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

Phase 2

Completed

• Conditions: Cystic Fibrosis
• Interventions: Drug: Tezacaftor/Ivacaftor; Drug: Placebo; Drug: Ivacaftor

• Registration Number: NCT02730208
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2016-03-31
• Study First Submitted QC: 2016-03-31
• Study First Posted: 2016-04-06
• Study Start: 2016-09
• Primary Completion: 2018-07
• Study Completion: 2018-07
• Results First Submitted: 2019-07-30
• Results First Submitted QC: 2019-07-30
• Results First Posted: 2019-08-21
• Status Verified: 2019-10
• Last Update Submitted: 2019-10-22
• Last Update Posted: 2019-10-23

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 41

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
TEZ/IVA	Tezacaftor/Ivacaftor	Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
TEZ/IVA	Ivacaftor	Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 72 weeks.
Placebo	Placebo	Participants received placebo matched to TEZ/IVA fixed dose combination tablet orally once daily in the morning and placebo matched to IVA tablet orally once daily in the evening for 72 weeks.

Primary Outcome Measures

Name	Time	Method
Absolute Change in Total Brody/CF-CT Score	From Baseline at Week 72	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.

Secondary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)	Day 1 up to Week 76