Energy Balance and Weight Gain With Ivacaftor Treatment

Completed

Conditions studied: Cystic Fibrosis

In brief

Ivacaftor is a novel, FDA approved new therapy that addresses Cystic fibrosis transmembrane conductance regulator (CFTR) dysfunctions in subjects with Cystic fibrosis (CF) and "gating mutations". The primary aim is to determine the mechanism(s) for weight gain in participants whom Ivacaftor treatment was initiated based on clinical indications by CF Care Team. This longitudinal study will assess in detail energy expenditure, weight gain, body composition, and lung function in 24 subjects ≥6 years old with CF with a gating mutation before treatment and after three months treatment with Ivacaftor. All subjects will be seen at the Children's Hospital of Philadelphia's Clinical Translational Research Center.

Key facts

Study ID

NCT02141464

Run by

Children's Hospital of Philadelphia

People needed

24

Starts

2014-03-01

Expected to finish

2016-11-01

Last updated by the study team

2017-07-19

Who can join

Age: 6 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Cystic fibrosis with one or two CFTR gating mutations
• Age: 6 years and older
• A clinical decision has been made for the subject to start Ivacaftor treatment
• In usual state of good health
• Family and subject commitment to the 3-month study protocol with two, 3-4 day visits to CHOP

You may not qualify if…

• FEV1 < 40% predicted
• Use of any inhibitors or inducers of cytochrome P450 (CYP) 3A
• Pregnancy or breast feeding
• Other illness affecting growth or nutritional status
• Subjects receiving total parenteral nutrition

Where it is running

• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States

Full record on ClinicalTrials.gov

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