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Comparison of Non-invasive Measurement Methods of Hepatic Fibrosis in Cystic Fibrosis (MUCO-FIBRO)

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ClinicalTrials.gov Identifier: NCT02342964
Recruitment Status : Recruiting
First Posted : January 21, 2015
Last Update Posted : December 21, 2017
Sponsor:
Collaborator:
Vaincre la Mucoviscidose
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Brief Summary:

Non-invasive methods of the quantification of fibrosis may help to assess the development of fibrosis at a specific moment of the evolution of the disease, in the order to decide the initiation of treatment with ursodeoxycholic acid which may slow the progression to hepatic cirrhosis:

  • FibroTest,
  • The ultrasound impulse elastography, Fibroscan (FS)
  • Ultrasound Elastography by ShearWave (SWE)
  • Magnetic resonance elastography (MRE). The purpose of this study is to assess the contribution of these methods in the diagnosis of hepatic fibrosis during the evolution of the cystic fibrosis.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Other: Measure of hepatic elasticity Not Applicable

Detailed Description:

The incidence of hepatobiliary disease in cystic fibrosis in the first 10 years of life is 2.5 to 1.8 / 100 patient-years, significantly decreasing during the second decade; generally 30-40% of children develop hepatic disease before 12 years. However, the hepatobiliary damage varies in its clinical and histological expression. The typical histological appearance of the liver is the focal biliary cirrhosis (or fibrosis) characterized by a heterogeneous distribution of portal fibrosis in the liver.

Complications of cirrhosis are responsible for 2.5% of deaths in patients with cystic fibrosis and represent the second cause of death after lung disease if we exclude deaths secondary to lung transplantation.

The diagnosis of hepatic disease at an early stage of development and the assessment of the progression of fibrosis are so difficult in the absence of sensitive and specific non-invasive diagnostic tests.

Non-invasive assessment of hepatic fibrosis is an important topic of study in recent years, because of the emergence of biological methods and quantitative elastography methods using ultrasound or Magnetic Resonance Imagery (MRI) and allowing quantification of the hepatic elasticity, which is assumed to be a reflection of fibrosis. The advantages of these methods are numerous:

  • The assessment of wider hepatic parenchyma then hepatic biopsy, allowing overcome the sampling bias
  • Painless and non-invasive methods for the patient
  • Simple to make
  • Do not require hospitalization and can be performed as outpatient
  • Immediately known results
  • Can be repeated as often as necessary during the patient's follow-up
  • Lower cost, morbidity and mortality compared to hepatic biopsy. Several non-invasive methods of fibrosis diagnosis have been studied in adults primarily for viral diseases and helped to limit the indications of hepatic biopsy. However, no method is now validated for the early diagnosis of hepatic disease and monitoring of children with cystic fibrosis.

The main objective of the study is to assess the contribution of FibroTest, the ultrasound impulse elastography, Fibroscan (FS), the ultrasound elastography by ShearWave (SWE) and Magnetic Resonance Elastography (MRE ) in the diagnosis of hepatic fibrosis in cystic fibrosis.

The secondary objectives are following :

  • To study the correlations between the fibrosis quantitative results obtained through the four techniques of measurement and the matches between the results of four techniques using the standards available at t0 and after a year (t1).
  • Study the correlations between clinical, biological and ultrasound endpoints of hepatic disease and various non-invasive measurement methods of fibrosis.
  • Evaluate the area under the ROC curve of the different tests and the diagnostic performance tests using analytical methods without gold standard by classifying the children into two groups: the presence of advanced fibrosis (stages F2 to F4 ) or not (F0 and F1 stages), contracting by a committee according to the results of any biopsy, clinical, laboratory and ultrasound endpoints independent on the tests.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Screening
Official Title: Comparative Study of Different Non-invasive Measurement Methods of Hepatic Fibrosis in Cystic Fibrosis
Actual Study Start Date : January 8, 2015
Estimated Primary Completion Date : January 2018
Estimated Study Completion Date : June 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
MUCO-FIBRO
Measures of hepatic elasticity
Other: Measure of hepatic elasticity

Measure of hepatic elasticity by four non-invasive methods:

Fibroscan, Fibrotest, SWE, MRE.





Primary Outcome Measures :
  1. Measure of hepatic elasticity by Fibro Test [ Time Frame: Day 0 ]
    The value of hepatic elasticity measured by each of the tests performed on the same day in the same patient

  2. Measure of hepatic elasticity by the ultrasound impulse elastography, Fibroscan (FS) [ Time Frame: Month 12 ]
    The value of hepatic elasticity measured by each of the tests performed on the same day in the same patient.


Secondary Outcome Measures :
  1. Measure of hepatic elasticity by Ultrasound Elastography by ShearWave (SWE): [ Time Frame: Day 0 ]
    The existence of advanced hepatic fibrosis (stages F2 to F4) will be defined by the presence of clinical, and / or biological and / or ultrasound abnormalities, defining two groups of children (F + :children with advanced fibrosis, F-: children with little or no fibrosis

  2. Measure of hepatic elasticity by Magnetic resonance elastography (MRE): [ Time Frame: Month 12 ]
    The existence of advanced hepatic fibrosis (stages F2 to F4) will be defined by the presence of clinical, and / or biological and / or ultrasound abnormalities, defining two groups of children (F + :children with advanced fibrosis, F-: children with little or no fibrosis



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Ages Eligible for Study:   6 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Children with cystic fibrosis aged 6-18 years. The diagnosis of cystic fibrosis will be based on testing the abnormal sweat (> 60 mEq / L of chloride sweat) and / or on the presence of two pathogen mutations of CFTR.
  • Written consent of parents or holders of parental rights.
  • Affiliated or benefiting from a national social insurance

Exclusion Criteria:

  • Presence of another disease associated with cystic fibrosis that may be responsible for a chronic hepatic disease such as deficiency of alpha-1antitrypsine ZZ genotype or chronic infection by virus B or C.
  • Child grafted
  • Actual febrile pulmonary infection that can change the value of Fibrotest
  • Important peri-hepatic ascites preventing the elasticity measurements by Fibroscan®
  • Severe respiratory insufficient preventing attainment of Magnetic Resonance Imagery

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02342964


Contacts
Contact: Dominique Debray, MD 00 33 1 44 49 41 52 dominique.debray@gmail.com
Contact: Laurence Lecomte, PhD +33 1 71 19 64 94 laurence.lecomte@nck.aphp.fr

Locations
France
Hôpital Necker Recruiting
Paris, France, 75015
Contact: Dominique Debray, Md, PhD    00 33 1 44 49 41 52    dominique.debray@gmail.com   
Contact: Laurence Lecomte, PhD    +33 1 71 19 64 94    laurence.lecomte@nck.aphp.fr   
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Vaincre la Mucoviscidose
Investigators
Principal Investigator: Dominique Debray, MD, PhD 00 33 1 44 49 41 52

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT02342964     History of Changes
Other Study ID Numbers: RCB-2013-A01579-36
First Posted: January 21, 2015    Key Record Dates
Last Update Posted: December 21, 2017
Last Verified: December 2017

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Cystic fibrosis
hepatic fibrosis
hepatic cirrhosis
non-invasive methods of measurement
hepatic elasticity

Additional relevant MeSH terms:
Fibrosis
Cystic Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases