Cystic Fibrosis | Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients
Cystic Fibrosis research study
What is the primary objective of this study?
This is a study of the short-term effects of ivacaftor on sweat chloride concentration and lung function in cystic fibrosis (CF) patients who fall outside current FDA approval. This new, first of its kind drug is approved for use only in CF patients with the G551D mutation in whom it safely confers considerable benefits. However, it is highly likely that CF patients with many other mutations can benefit similarly from this drug, some of whom can be identified by phenotype or genotype. We will enroll up to 30 CF subjects with clinical presentations in which there is one or more signs of residual CF channel function. The signs of residual function include: normal digestion, concentration of chloride in sweat between 55 and 85, or milder than expected CF disease in a CF patient with severe gene mutations. The primary outcome measure will be the difference in sweat chloride concentration measured in subjects on placebo and on ivacaftor. Secondary outcome measured will be lung function.
Who is eligible to participate?
Inclusion Criteria: - Two mutations known to cause cystic fibrosis and a sweat chloride concentration greater than or equal to 55 mmol - Greater than or equal to 6 years of age Exclusion Criteria: - Homozygous F508del with a sweat chloride greater than 85 mmol - Taking medication known to interact with ivacaftor and chooses not to discontinue that medication - Is pregnant or planning to become pregnant during the study period - Less than 6 years of age
Which medical condition, disease, disorder, syndrome, illness, or injury is researched?
Interventions can include giving participants drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available or noninvasive approaches such as surveys, education, and interviews.
Drug:ivacaftorProspective randomized double-blinded placebo controlled crossover study of the effects of ivacaftor in selected cystic fibrosis patients
Research studies and clinical trials typically have two or more research arms. An arm is a group of people who receive the same treatment in the study.
ivacaftor-placeboThe ivacaftor-placebo arm receives a 2 week course of ivacaftor 150 mg twice daily followed by a 2 week washout period followed by a 2 week placebo course.
placebo-ivacaftorThe placebo-ivacaftor arm receives a 2 week placebo course followed by a 2 week washout period followed by a 2 week course of ivacaftor 150 mg twice daily.
Start Date: October 2013
Completed Date: December 2015
Primary Outcome: Sweat chloride concentration
Secondary Outcome: Spirometry
Study sponsors, principal investigator, and references
Principal Investigator: Dennis W Nielson, MD, PhD
Lead Sponsor: University of California, San Francisco