Longitudinal Observational Study on the Course of Cystic Fibrosis Lung Disease in Patients Following Newborn Screening (TRACK-CF)

  • STATUS
    Recruiting
  • End date
    Dec 3, 2030
  • participants needed
    200
  • sponsor
    Heidelberg University
Updated on 3 June 2022

Summary

The purpose of this study is to further characterize early CF lung disease in newborns, infants and toddlers with cystic fibrosis (CF).

Description

Cystic fibrosis (CF) is the most common lethal genetic multisystem disease in Germany. Although life expectancy increased over the last decades, most of the CF patients die in young adulthood due to chronic CF lung disease with respiratory failure. CF lung disease is caused by a disturbed transport of salt and water by airway epithelia and dehydration of airway surfaces as a result of the underlying genetic defect in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gen. Up to now, no causal therapies for the majority of patients with CF are available. Little is known about onset and natural course as well as influencing factors of CF lung disease. Therefore, the first aim of this prospective, multicenter, uncontrolled, non-randomized, explorative longitudinal study is characterization of the onset and early course of CF lung disease. For this reason we will primarily include patients diagnosed by CF newborn screening (CF-NBS) or for any other reason in the first four months of life (early diagnosed, ED). In a second step we will compare data from these patients to those diagnosed clinically later in life (late diagnosed, LD). This will allow us to investigate the effect of early diagnosis and start of therapy. Starting at diagnosis, we will use data from annual routine check-ups (imaging like chest MRI, pulmonary function tests, microbiology from swabs and sputum, laboratory values, anthropometry) as well as data from a facultative, study-related bronchoscopy with lavage (microbiology, inflammation and immunology) for correlation with the course of CF lung disease (generation of hypotheses). Further study-related investigations are monthly telephone interviews on bronchopulmonary symptoms by a study nurse on the basis of a questionnaire and quarterly assessment of health-related quality of life on the basis of a validated questionnaire.

We expect to gain a deeper insight into onset and early course of CF lung disease from the results of this study. So far, there is no trial that investigated the different aspects of CF lung disease (function, morphology, infectiology, inflammation) complementary in a longitudinal setting. We assume that knowledge on the natural history of CF lung disease in the vulnerable phase of early childhood has a great impact on the future development of new therapies (from symptomatic to causal). This shall lead to a further improvement in life expectancy and quality of life of patients with CF.

Details
Condition Cystic Fibrosis Lung Disease
Clinical Study IdentifierNCT02270476
SponsorHeidelberg University
Last Modified on3 June 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Newly diagnosed patients with Cystic Fibrosis (CF). Diagnosis of CF: at least one of the following three international accepted criteria is fulfilled: i) sweat chloride ≥ 60mEq/L and/or ii) 2 CF-causing mutations in the CFTR gene and/or iii) changes typical for CF in the transepithelial potential difference in nasal or rectal epithelium
Age and mode of diagnosis
Early diagnosed (ED): Initial diagnosis following CF-NBS or for other reasons in the first 4 months of life (in preterms corrected age of 4 months) after January 1st, 2006. Other reasons could be prenatal diagnostics, meconium ileus or positive family history
Late diagnosed (LD): Diagnosed after the fourth month of life due to clinical symptoms; initial diagnosis after January 1st, 2006

Exclusion Criteria

All patients are excluded who themselves or whose parents do not want to participate or
that withdraw from the study; or those in whom the diagnosis of CF is unsure
Further exclusion criteria are
Preterms <30th week of gestation
Longer period of mechanical ventilation in first 3 months of life
A significant medical disease or condition other than CF likely to interfere with the
child's ability to complete the entire protocol
Previous major surgery except for meconium ileus or atresia of the intestine
Other major organ dysfunction, excluding pancreatic or hepatic dysfunction or another
condition due to CF
Physical findings that would compromise the safety of the subject or the quality of
Chronic lung disease other than CF (e.g. bronchopulmonary dysplasia)
the study data as determined by investigator
History of adverse reaction to medication for sedation or known claustrophobia
Criteria, which lead to a displacement of the procedures in sedation until the child has
recovered: - Clinically significant upper airway obstruction as determined by investigator
(e.g
severe laryngomalacia, markedly enlarged tonsils, significant snoring, diagnosed
obstructive sleep apnoea)
Severe gastroesophageal reflux, defined as persistent frequent emesis despite anti-reflux
therapy
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