Cystic FibrosisNCT06063785
Multispectral Optoacoustic Tomography in Patients With Cystic Fibrosis
An observational study of Cystic Fibrosis and Liver Fibrosis, run by University of Erlangen-Nürnberg Medical School at 1 site in Germany.
Trial summary
Cystic fibrosis (CF) is the most common hereditary disease in Central Europe. The disease is caused by a mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR). In the liver, fibrotic remodeling can lead to liver cirrhosis in the long term. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Cystic Fibrosis, Liver Fibrosis, Gastrointestinal Transport Disorder
- What is being tested?
- Acoustic Radiation Forced Impulse Imaging and Multispectral Optoacoustic Tomography
- How many people
- 30 participants
- Where
- 1 siteGermany
- Study dates
- 1 Oct 2023to 31 Mar 2025
- 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 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: It is planned to study a total of 10 patients each with cystic fibrosis with/without CF-related liver disease and 10 healthy volunteers.
Who may be able to join
- Patient cohort "Cystic Fibrosis without CF-related liver disease":
- Molecular genetic confirmed diagnosis of cystic fibrosis.
- Age over 18 years
- Written informed consent
- Patient cohort "Cystic Fibrosis with CF-related liver disease":
- Molecular genetic confirmed diagnosis of cystic fibrosis
Show all 15Show fewer
- Presence of CF-related liver disease based on Colombo criteria:
- Hepato- and/or splenomegaly
- Persistent elevation of transaminases in the serum
- Sonographic evidence of liver involvement
- Age over 18 years
- Written informed consent
- "Volunteer Subjects":
- Age over 18 years
- Written informed consent
Who cannot join
- General:
- Pregnancy
- Breastfeeding mothers
- Tattoo in the area of the examination
- Subcutaneous fat tissue over 3 cm
- Patient cohort "Cystic fibrosis without CF-related liver disease":
Show all 19Show fewer
- Taking systemic glucocorticoids or immunosuppressants as part of a permanent medication regimen.
- Presence of CF-related liver disease based on Colombo criteria:
- Hepato- and/or splenomegaly.
- Persistent elevation of transaminases in the serum
- Sonographic evidence of liver involvement.
- Acute exacerbation of infection
- Patient cohort "Cystic fibrosis with CF-related liver disease":
- Taking systemic glucocorticoids or immunosuppressants as part of a permanent medication regimen.
- Decompensation of CF-related liver disease
- Acute exacerbation of infection
- "volunteer subjects":
- Presence of liver disease
- Use of systemic glucocorticoids or immunosuppressants in the context of permanent medication
Trial locations and contacts
1 site in 1 country.
Germany1 site, 1 recruiting
- Recruiting
University Hospital Erlange, Department of Pediatrics
Erlangen, Bavaria, 91054
Alexander Schnell, Dr. med., +4991318533118, alexander.schnell@uk-erlangen.de
Adrian P Regensburger, PD Dr. med., +4991318533118, adrian.regensburger@uk-erlangen.de
About this study
Official title: Multispectral Optoacoustic Tomography for the Assessment of Liver Fibrosis and Gastrointestinal Transit in Patients With Cystic Fibrosis
Cystic fibrosis (CF) is the most common hereditary disease in Central Europe. The disease is caused by a mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR). In the liver, fibrotic remodeling can lead to liver cirrhosis in the long term. Early detection of CF hepatopathy is essential to therapeutically slow down the progression of fibrotic remodeling mechanisms. Newborns suffering from CF have a significantly increased risk for the occurrence of meconium ileus and also with advancing age there are symptoms ranging from chronic constipation to Distal Intestinal Obstruction Syndrome (DIOS), due to a reduction of intestinal motility.
In this study, the degree of liver fibrosis will now be investigated in adult patients with cystic fibrosis using Multispectral Optoacoustic Imaging (MSOT). In addition, gastrointestinal passage will be studied non-invasively to investigate another affection of the gastrointestinal system.
