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

Cystic Fibrosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02934698
• Other study ID #: Pro00052526
• Status: Completed
• Phase: Phase 3
• Start date: November 1, 2016
• Est. completion date: May 2018

Study information

• Verified date: July 2018
• Source: Medical University of South Carolina
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 2
• Est. completion date: May 2018
• Est. primary completion date: May 2018
• Accepts healthy volunteers: No
• Gender: Female
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

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.

Locations

Country	Name	City	State
n/a

Sponsors (2)

Lead Sponsor	Collaborator
Medical University of South Carolina	Vertex Pharmaceuticals Incorporated

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Forced Expiratory Volume	Absolute change in percent predicted in 1 second FEV1 from baseline through week 24	24 weeks
Secondary	Sputum Results	Achievement of mycobacterial culture conversion (negative culture)	24 weeks
Secondary	Sweat Chloride	Testing efficacy through gathering absolute change in sweat chloride from baseline through week 24	24 Weeks