Energy Balance and Weight Gain With Ivacaftor Treatment

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Total Enrollment

24 participants

Study Classification

OBSERVATIONAL

Study Start Date

2014-03-31

Study Completion Date

2016-11-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Study of Ivacaftor in Subjects With Cystic Fibrosis (CF) Who Have a Non-G551D CF Transmembrane Conductance Regulator (CFTR) Gating Mutation

NCT01614470

Cystic Fibrosis

COMPLETED PHASE3

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

NCT01946412

Cystic Fibrosis

COMPLETED PHASE3

Ivacaftor Treatment in 4 Month to 2 Year Old CF Subjects

NCT03783286

Cystic Fibrosis

COMPLETED

Short Term Effects of Ivacaftor in Non-G551D Cystic Fibrosis Patients

NCT01784419

Cystic Fibrosis

COMPLETED NA

Study of Ivacaftor in Cystic Fibrosis Subjects 2 Through 5 Years of Age With a CFTR Gating Mutation

NCT01705145

Cystic Fibrosis

COMPLETED PHASE3

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Cystic fibrosis (CF) is a genetic disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR), a chloride channel. Most CF mutations either reduce the number of CFTR channels at the cell surface (synthesis or processing mutations) or impair channel function (gating or conductance mutations). Ivacaftor (Kalydeco, VX-770) is a novel, FDA approved new therapy that addresses CFTR dysfunctions in subjects with CF and "gating mutations", specifically; it potentiates CFTR channel function. For mutations like G551D that permit CFTR expression at the cell membrane but compromise its activity, Ivacaftor increases the probability that the channel is open and active. In previous randomized, double-blind, placebo controlled trials, Ivacaftor treatment resulted in clinically significantly improvements in pulmonary function, weight and body mass index (BMI), and significant decreases in sweat chloride reflective of increased CFTR activity. The improvements in lung function and weight occurred over the first 8 weeks of treatment, plateaued and were sustained over the 48 weeks of the trial. The mechanism for the rapid and sustained weight gain is not known. Several mechanisms are considered in this proposal which may result in improved energy balance and energy utilization, and weight gain. These include decreased resting energy expenditure, increased energy and fat absorption from the gut, improved pancreatic enzyme and pH secretion, and increased energy intake. Improvements in weight and BMI status are expected to result from this improvement in energy balance and utilization, with potential beneficial effects on muscle mass and function and quality of life.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* 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

Exclusion Criteria

* 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

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No