Read the detailed description
Cystic fibrosis (CF) is the most common hereditary disease in Central Europe, with an incidence of approximately 3,300 to 4,800 new cases. The disease follows an autosomal recessive pattern of inheritance, the cause being a mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR). In the liver, fibrotic remodeling can lead to liver cirrhosis in the long term. Early detection of CF hepatopathy is essential to therapeutically slow down the progression of fibrotic remodeling mechanisms. Over the past decade, measurements of liver stiffness using Acoustic Radiation Force Impulse Imaging (ARFI) have proven to be a valid tool for measuring fibrotic tissue remodeling in CF in adults and children. Furthermore, in the gastrointestinal tract, serious consequences result from the absence of the CFTR channel. Newborns suffering from CF have a significantly increased risk for the occurrence of meconium ileus and also with advancing age there are symptoms ranging from chronic constipation to DIOS (Distal Intestinal Obstruction Syndrome), due to a reduction of intestinal motility.
By means of new imaging methods, such as multispectral optoacoustic tomography, it is possible to examine not only the body's own substances but also substances foreign to the body. With Multispectral Optoacoustic Imaging (MSOT), similar to conventional sonography, a transducer is placed on the skin and instead of sound, energy is applied to the tissue by means of light flashes. This leads to a constant alternation of minimal expansions and contractions (thermoelastic expansion) of individual tissue components or molecules. Previous studies have shown that quantitative determination of hemoglobin can provide information on blood flow and inflammatory activity in the intestines of adult patients with Crohn's disease. Also, fibrotic changes in the liver can probably be detected with this method, similar to that in muscle tissue. Furthermore, we have recently shown that orally ingested Indocyanine green (ICG) can be detected in the small intestine and thus conclusions can be drawn about gastrointestinal passage, without the use of ionizing radiation. In this study, the degree of liver fibrosis will now be investigated in adult patients with cystic fibrosis using MSOT. In addition, gastrointestinal passage will be studied non-invasively to investigate another affection of the gastrointestinal system.
- Being tested
- Acoustic Radiation Forced Impulse Imaging, Multispectral Optoacoustic Tomography
- Keywords
- Cystic Fibrosis, MSOT, Liver Fibrosis, Gastrointestinal Transit
- Medical subject headings
- Cystic Fibrosis, Liver Cirrhosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Acoustic Radiation Forced Impulse ImagingDiagnostic test
Also called ARFI
Measurement of Liver stiffness
Multispectral Optoacoustic TomographyDiagnostic test
Also called MSOT
Measurement of optoacoustic spectra in liver and gastrointestinal tract
| Study group | Role | What they receive |
|---|---|---|
| CF with liver affection | CF patients with proven CF hepatopathy Receives: Diagnostic Test: Acoustic Radiation Forced Impulse Imaging; Diagnostic Test: Multispectral Optoacoustic Tomography | |
| CF without liver affection | CF patients without proven CF hepatopathy Receives: Diagnostic Test: Acoustic Radiation Forced Impulse Imaging; Diagnostic Test: Multispectral Optoacoustic Tomography | |
| Healthy volunteers | Healthy volunteers without liver affection Receives: Diagnostic Test: Acoustic Radiation Forced Impulse Imaging; Diagnostic Test: Multispectral Optoacoustic Tomography |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quantitative collagen signal (Liver)
in arbitrary units
Measured: Day 1
Quantitative Indocyanine Green (ICG) signal (Intestinal)
Day 1
Measured: 0, 60, 90, 120, 180, 240, 300min post ICG intake
Secondary outcomes (13)
Quantitative oxy/deoxygenated hemoglobin signal (Liver)
in arbitrary units
Measured: Day 1
Quantitative oxy/deoxygenated hemoglobin signal (Intestinal)
in arbitrary units
Measured: 0, 60, 90, 120, 180, 240, 300min post ICG intake
Quantitative single wave lengths (Intestinal)
in arbitrary units
Measured: 0, 60, 90, 120, 180, 240, 300min post ICG intake
Quantitative single wave lengths (Liver)
in arbitrary units
Measured: Day 1
Optoacoustic spectrum (Intestinal)
in arbitrary units, normalized
Measured: 0, 60, 90, 120, 180, 240, 300min post ICG intake
Optoacoustic spectrum (Liver)
