Cystic Fibrosis Lung Disease Clinical Trial
— TRACK-CFOfficial title:
Longitudinal Observational Study on the Course of Cystic Fibrosis Lung Disease in Patients Following Newborn Screening
NCT number | NCT02270476 |
Other study ID # | UKH-TRACK-1 |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | December 2011 |
Est. completion date | December 2030 |
The purpose of this study is to further characterize early CF lung disease in newborns, infants and toddlers with cystic fibrosis (CF).
Status | Recruiting |
Enrollment | 200 |
Est. completion date | December 2030 |
Est. primary completion date | December 2030 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | Inclusion Criteria: 1. 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. 2. 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: 1. Preterms <30th week of gestation 2. Longer period of mechanical ventilation in first 3 months of life 3. A significant medical disease or condition other than CF likely to interfere with the child's ability to complete the entire protocol 4. Previous major surgery except for meconium ileus or atresia of the intestine 5. Other major organ dysfunction, excluding pancreatic or hepatic dysfunction or another condition due to CF 6. Physical findings that would compromise the safety of the subject or the quality of the study data as determined by investigator 7. Chronic lung disease other than CF (e.g. bronchopulmonary dysplasia) 8. 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 |
Country | Name | City | State |
---|---|---|---|
Germany | University Hospital Gießen and Marburg GmbH | Gießen | Hessen |
Germany | Medizinische Hochschule Hannover | Hannover | Niedersachsen |
Germany | University Children's Hospital Heidelberg, Cystic Fibrosis Centre | Heidelberg | Baden-Württemberg |
Germany | University Children's Hospital Schleswig-Holstein | Lübeck | Schleswig-Holstein |
Lead Sponsor | Collaborator |
---|---|
Heidelberg University | German Center for Lung Research |
Germany,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Proportion with morphological and/or perfusion changes due to CF lung disease after chest MRI score in both groups | At age of 1, 2, 3, ...., 10 years of age | ||
Primary | Proportion of patients with impairments in pulmonary function tests (e.g. multiple breath washout (MBW)) in both groups | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Rate of protocol-defined pulmonary exacerbations in both groups (ED vs. LD) that are necessitating an antibiotic therapy orally, intravenously or per inhalation | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Spontaneous development of infection or spectrum of pathogens, respectively, in throat and nose swabs as well as other airway secretions from routine diagnostics and if applicable bronchoalveolar lavage fluid (BALF) | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | From the patients in whom PsA or other CF pathogens could not be isolated at the beginning of their participation, comparison of the portion of patients with a positive culture during participation in both groups (ED and LD) | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Time to first detection of a CF pathogen in both groups | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Time to first pulmonary exacerbation in both groups | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Portion of patients with increased biochemical inflammatory markers in both groups and magnitude of elevation | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Frequency of symptoms from monthly telephone interviews in both groups | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Health-related quality of life in both groups quarterly via Cystic Fibrosis Questionnaire (CFQ) | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Development of body weight, body height, ideal weight-for-height (IWFH), Body-Mass-Index (BMI), respiratory rate and oxygen saturation at room air in both groups | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Proportion with morphological changes due to CF lung disease after modified Chrispin-Norman Score for assessment of chest X-ray in both groups | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Magnitude and severity of alterations typical for CF by assessment with chest MRI and X-ray score in both groups | At age of 1, 2, 3, ...., 10 years of age | ||
Secondary | Magnitude of impairment of pulmonary function test in both groups | At age of 1, 2, 3, ...., 10 years of age |
Status | Clinical Trial | Phase | |
---|---|---|---|
Completed |
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