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The purpose of the study is to evaluate the natural history of exocrine pancreatic function by assessing Fecal elastase-1 (FE-1) in infants with CF during their first year of life.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort A | Observational follow-up study to characterize the natural history of exocrine pancreatic function by measuring FE-1 in infants with CF. |
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| Measure | Description | Time Frame |
|---|---|---|
| Proportion of Participants with FE-1 greater than or equal to (≥) 200 microgram per gram (µg/g) Over Time | From Enrollment up to the Infant turning 12 Months of Age |
| Measure | Description | Time Frame |
|---|---|---|
| FE-1 level Over Time | From Enrollment up to the Infant turning 12 Months of Age |
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Key Inclusion Criteria:
Key Exclusion Criteria:
Other protocol defined Inclusion/Exclusion criteria apply.
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Infants with CF less than (<) 6 months of age at the index date who are not eligible for commercial Kalydeco™ (according to local product labels). The day of enrollment into this study will be referred to as the "index date".
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Johns Hopkins All Children's Hospital Outpatient Care Center | St. Petersburg | Florida | 33701 | United States | ||
Details on Vertex data sharing criteria and process for requesting access can be found at: https: https://www.vrtx.com/our-science/clinical-trials-data-sharing/
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| St. Luke's Cystic Fibrosis Center of Idaho |
| Boise |
| Idaho |
| 83702 |
| United States |
| Ann & Robert H. Lurie Children's Hospital of Chicago | Chicago | Illinois | 60611 | United States |
| Riley Hospital for Children at Indiana University Health | Indianapolis | Indiana | 46202 | United States |
| Boston Childrens Hospital | Boston | Massachusetts | 02115 | United States |
| Michigan Medicine | Ann Arbor | Michigan | 48109 | United States |
| Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota | Minneapolis | Minnesota | 55404 | United States |
| Billings Clinic | Billings | Montana | 59101 | United States |
| Children's Nebraska | Omaha | Nebraska | 68114 | United States |
| Duke University Medical Center | Durham | North Carolina | 27710 | United States |
| Akron Children's Hospital | Akron | Ohio | 44308 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center | Cleveland | Ohio | 44106 | United States |
| Dayton Children's Hospital | Dayton | Ohio | 45404 | United States |
| ProMedica Toledo Hospital/Toledo Children's Hospital/Pediatric Pulmonary & Cystic Fibrosis Center | Toledo | Ohio | 43606 | United States |
| Prisma Health Clinical Research Unit - Midlands | Columbia | South Carolina | 29203 | United States |
| Sanford Children's Speciality Clinic | Sioux Falls | South Dakota | 57105 | United States |
| The University of Texas Health Science Center at Tyler | Tyler | Texas | 75708 | United States |
| Virginia Commonwealth University Hospital Systems, Children's Pavilion | Richmond | Virginia | 23298 | United States |
| Providence Pediatric Pulmonary & Cystic Fibrosis Clinic | Spokane | Washington | 99204 | United States |
| West Virginia University | Morgantown | West Virginia | 26506 | United States |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | United States |
| The Kids Research Institute Australia | Nedlands | Australia |
| Women's and Children's Hospital | North Adelaide | Australia |
| Queensland Children's Hospital | South Brisbane | Australia |
| McGill University Health Centre, Glen Site, Montreal Children's Hospital | Montreal | Canada |
| Centre Hospitalier de Quebec - Universite Laval | Québec | Canada |
| The Hospital for Sick Children | Toronto | Canada |
| British Columbia Children's Hospital | Vancouver | Canada |
| Fakultni nemocnice v Motole | Prague | Czechia |
| Charite Paediatric Pulmonology Department | Berlin | Germany |
| Kinderklinik III, Abt. fur Pneumologie | Essen | Germany |
| Klinikum Westbrandenburg (CF) | Potsdam | Germany |
| Azienda Ospedaliero Universitaria Ospedale Pediatrico Meyer | Florence | Italy |
| Canterbury District Health Board | Christchurch | New Zealand |
| Starship Children's Hospital | Grafton | New Zealand |
| Pediatric Hospital Polanki named of Maciej Płażyński | Gdansk | Poland |
| Hospital Universitario 12 de Octubre | Madrid | Spain |
| Hospital Universitario Ramon y Cajal | Madrid | Spain |
| Hospital Universitario Virgen del Rocio | Seville | Spain |
| Kinderspital Zurich | Zurich | Switzerland |
| University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital for Children | Bristol | United Kingdom |
| Royal Hospital for Children | Glasgow | United Kingdom |
| Leeds Regional Children's Cystic Fibrosis Centre | Leeds | United Kingdom |
| Leicester Royal Infirmary, University of Leicester | Leicester | United Kingdom |
| Great Ormond Street Hospital for Children | London | United Kingdom |
| The Royal London Children's Hospital | London | United Kingdom |
| Royal Manchester Children's Hospital | Manchester | United Kingdom |
| Nottingham University Hospitals NHS Trust, Children's Clinical Research Facility | Nottingham | United Kingdom |
| Royal Stoke University Hospital | Stoke-on-Trent | United Kingdom |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
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