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A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have a CFTR Gating Mutation

This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational ar...

A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Ivacaftor in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age and Have a CFTR Gating Mutation

The purpose of this study is to evaluate the safety of ivacaftor treatment, and PK of ivacaftor and...

A Two-Part Multicenter Prospective Longitudinal Study of CFTR-dependent Disease Profiling in Cystic Fibrosis (PROSPECT)

Identify and validate biomarkers that might reflect partial restoration of CFTR function and can be...

Genetic Modifiers of Cytic Fibrosis Related Diabetes

This research is being done to find the genes and other factors that are responsible for differences...

Long-term Study in US Cystic Fibrosis Patients Receiving Digestive Enzyme Supplements to Assess Narrowing of the Large Intestine Causing Adverse Intestinal Symptoms (Fibrosing Colonopathy)

This is a long-term study in cystic fibrosis patients who are participating in the Cystic Fibrosis P...

Observational Study in Cystic Fibrosis Patients Using TOBI® Podhaler™ or Other FDA Approved Inhaled Antipseudomonal Antibacterial Drugs

This is a multicenter, prospective, two cohort, observational study over a 5-year period in Cystic F...

Safety and Efficacy of 2 Treatment Regimens of Aztreonam for Inhalation Solution in Children With Cystic Fibrosis and New Onset Pseudomonas Aeruginosa Infection (ALPINE2)

The primary objective of this study is to evaluate the safety and efficacy of a 14-day course versus...

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