A Phase 2 Study to Evaluate Effects of VX-661/Ivacaftor on Lung and Extrapulmonary Systems in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Cystic Fibrosis Clinical Trial

Official title:

A Phase 2, Randomized, Double-Blind, Placebo Controlled, Parallel-Group, Exploratory Study to Evaluate Effects of VX-661 in Combination With Ivacaftor on Lung and Extrapulmonary Systems in Subjects Aged 18 Years and Older With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02508207
• Other study ID #: VX14-661-111
• Status: Completed
• Phase: Phase 2
• Start date: February 2016
• Est. completion date: June 2017

Study information

• Verified date: June 2018
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

To evaluate the clinical mechanisms of action in lung and extrapulmonary systems of VX-661 (tezacaftor; TEZ) in combination with ivacaftor (IVA) (TEZ/IVA) in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 34
• Est. completion date: June 2017
• Est. primary completion date: June 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Male and female participants, homozygous for the F508del CFTR mutation

• Confirmed diagnosis of CF by sweat chloride testing

• Forced Expiratory Volume in 1 Second (FEV1) =40% and =90% of predicted normal for age, sex, and height at Screening Visit

• Stable CF disease as judged by the investigator.

Exclusion Criteria:

• History of any comorbidity that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject.

• An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1

• History or evidence of clinically significant findings on ophthalmologic examination during the Screening Period.

• History of solid organ or hematological transplantation

• Pregnant or nursing females

• Participants who have had radiation exposure within 1 year before the first mucociliary clearance (MCC) procedure that would cause them to exceed federal regulations by participating in this study

• In the opinion of the investigator, unable to adequately perform inhalation maneuvers during the MCC procedures

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Tezacaftor/Ivacaftor
Tezacaftor/Ivacaftor FDC
• Ivacaftor

• Tezacaftor/Ivacaftor matching placebo

• Ivacaftor matching placebo

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute Change From Baseline in Mucociliary Clearance (MCC) at Day 28	MCC was assessed using an imaging technique that enables the tracking of mucus within the airways. MCC was expressed as the percentage of whole-lung clearance through 60 minutes at Baseline and Day 28.	Baseline, Day 28
Secondary	Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) at Day 28	Percent predicted FEV1 is the ratio of FEV1 to the predicted FEV1, expressed as a percentage. FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.	Baseline, Day 28
Secondary	Absolute Change From Baseline in Small-bowel Area Under the Curve (AUC) Over 1-minute Mean pH Increments at Day 29	Absolute change from Baseline in small bowel AUC over 1-minute mean pH increments through 30 minutes at Day 29 was assessed.	Baseline, Day 29
Secondary	Absolute Change From Baseline in Sweat Chloride at Day 29		Baseline, Day 29
Secondary	Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)		Baseline up to Day 57