title: Novel end points for clinical trials in young children with cystic fibrosis.
abstract: Cystic fibrosis (CF) lung disease commences early in the disease progression and is the most common cause of mortality. While new CF disease-modifying agents are currently undergoing clinical trial evaluation, the implementation of such trials in young children is limited by the lack of age-appropriate clinical trial end points. Advances in infant and preschool lung function testing, imaging of the chest and the development of biochemical biomarkers have led to increased possibility of quantifying mild lung disease in young children with CF and objectively monitoring disease progression over the course of an intervention. Despite this, further standardization and development of these techniques is required to provide robust objective measures for clinical trials in this age group.
authors.affiliation: Telethon Institute for Child Health Research, Centre for Child Health Research, University of Western Australia, Perth, Australia.
authors.forename: Shannon J
authors.initials: SJ
authors.lastname: Simpson
authors.forename: Lauren S
authors.initials: LS
authors.lastname: Mott
authors.forename: Charles R
authors.initials: CR
authors.lastname: Esther
authors.forename: Stephen M
authors.initials: SM
authors.lastname: Stick
authors.forename: Graham L
authors.initials: GL
authors.lastname: Hall
mesh_terms: D000367:Age Factors; D015415:Biomarkers / Q000378:metabolism; D002648:Child; D002675:Child, Preschool; D002986:Clinical Trials as Topic / Q000379:methods*; D003550:Cystic Fibrosis / Q000175:diagnosis / Q000378:metabolism / Q000503:physiopathology / Q000628:therapy*; D003952:Diagnostic Imaging / Q000379:methods; D042241:Early Diagnosis; D023381:Endpoint Determination; D006801:Humans; D007223:Infant; D008168:Lung / Q000000981:diagnostic imaging / Q000378:metabolism / Q000473:pathology / Q000503:physiopathology; D008279:Magnetic Resonance Imaging; D011237:Predictive Value of Tests; D012107:Research Design; D012129:Respiratory Function Tests; D014057:Tomography, X-Ray Computed; D016896:Treatment Outcome
chemical_list: D015415:Biomarkers