in arbitrary units, normalized
Measured: Day 1
Shear wave velocity (Liver)
in m/s
Measured: Day 1
Attenuation coefficient (Liver)
in dB/cm/MHz
Measured: Day 1
Chenodeoxycholic acid (CDCA) and respective glycine and taurine conjugates
µmol/l and /g stool
Measured: Day 1
Cholic acid (CA) and respective glycine and taurine conjugates
µmol/l and /g stool
Measured: Day 1
Deoxycholic acid (DCA) and respective glycine and taurine conjugates
µmol/l and /g stool
Measured: Day 1
Lithocholic acid (LCA) and respective glycine and taurine conjugates
µmol/l and /g stool
Measured: Day 1
Ursodeoxycholic acid (UDCA) and respective glycine and taurine conjugates
µmol/l and /g stool
Measured: Day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Oct 2023
- Study start1 Oct 2023
- Primary completion30 Sep 2024 (estimated)
- Study completion31 Mar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University of Erlangen-Nürnberg Medical SchoolThe 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Erlangen-Nürnberg Medical School
- Sponsor type
- University, hospital or other
- Collaborators
- Adrian Regensburger
- Responsible party
- Principal investigator, Alexander Schnell, University of Erlangen-Nürnberg Medical School
- Investigators
- Alexander Schnell, Principal investigator, Department of Pediatric and Adolescent Medicine, University Hospital ErlangenAdrian P Regensburger, Principal investigator, Department of Pediatric and Adolescent Medicine, University Hospital Erlangen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06063785
- Sponsor's protocol ID
- CF_MSOT
- Status last verified
- Oct 2023
- First submitted
- 5 Sep 2023
- Last updated
- 23 Oct 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multispectral Optoacoustic Tomography for the Assessment of Liver Fibrosis and Gastrointestinal Transit in Patients With Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT06063785
- Acronym / sponsor ID
- CF_MSOT
- Keywords
- Cystic Fibrosis, MSOT, Liver Fibrosis, Gastrointestinal Transit
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University of Erlangen-Nürnberg Medical SchoolUniversity, hospital or other
- Collaborators
- Adrian Regensburger
- Study start
- 1 Oct 2023 (actual)
- Primary completion
- 30 Sep 2024 (estimated)
- Study completion
- 31 Mar 2025 (estimated)
- First posted
- 3 Oct 2023 (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.
Cystic fibrosis (CF) is the most common hereditary disease in Central Europe. The disease is caused by a mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR). In the liver, fibrotic remodeling can lead to liver cirrhosis in the long term. Early detection of CF hepatopathy is essential to therapeutically slow down the progression of fibrotic remodeling mechanisms. Newborns suffering from CF have a significantly increased risk for the occurrence of meconium ileus and also with advancing age there are symptoms ranging from chronic constipation to Distal Intestinal Obstruction Syndrome (DIOS), due to a reduction of intestinal motility.
In this study, the degree of liver fibrosis will now be investigated in adult patients with cystic fibrosis using Multispectral Optoacoustic Imaging (MSOT). In addition, gastrointestinal passage will be studied non-invasively to investigate another affection of the gastrointestinal system.
Detailed description
Cystic fibrosis (CF) is the most common hereditary disease in Central Europe, with an incidence of approximately 3,300 to 4,800 new cases. The disease follows an autosomal recessive pattern of inheritance, the cause being a mutation in the cystic fibrosis transmembrane conductance regulator gene (CFTR). In the liver, fibrotic remodeling can lead to liver cirrhosis in the long term. Early detection of CF hepatopathy is essential to therapeutically slow down the progression of fibrotic remodeling mechanisms. Over the past decade, measurements of liver stiffness using Acoustic Radiation Force Impulse Imaging (ARFI) have proven to be a valid tool for measuring fibrotic tissue remodeling in CF in adults and children. Furthermore, in the gastrointestinal tract, serious consequences result from the absence of the CFTR channel. Newborns suffering from CF have a significantly increased risk for the occurrence of meconium ileus and also with advancing age there are symptoms ranging from chronic constipation to DIOS (Distal Intestinal Obstruction Syndrome), due to a reduction of intestinal motility.
