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

Primary Purpose

Cystic Fibrosis

Status

Completed

Phase

Phase 3

Locations

Study Type

Interventional

Intervention

Ivacaftor

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)FemaleDoes not accept healthy volunteers

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.

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Ivacaftor

Arm Description

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

Outcomes

Primary Outcome Measures

Forced Expiratory Volume

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

Secondary Outcome Measures

Sputum Results

Achievement of mycobacterial culture conversion (negative culture)

Sweat Chloride

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

Full Information

NCT ID

NCT02934698

First Posted

October 13, 2016

Last Updated

July 10, 2018

Sponsor

Medical University of South Carolina

Collaborators

Vertex Pharmaceuticals Incorporated

1. Study Identification

Unique Protocol Identification Number

NCT02934698

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

July 2018

Overall Recruitment Status

Completed

Study Start Date

November 1, 2016 (Actual)

Primary Completion Date

May 2018 (Actual)

Study Completion Date

May 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Medical University of South Carolina

Collaborators

Vertex Pharmaceuticals Incorporated

4. Oversight

Data Monitoring Committee

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

2 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Ivacaftor

Arm Type

Experimental

Arm Description

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 Type

Drug

Intervention Name(s)

Ivacaftor

Intervention Description

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 Outcome Measure Information:

Title

Forced Expiratory Volume

Description

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

Time Frame

24 weeks

Secondary Outcome Measure Information:

Title

Sputum Results

Description

Achievement of mycobacterial culture conversion (negative culture)

Time Frame

24 weeks

Title

Sweat Chloride

Description

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

Time Frame

24 Weeks

10. Eligibility

Sex

Female

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

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.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Patrick A Flume, MD

Organizational Affiliation

Medical University of South Carolina

Official's Role

Principal Investigator

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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