Congenital Heart DiseaseNCT03886935
Metabolic Remodeling in Fontan Patients
An observational study of Congenital Heart Disease and Metabolism Disorder, run by Medical University Innsbruck.
Trial summary
Assessment of metabolic alterations in adult Fontan patients with a dominant left ventricle with the help of serum examinations (Metabolomics). The aim is to find a tool for the completion of the (semi-)invasive monitoring of Fontan hemodynamics. Read more about this study
- Who is it for?
- Any ageHealthy volunteers can take part
- Conditions the study is for
- Congenital Heart Disease, Metabolism Disorder
- What is being tested?
- Metabolomics
- How many people
- 100 participants
- Where
- No sites listed yet
- Study dates
- 1 Jan 2017to 1 Jan 2019
- Phase
- Observational
- 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 Any age
- All sexes
- Healthy volunteers accepted
- Child, Adult, Older adult
Study population: Patients ≥ 18 years with a Fontan circulation with a dominant left ventricle. Controls: age- and sex-matched healthy biventricular controls ≥ 18 years
Who may be able to join
- Fontan circulation (right systemic ventricle), biventricular heart with heart failure resp.
- ≥ 18 years
- Written informed consent of patients
- 8 hours fasting period before blood sample
Who cannot join
- Intake of medication directly affecting metabolic (e.g. metabolism of lipids (statine)) or hemodynamic state (e.g. beta-blockers, sildenafil) (other than angiotensin converting enzyme (ACE)-inhibitors and anticoagulation therapy)
- Cachectic disease
- Non-congestive hepatic or renal dysfunction
- (Inherited) metabolic disorders
About this study
Official title: Metabolic Remodeling in Fontan Patients: a Metabolomics Study
Assessment of metabolic alterations in adult Fontan patients with a dominant left ventricle with the help of serum examinations (Metabolomics). The aim is to find a tool for the completion of the (semi-)invasive monitoring of Fontan hemodynamics.
Read the detailed description
Patients who are born with just one single heart chamber need to undergo surgical therapy allowing the single heart chamber to pump the blood into the systemic circulation and allowing the blood to flow passively to the lungs (Fontan circulation). Regular ultrasound, cardiopulmonary exercise testing, and invasive diagnostic tools (catheterization, general anaesthesia needed) are necessary to early find out cardiac, vascular, or circulatory impairment. It is still very difficult to diagnose and therapy failure of this Fontan system early enough.
It is reported that in patients with a failing two-chambered heart, the energy source for the heart and the body in general switches from the use of lipids to the use of sugar and ketone bodies. First studies show decreased concentrations of membrane lipids in Fontan patients with a left dominant ventricle, and the energy metabolism has not been focused yet in those patients.
The investigators hypothesize that there are differences in the pattern of the structural metabolism in adult Fontan patients with a left-dominant vs. a right-dominant ventricle. Furthermore the investigators hypothesize that there are alterations in the energy metabolism in adult Fontan patients in comparison to healthy two-chambered controls, and that those alterations correlate with the grade of impairment of cardiopulmonary function.
With the help of a special biochemical examination (mass spectrometry-based Metabolomics study) blood of Fontan patients will be analyzed, and the results will be correlated with the results of ultrasound and cardiopulmonary exercise testing. The aim of this study is to establish sensitive blood markers indicating cardiac, vascular, circulatory or further organ dysfunction in Fontan patients. This should allow optimal Fontan system monitoring with an optimal timing of an additional invasive diagnostic catheterization and of nutritional, medical or interventional therapy.
- Conditions
- Congenital Heart Disease, Metabolism Disorder
- Being tested
- Metabolomics
- Medical subject headings
- Heart Defects, Congenital, Metabolic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MetabolomicsDiagnostic test
| Study group | Role | What they receive |
|---|---|---|
| Fontan patients | The serum of Fontan patients is compared to the serum of healthy biventricular controls (Metabolomics) Receives: Diagnostic Test: Metabolomics | |
| Healthy biventricular controls | The serum of Fontan patients is compared to the serum of healthy biventricular controls (Metabolomics) Receives: Diagnostic Test: Metabolomics |
What is being measured
The primary outcome is the main question the study is designed to answer.
