Status:
RECRUITING
Longitudinal Observational Study on the Course of Cystic Fibrosis Lung Disease in Patients Following Newborn Screening
Lead Sponsor:
Heidelberg University
Collaborating Sponsors:
German Center for Lung Research
Conditions:
Cystic Fibrosis Lung Disease
Eligibility:
All Genders
Brief Summary
The purpose of this study is to further characterize early CF lung disease in newborns, infants and toddlers with cystic fibrosis (CF).
Detailed 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 c...
Eligibility Criteria
Inclusion
- 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
- 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 the study data as determined by investigator
- Chronic lung disease other than CF (e.g. bronchopulmonary dysplasia)
- 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
Key Trial Info
Start Date :
December 1 2011
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
December 1 2030
Estimated Enrollment :
200 Patients enrolled
Trial Details
Trial ID
NCT02270476
Start Date
December 1 2011
End Date
December 1 2030
Last Update
November 29 2023
Active Locations (4)
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1
University Children's Hospital Heidelberg, Cystic Fibrosis Centre
Heidelberg, Baden-Wurttemberg, Germany, 69120
2
University Hospital Gießen and Marburg GmbH
Giessen, Hesse, Germany, 35392
3
Medizinische Hochschule Hannover
Hanover, Lower Saxony, Germany, 30625
4
University Children's Hospital Schleswig-Holstein
Lübeck, Schleswig-Holstein, Germany, 23538