2014to 8 Feb 2017
- Phase
- Interventional
- 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
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age > 18 years
- male
- Cystic fibrosis is verified
- pancreatic insufficiency
- Decline of plasma phosphatidylcholine and steatosis of the liver
- informed consent is given
Who cannot join
- 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
About this study
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
Read the 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.
- Conditions
- Cystic Fibrosis Liver Disease
- Being tested
- Choline Chloride
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Choline ChlorideDrug
| Study group | Role | What they receive |
|---|---|---|
| Choline Chloride | Other | Patients receive choline chloride (1g Choline) three times a day for 12.6 weeks as a food supply Receives: Drug: Choline Chloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 3 months
Secondary outcomes (6)
Lipid storage of the liver
Comparison of steatosis of the liver (measured by magnetic resonance spectroscopy or sonography) before to after the choline substitution
Measured: 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
Measured: 3 months
FVC
Comparison of FVC before to after the choline substitution
Measured: 3 months
FEV1
Comparison of FEV1 before to after the choline substitution
Measured: 3 months
MEF25
Comparison of MEF25 before to after the choline substitution
Measured: 3 months
MMEF
Comparison of MMEF before to after the choline substitution
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Oct 2017
- Study start6 Nov 2014
- Primary completion24 Apr 2016
- Study completion8 Feb 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic Fibrosis Liver DiseaseStudies of the same condition, open and under way.
- More trials run by University Hospital TuebingenThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Pilot study to evolve the effect ot the treatment by comparing data before and after the treatment within the same patients
- Masking
- None (open label)
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital Tuebingen
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Joachim Riethmüller, PD, Principal investigator, Klinik für Kinder- und Jugendmedizin Tübingen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03312140
- Sponsor's protocol ID
- Phos-CF II V1.2
- Status last verified
- Oct 2017
- First submitted
- 4 Oct 2017
- Last updated
- 30 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Examination of the Lipid Metabolism of the Liver After Choline Substitution in Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT03312140
- Acronym / sponsor ID
- Phos-CF II V1.2
- Conditions
- Cystic Fibrosis Liver Disease
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University Hospital TuebingenUniversity, hospital or other
- Study start
- 6 Nov 2014 (actual)
- Primary completion
- 24 Apr 2016 (actual)
- Study completion
- 8 Feb 2017 (actual)
- First posted
- 17 Oct 2017 (actual)
- 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.
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
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Pilot study to evolve the effect ot the treatment by comparing data before and after the treatment within the same patients
| Arm | Type | Receives |
|---|---|---|
| Choline Chloride | Other | Drug: Choline ChloridePatients receive choline chloride (1g Choline) three times a day for 12.6 weeks as a food supply |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age > 18 years
- male
- Cystic fibrosis is verified
- pancreatic insufficiency
- Decline of plasma phosphatidylcholine and steatosis of the liver
Key exclusion criteria (8 listed)
- cirrhosis of the liver
- Hyperreactivity to choline containing food
- allergic bronchopulmonary aspergillosis or other acute exacerbations of pulmonary infections
- FEV1 < 40%
- smoker
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Choline Chloride | Drug | Choline Chloride |
Primary outcome
Measurement of D9-Methyl-Choline Kinetic
Time frame: 3 months
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
6 secondary outcomes
Lipid storage of the liver
Time frame: 3 months
Augmentation of creatinine concentration within the calf muscle
Time frame: 3 months
FVC
Time frame: 3 months
FEV1
Time frame: 3 months
MEF25
Time frame: 3 months
MMEF
Time frame: 3 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2017.
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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.
Joachim Riethmüller, PD principal investigator
Klinik für Kinder- und Jugendmedizin Tübingen
Questions about this trial
What is this clinical trial studying?
A study testing Choline Chloride in people with Cystic Fibrosis Liver Disease, run by University Hospital Tuebingen.
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 6 inclusion and 8 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: finished, as of the sponsor's last update on 30 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measurement of D9-Methyl-Choline Kinetic (time frame: 3 months). 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 30 Nov 2017.