Metabolism of lipids
Measurement of the serum concentration of the extended metabolism of lipids using the technique of Metabolomics (AbsoluteIDQ p180 kit, Biocrates Life Sciences AG, Innsbruck, Austria)
Measured: blood sampling at one single point of time (baseline/day 1) in the context of a planned patient presentation at the pediatric cardiology outpatient clinic (without assessment of changes)
Metabolism of amino acids and related compounds
Measurement of the serum concentration of the extended metabolism of lamino acids using the technique of Metabolomics (AbsoluteIDQ p180 kit, Biocrates Life Sciences AG, Innsbruck, Austria)
Measured: blood sampling at one single point of time (baseline/day 1) in the context of a planned patient presentation at the pediatric cardiology outpatient clinic (without assessment of changes)
Metabolism of carbohydrates
Measurement of the serum concentration of the extended metabolism of carbohydrates using the technique of Metabolomics (AbsoluteIDQ p180 kit, Biocrates Life Sciences AG, Innsbruck, Austria)
Measured: blood sampling at one single point of time (baseline/day 1) in the context of a planned patient presentation at the pediatric cardiology outpatient clinic (without assessment of changes)
Metabolism of ketone bodies
Measurement of the serum concentration of ketone bodies using the technique of Metabolomics (AbsoluteIDQ p180 kit, Biocrates Life Sciences AG, Innsbruck, Austria)
Measured: blood sampling at one single point of time (baseline/day 1) in the context of a planned patient presentation at the pediatric cardiology outpatient clinic (without assessment of changes)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2019
- Study start1 Jan 2017
- Primary completion31 Dec 2018
- Study completion1 Jan 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Congenital Heart DiseaseStudies of the same condition, open and under way.
- More trials run by Medical University InnsbruckThe 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
- Observational
- Observational model
- Case-control
- Time perspective
- Prospective
- Enrolment
- 100 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical University Innsbruck
- Sponsor type
- University, hospital or other
- Collaborators
- Biocrates Life Sciences AG, Innsbruck, Austria, Tiroler Wissenschaftsförderung
- Responsible party
- Sponsor
- Investigators
- Daniela Karall, Prof., Principal investigator, Medical University Innsbruck
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03886935
- Sponsor's protocol ID
- 201810011837
- Status last verified
- Jan 2017
- First submitted
- 11 Mar 2019
- Last updated
- 24 Feb 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Participant data will be available only to the head of the study (Dr. Miriam Michel) (Excel- and SPSS-files)
Publications
Michel M, Salvador C, Wiedemair V, Adam MG, Laser KT, Dubowy KO, Entenmann A, Karall D, Geiger R, Zlamy M, Scholl-Burgi S. Method comparison of HPLC-ninhydrin-photometry and UHPLC-PITC-tandem mass spectrometry for serum amino acid analyses in patients with complex congenital heart disease and controls. Metabolomics. 2020 Dec 15;16(12):128. doi: 10.1007/s11306-020-01741-8.
PubMed 33319318Michel M, Dubowy KO, Zlamy M, Karall D, Adam MG, Entenmann A, Keller MA, Koch J, Odri Komazec I, Geiger R, Salvador C, Niederwanger C, Muller U, Scholl-Burgi S, Laser KT. Targeted metabolomic analysis of serum phospholipid and acylcarnitine in the adult Fontan patient with a dominant left ventricle. Ther Adv Chronic Dis. 2020 Apr 27;11:2040622320916031. doi: 10.1177/2040622320916031. eCollection 2020.
PubMed 32426103
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Metabolic Remodeling in Fontan Patients: a Metabolomics Study
- ClinicalTrials.gov ID
- NCT03886935
- Acronym / sponsor ID
- 201810011837
- Conditions
- Congenital Heart Disease, Metabolism Disorder
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 100 participants (actual)
- Lead sponsor
- Medical University InnsbruckUniversity, hospital or other
- Collaborators
- Biocrates Life Sciences AG, Innsbruck, Austria, Tiroler Wissenschaftsförderung
- Study start
- 1 Jan 2017 (actual)
- Primary completion
- 31 Dec 2018 (actual)
- Study completion
- 1 Jan 2019 (actual)
- First posted
- 22 Mar 2019 (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.
Assessment of metabolic alterations in adult Fontan patients with a dominant left ventricle with the help of serum examinations (Metabolomics). The aim is to find a tool for the completion of the (semi-)invasive monitoring of Fontan hemodynamics.
Detailed description
Patients who are born with just one single heart chamber need to undergo surgical therapy allowing the single heart chamber to pump the blood into the systemic circulation and allowing the blood to flow passively to the lungs (Fontan circulation). Regular ultrasound, cardiopulmonary exercise testing, and invasive diagnostic tools (catheterization, general anaesthesia needed) are necessary to early find out cardiac, vascular, or circulatory impairment. It is still very difficult to diagnose and therapy failure of this Fontan system early enough.
It is reported that in patients with a failing two-chambered heart, the energy source for the heart and the body in general switches from the use of lipids to the use of sugar and ketone bodies. First studies show decreased concentrations of membrane lipids in Fontan patients with a left dominant ventricle, and the energy metabolism has not been focused yet in those patients.
The investigators hypothesize that there are differences in the pattern of the structural metabolism in adult Fontan patients with a left-dominant vs. a right-dominant ventricle. Furthermore the investigators hypothesize that there are alterations in the energy metabolism in adult Fontan patients in comparison to healthy two-chambered controls, and that those alterations correlate with the grade of impairment of cardiopulmonary function.
