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Clinical Trials/NCT02934698
NCT02934698
Completed
Phase 3

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

Medical University of South Carolina0 sites2 target enrollmentNovember 1, 2016
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ConditionsCystic Fibrosis
InterventionsIvacaftor
DrugsIvacaftor

Overview

Phase
Phase 3
Intervention
Ivacaftor
Conditions
Cystic Fibrosis
Sponsor
Medical University of South Carolina
Enrollment
2
Primary Endpoint
Forced Expiratory Volume
Status
Completed
Last Updated
7 years ago
OverviewInvestigatorsEligibility CriteriaArms & InterventionsOutcomesSimilar Trials

Overview

Brief Summary

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.

Detailed Description

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

Registry
clinicaltrials.gov
Start Date
November 1, 2016
End Date
May 2018
Last Updated
7 years ago
Study Type
Interventional
Study Design
Single Group
Sex
Female

Investigators

Responsible Party
Sponsor

Eligibility Criteria

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.

Arms & Interventions

Ivacaftor

There is only one arm to this study. The two sisters with Cystic Fibrosis will both receive Ivacaftor for 6 months for their treatment.

Intervention: Ivacaftor

Outcomes

Primary Outcomes

Forced Expiratory Volume

Time Frame: 24 weeks

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

Secondary Outcomes

  • Sputum Results(24 weeks)
  • Sweat Chloride(24 Weeks)

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