Energy Balance and Weight Gain With Ivacaftor Treatment

NCT02141464 | Completed

• 1 other identifier: 13-010622
• Study Type: observational
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• Reduction in resting energy expenditure

  3 months

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

24 subjects ≥6 years old with CF with a gating mutation before treatment (baseline) and after three months treatment with Ivacaftor.

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

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• Vertex Pharmaceuticals Incorporated: collaborator

Study Sites (1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Related Publications (2)

• Stallings VA, Sainath N, Oberle M, Bertolaso C, Schall JI. Energy Balance and Mechanisms of Weight Gain with Ivacaftor Treatment of Cystic Fibrosis Gating Mutations. J Pediatr. 2018 Oct;201:229-237.e4. doi: 10.1016/j.jpeds.2018.05.018. Epub 2018 Jul 18.

  PMID: 30029855 DERIVED

• Sainath NN, Schall J, Bertolaso C, McAnlis C, Stallings VA. Italian and North American dietary intake after ivacaftor treatment for Cystic Fibrosis Gating Mutations. J Cyst Fibros. 2019 Jan;18(1):135-143. doi: 10.1016/j.jcf.2018.06.004. Epub 2018 Jul 6.

  PMID: 29983355 DERIVED

Biospecimen

Retention: SAMPLES WITHOUT DNA

serum, urine, stool

MeSH Terms

Conditions

Cystic Fibrosis

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Study Officials

• Virginia Stallings, MD

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 15, 2014
• First Posted: May 19, 2014
• Study Start: March 1, 2014
• Primary Completion: February 1, 2016
• Study Completion: November 1, 2016
• Last Updated: July 19, 2017

Record last verified: 2017-07

Locations

US (1)