With the help of a special biochemical examination (mass spectrometry-based Metabolomics study) blood of Fontan patients will be analyzed, and the results will be correlated with the results of ultrasound and cardiopulmonary exercise testing. The aim of this study is to establish sensitive blood markers indicating cardiac, vascular, circulatory or further organ dysfunction in Fontan patients. This should allow optimal Fontan system monitoring with an optimal timing of an additional invasive diagnostic catheterization and of nutritional, medical or interventional therapy.
Study design
- Observational model
- Case-control
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Fontan patients | – | Diagnostic Test: MetabolomicsThe serum of Fontan patients is compared to the serum of healthy biventricular controls (Metabolomics) |
| Healthy biventricular controls | – | Diagnostic Test: MetabolomicsThe serum of Fontan patients is compared to the serum of healthy biventricular controls (Metabolomics) |
Who may qualify
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients ≥ 18 years with a Fontan circulation with a dominant left ventricle. Controls: age- and sex-matched healthy biventricular controls ≥ 18 years
Key inclusion criteria (4 listed)
- Fontan circulation (right systemic ventricle), biventricular heart with heart failure resp.
- ≥ 18 years
- Written informed consent of patients
- 8 hours fasting period before blood sample
Key exclusion criteria (4 listed)
- Intake of medication directly affecting metabolic (e.g. metabolism of lipids (statine)) or hemodynamic state (e.g. beta-blockers, sildenafil) (other than angiotensin converting enzyme (ACE)-inhibitors and anticoagulation therapy)
- Cachectic disease
- Non-congestive hepatic or renal dysfunction
- (Inherited) metabolic disorders
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 |
|---|---|---|
| Metabolomics | Diagnostic test | Fontan patients, Healthy biventricular controls |
Primary outcome
Metabolism of lipids
Time frame: blood sampling at one single point of time (baseline/day 1) in the context of a planned patient presentation at the pediatric cardiology outpatient clinic (without assessment of changes)
Measurement of the serum concentration of the extended metabolism of lipids using the technique of Metabolomics (AbsoluteIDQ p180 kit, Biocrates Life Sciences AG, Innsbruck, Austria)
Metabolism of amino acids and related compounds
Time frame: blood sampling at one single point of time (baseline/day 1) in the context of a planned patient presentation at the pediatric cardiology outpatient clinic (without assessment of changes)
Measurement of the serum concentration of the extended metabolism of lamino acids using the technique of Metabolomics (AbsoluteIDQ p180 kit, Biocrates Life Sciences AG, Innsbruck, Austria)
Metabolism of carbohydrates
Time frame: blood sampling at one single point of time (baseline/day 1) in the context of a planned patient presentation at the pediatric cardiology outpatient clinic (without assessment of changes)
Measurement of the serum concentration of the extended metabolism of carbohydrates using the technique of Metabolomics (AbsoluteIDQ p180 kit, Biocrates Life Sciences AG, Innsbruck, Austria)
Metabolism of ketone bodies
Time frame: blood sampling at one single point of time (baseline/day 1) in the context of a planned patient presentation at the pediatric cardiology outpatient clinic (without assessment of changes)
Measurement of the serum concentration of ketone bodies using the technique of Metabolomics (AbsoluteIDQ p180 kit, Biocrates Life Sciences AG, Innsbruck, Austria)
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 24 Feb 2021.
- Derived from this study: PubMed 33319318Michel M, Salvador C, Wiedemair V, Adam MG, Laser KT, Dubowy KO, Entenmann A, Karall D, Geiger R, Zlamy M, Scholl-Burgi S. Method comparison of HPLC-ninhydrin-photometry and UHPLC-PITC-tandem mass spectrometry for serum amino acid analyses in patients with complex congenital heart disease and controls. Metabolomics. 2020 Dec 15;16(12):128. doi: 10.1007/s11306-020-01741-8.
- Derived from this study: PubMed 32426103Michel M, Dubowy KO, Zlamy M, Karall D, Adam MG, Entenmann A, Keller MA, Koch J, Odri Komazec I, Geiger R, Salvador C, Niederwanger C, Muller U, Scholl-Burgi S, Laser KT. Targeted metabolomic analysis of serum phospholipid and acylcarnitine in the adult Fontan patient with a dominant left ventricle. Ther Adv Chronic Dis. 2020 Apr 27;11:2040622320916031. doi: 10.1177/2040622320916031. eCollection 2020.
- Individual participant dataSharing planned: No. Participant data will be available only to the head of the study (Dr. Miriam Michel) (Excel- and SPSS-files)
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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.
Daniela Karall, Prof. principal investigator
Medical University Innsbruck
Questions about this trial
What is this clinical trial studying?
An observational study of Congenital Heart Disease and Metabolism Disorder, run by Medical University Innsbruck.
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 4 inclusion and 4 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 24 Feb 2021. 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?
Metabolism of lipids (time frame: blood sampling at one single point of time (baseline/day 1) in the context of a planned patient presentation at the pediatric cardiology outpatient clinic (without assessment of changes)). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03886935, which also lists 2 linked publications.
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 24 Feb 2021.