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

July 10, 2018 updated by: Medical University of South Carolina
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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Study Type

Interventional

Enrollment (Actual)

2

Phase

  • Phase 3

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Female

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: Ivacaftor
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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Forced Expiratory Volume
Time Frame: 24 weeks
Absolute change in percent predicted in 1 second FEV1 from baseline through week 24
24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Sputum Results
Time Frame: 24 weeks
Achievement of mycobacterial culture conversion (negative culture)
24 weeks
Sweat Chloride
Time Frame: 24 Weeks
Testing efficacy through gathering absolute change in sweat chloride from baseline through week 24
24 Weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Medical University of South Carolina

Collaborators

Vertex Pharmaceuticals Incorporated

Investigators

  • Principal Investigator: Patrick A Flume, MD, Medical University of South Carolina

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 1, 2016

Primary Completion (Actual)

May 1, 2018

Study Completion (Actual)

May 1, 2018

Study Registration Dates

First Submitted

October 13, 2016

First Submitted That Met QC Criteria

October 14, 2016

First Posted (Estimate)

October 17, 2016

Study Record Updates

Last Update Posted (Actual)

August 6, 2018

Last Update Submitted That Met QC Criteria

July 10, 2018

Last Verified

July 1, 2018

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • Pro00052526

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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