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

October 22, 2019 updated by: Vertex Pharmaceuticals Incorporated

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

41

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
      • Chermside, Australia
      • Melbourne, Australia
      • Nedlands, Australia
      • New Lambton Heights, Australia
      • Parkville SIC, Australia
      • Randwick, Australia
      • South Brisbane, Australia
      • Subiaco, Australia
      • Westmead, Australia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: TEZ/IVA
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.
Drug: Tezacaftor/Ivacaftor
TEZ 100 mg/IVA 150 mg fixed-dose combination tablet.
Other Names:
  • TEZ/IVA; VX-661/VX-770
Drug: Ivacaftor
IVA 150 mg tablet.
Other Names:
  • VX-770; IVA
Placebo Comparator: 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.
Drug: Placebo
Placebo matched to TEZ/IVA fixed-dose combination tablet.
Drug: Placebo
Placebo matched to IVA tablet.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Absolute Change in Total Brody/CF-CT Score
Time Frame: 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.
From Baseline at Week 72

Secondary Outcome Measures

Outcome Measure
Time Frame
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Day 1 up to Week 76
Day 1 up to Week 76

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

September 1, 2016

Primary Completion (Actual)

July 1, 2018

Study Completion (Actual)

July 1, 2018

Study Registration Dates

First Submitted

March 31, 2016

First Submitted That Met QC Criteria

March 31, 2016

First Posted (Estimate)

April 6, 2016

Study Record Updates

Last Update Posted (Actual)

October 23, 2019

Last Update Submitted That Met QC Criteria

October 22, 2019

Last Verified

October 1, 2019

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX15-661-112

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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