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NCT Number: NCT02934698

An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations

This postmarketing N of 2 study is designed to evaluate the efficacy and safety of open-label ivacaftor treatment in two sisters with cystic fibrosis and pancreatic sufficiency.

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Key information

Conditions

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

Age range

18 year and older

Sex eligibility

Female

Study type

Interventional

Phase

Phase 3

About this study

The two sisters have a splicing mutation that is predicted to respond favorably to ivacaftor therapy. In addition to measurement of usual clinical outcomes (i.e. lung function, nutritional status), there is great interest on the impact on nontuberculous mycobacteria (NTM) airways infection.

Subjects will undergo sputum cultures at baseline and monthly during treatment, initially in the absence of anti-NTM therapy but with the intent to treat with antibiotics if there is persistence of the infection in cultures. Other clinical outcomes will include changes in sweat chloride, lung function and weight. Safety measures will include periodic assessment of liver enzymes. All serious and non-serious adverse events will be collected

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Subjects are >18 years of age and able to provide informed consent.
  • Subjects reside in the US and are willing to be treated with ivacaftor.
  • Subjects have the splicing mutation of interest.
  • Subjects are willing and able to perform requirements of the study.

Exclusion criteria

  • There are no relevant exclusion criteria for this n-of-2 study.

Treatment and study plan

Ivacaftor

Drug

Subjects will be treated with ivacaftor for 6 months and followed for 7 months and will undergo assessments along the way to measure sweat chloride and sputum amounts.

Primary outcomes

  1. Forced Expiratory Volume

    Time frame: 24 weeks

    Absolute change in percent predicted in 1 second FEV1 from baseline through week 24

Secondary outcomes

  1. Sputum Results

    Time frame: 24 weeks

    Achievement of mycobacterial culture conversion (negative culture)

  2. Sweat Chloride

    Time frame: 24 Weeks

    Testing efficacy through gathering absolute change in sweat chloride from baseline through week 24

Sponsors and collaborators

Lead sponsor

Medical University of South Carolina

Other

Collaborators

  • Vertex Pharmaceuticals Incorporated

Registry information

Important dates

Study start
2016
Primary completion
2018
Study completion
2018
First posted
Oct 17, 2016
Registry last updated
Aug 6, 2018

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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