By means of new imaging methods, such as multispectral optoacoustic tomography, it is possible to examine not only the body's own substances but also substances foreign to the body. With Multispectral Optoacoustic Imaging (MSOT), similar to conventional sonography, a transducer is placed on the skin and instead of sound, energy is applied to the tissue by means of light flashes. This leads to a constant alternation of minimal expansions and contractions (thermoelastic expansion) of individual tissue components or molecules. Previous studies have shown that quantitative determination of hemoglobin can provide information on blood flow and inflammatory activity in the intestines of adult patients with Crohn's disease. Also, fibrotic changes in the liver can probably be detected with this method, similar to that in muscle tissue. Furthermore, we have recently shown that orally ingested Indocyanine green (ICG) can be detected in the small intestine and thus conclusions can be drawn about gastrointestinal passage, without the use of ionizing radiation. In this study, the degree of liver fibrosis will now be investigated in adult patients with cystic fibrosis using MSOT. In addition, gastrointestinal passage will be studied non-invasively to investigate another affection of the gastrointestinal system.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| CF with liver affection | – | Diagnostic Test: Acoustic Radiation Forced Impulse Imaging, Diagnostic Test: Multispectral Optoacoustic TomographyCF patients with proven CF hepatopathy |
| CF without liver affection | – | Diagnostic Test: Acoustic Radiation Forced Impulse Imaging, Diagnostic Test: Multispectral Optoacoustic TomographyCF patients without proven CF hepatopathy |
| Healthy volunteers | – | Diagnostic Test: Acoustic Radiation Forced Impulse Imaging, Diagnostic Test: Multispectral Optoacoustic TomographyHealthy volunteers without liver affection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- It is planned to study a total of 10 patients each with cystic fibrosis with/without CF-related liver disease and 10 healthy volunteers.
Key inclusion criteria (15 listed)
- Molecular genetic confirmed diagnosis of cystic fibrosis.
- Age over 18 years
- Written informed consent
- Molecular genetic confirmed diagnosis of cystic fibrosis
- Presence of CF-related liver disease based on Colombo criteria:
Key exclusion criteria (19 listed)
- Pregnancy
- Breastfeeding mothers
- Tattoo in the area of the examination
- Subcutaneous fat tissue over 3 cm
- Taking systemic glucocorticoids or immunosuppressants as part of a permanent medication regimen.
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 |
|---|---|---|
| Acoustic Radiation Forced Impulse ImagingAlso: ARFIMeasurement of Liver stiffness | Diagnostic test | CF with liver affection, CF without liver affection, Healthy volunteers |
| Multispectral Optoacoustic TomographyAlso: MSOTMeasurement of optoacoustic spectra in liver and gastrointestinal tract | Diagnostic test | CF with liver affection, CF without liver affection, Healthy volunteers |
Primary outcome
Quantitative collagen signal (Liver)
Time frame: Day 1
in arbitrary units
Quantitative Indocyanine Green (ICG) signal (Intestinal)
Time frame: 0, 60, 90, 120, 180, 240, 300min post ICG intake
Day 1
13 secondary outcomes
Quantitative oxy/deoxygenated hemoglobin signal (Liver)
Time frame: Day 1
Quantitative oxy/deoxygenated hemoglobin signal (Intestinal)
Time frame: 0, 60, 90, 120, 180, 240, 300min post ICG intake
Quantitative single wave lengths (Intestinal)
Time frame: 0, 60, 90, 120, 180, 240, 300min post ICG intake
Quantitative single wave lengths (Liver)
Time frame: Day 1
Optoacoustic spectrum (Intestinal)
Time frame: 0, 60, 90, 120, 180, 240, 300min post ICG intake
Optoacoustic spectrum (Liver)
Time frame: Day 1
Shear wave velocity (Liver)
Time frame: Day 1
Attenuation coefficient (Liver)
Time frame: Day 1
Chenodeoxycholic acid (CDCA) and respective glycine and taurine conjugates
Time frame: Day 1
Cholic acid (CA) and respective glycine and taurine conjugates
Time frame: Day 1
Deoxycholic acid (DCA) and respective glycine and taurine conjugates
Time frame: Day 1
Lithocholic acid (LCA) and respective glycine and taurine conjugates
Time frame: Day 1
Ursodeoxycholic acid (UDCA) and respective glycine and taurine conjugates
Time frame: Day 1
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Oct 2023.
- Individual participant dataSharing planned: No
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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.
Alexander Schnell principal investigator
Department of Pediatric and Adolescent Medicine, University Hospital Erlangen
Adrian P Regensburger principal investigator
Department of Pediatric and Adolescent Medicine, University Hospital Erlangen
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis and Liver Fibrosis, run by University of Erlangen-Nürnberg Medical School at 1 site in Germany.
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 15 inclusion and 19 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: status not confirmed, as of the sponsor's last update on 23 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Alexander Schnell (+4991318533118, alexander.schnell@uk-erlangen.de), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Quantitative collagen signal (Liver) (time frame: Day 1). The study lists 2 primary outcomes in all. 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 23 Oct 2023.