NCT06063785

Brief 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. 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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
30

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Oct 2023

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 5, 2023

Completed
26 days until next milestone

Study Start

First participant enrolled

October 1, 2023

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 3, 2023

Completed
12 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 30, 2024

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2025

Completed
Last Updated

October 23, 2023

Status Verified

October 1, 2023

Enrollment Period

1 year

First QC Date

September 5, 2023

Last Update Submit

October 19, 2023

Conditions

Keywords

Cystic FibrosisMSOTLiver FibrosisGastrointestinal Transit

Outcome Measures

Primary Outcomes (2)

  • Quantitative collagen signal (Liver)

    in arbitrary units

    Day 1

  • Quantitative Indocyanine Green (ICG) signal (Intestinal)

    Day 1

    0, 60, 90, 120, 180, 240, 300min post ICG intake

Secondary Outcomes (13)

  • Quantitative oxy/deoxygenated hemoglobin signal (Liver)

    Day 1

  • Quantitative oxy/deoxygenated hemoglobin signal (Intestinal)

    0, 60, 90, 120, 180, 240, 300min post ICG intake

  • Quantitative single wave lengths (Intestinal)

    0, 60, 90, 120, 180, 240, 300min post ICG intake

  • Quantitative single wave lengths (Liver)

    Day 1

  • Optoacoustic spectrum (Intestinal)

    0, 60, 90, 120, 180, 240, 300min post ICG intake

  • +8 more secondary outcomes

Study Arms (3)

CF with liver affection

CF patients with proven CF hepatopathy

Diagnostic Test: Acoustic Radiation Forced Impulse ImagingDiagnostic Test: Multispectral Optoacoustic Tomography

CF without liver affection

CF patients without proven CF hepatopathy

Diagnostic Test: Acoustic Radiation Forced Impulse ImagingDiagnostic Test: Multispectral Optoacoustic Tomography

Healthy volunteers

Healthy volunteers without liver affection

Diagnostic Test: Acoustic Radiation Forced Impulse ImagingDiagnostic Test: Multispectral Optoacoustic Tomography

Interventions

Measurement of Liver stiffness

Also known as: ARFI
CF with liver affectionCF without liver affectionHealthy volunteers

Measurement of optoacoustic spectra in liver and gastrointestinal tract

Also known as: MSOT
CF with liver affectionCF without liver affectionHealthy volunteers

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
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.

You may qualify if:

  • 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
  • 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

You may not qualify if:

  • 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":
  • 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
  • +4 more criteria

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University Hospital Erlange, Department of Pediatrics

Erlangen, Bavaria, 91054, Germany

RECRUITING

Biospecimen

Retention: SAMPLES WITHOUT DNA

Serum samples for bile acid profiling Stool samples for bile acid profiling

MeSH Terms

Conditions

Cystic FibrosisLiver Cirrhosis

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesLiver DiseasesFibrosisPathologic ProcessesPathological Conditions, Signs and Symptoms

Study Officials

  • Alexander Schnell

    Department of Pediatric and Adolescent Medicine, University Hospital Erlangen

    PRINCIPAL INVESTIGATOR
  • Adrian P Regensburger

    Department of Pediatric and Adolescent Medicine, University Hospital Erlangen

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

September 5, 2023

First Posted

October 3, 2023

Study Start

October 1, 2023

Primary Completion

September 30, 2024

Study Completion

March 31, 2025

Last Updated

October 23, 2023

Record last verified: 2023-10

Data Sharing

IPD Sharing
Will not share

Locations