A Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Cystic Fibrosis Clinical Trial

Official title:

A Phase 2, Randomized, Placebo-Controlled, Double-blind Study to Evaluate the Effect of VX-661 in Combination With Ivacaftor on Chest Imaging Endpoints in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02730208
• Other study ID #: VX15-661-112
• Status: Completed
• Phase: Phase 2
• Start date: September 2016
• Est. completion date: July 2018

Study information

• Verified date: October 2019
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The primary purpose of study is to evaluate the treatment effect of tezacaftor in combination with ivacaftor (TEZ/IVA) on chest imaging endpoints using low-dose computed tomography (LDCT) at Week 72, and to evaluate the safety of TEZ/IVA through Week 72.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 41
• Est. completion date: July 2018
• Est. primary completion date: July 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Homozygous for the F508del CFTR mutation

• Confirmed diagnosis of CF

• Percent predicted forced expiratory volume (ppFEV1) =70% of predicted normal for age, sex, and height during screening.

• 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 (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug)

• Pregnant or nursing females.

• Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.

• Any contraindication to undergoing chest imaging, as per the site's institutional guidelines

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Tezacaftor/Ivacaftor
TEZ 100 mg/IVA 150 mg fixed-dose combination tablet.
• Ivacaftor
IVA 150 mg tablet.
• Placebo
Placebo matched to TEZ/IVA fixed-dose combination tablet.
• Placebo
Placebo matched to IVA tablet.

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

Australia, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute Change in Total Brody/CF-CT Score	The exploratory Brody/CF-CT score semi-quantitatively scores the degree of structural lung disease as shown on CT in participants with CF. The score ranges from a minimum of 0 to a maximum of 219 with higher scores indicating more severe structural lung disease.	From Baseline at Week 72
Secondary	Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)		Day 1 up to Week 76