A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN)
What is the goal of the study?
This two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on endocrine growth factors and height, gastrointestinal function and gut microbiome, lung function and respiratory microbiome, liver and pancreatic function, sweat chloride, inflammatory markers, and bone health will be conducted in infants and young children with cystic fibrosis. Total duration of the study is expected to be 6 years. Part A will be a prospective cross-sequential study to describe the natural history of hormonal growth factors in early childhood and assess the feasibility of additional measurements. In Part A, consented participants will have up to 6 visits over a period of up to 3 years with collection, analysis and storage of biosamples such as blood, stool, urine, respiratory cultures and sweat. Research and clinical diagnostic testing data, clinical data and parent reported outcome measures will be collected at protocol specified time points. It is anticipated that 90 patients who are less than 5 years of age will enroll in Part A nationally with about 3 - 5 participants at Seattle Children's. Seattle Children's will be a site for the continuous glucose monitoring sub-study with separate consent obtained for participation. Part B will be a prospective longitudinal study to observe the effects of administration of FDA approved CFTR modulators, either ivacaftor or elexacaftor/tezacaftor/ivacaftor (elex/tez/iva) on growth. In Part B, consented participants will have one ?before ivacaftor or elex/tez/iva? visit within 30 days before initiation of the therapy and five ?after ivacaftor or elex/tez/iva? visits over a 24-month follow-up period. Specimen and data collection will be similar to Part A of the study. When possible, study visits and procedures will occur in conjunction with routine CF Clinic visits which are recommended quarterly in accordance with CF Foundation guidelines.Up to 120 patients who are under 6 years of age will be enrolled nationally in Part B with approximately 8 enrolled at Seattle Children's. Seattle Children's will be a site for the continuous glucose monitoring sub-study with separate consent obtained for participation.
Who can participate in the study?
Please contact the study team listed below to learn more.