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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

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 2
Locations
Australia
Study Type
Interventional
Intervention
Tezacaftor/Ivacaftor
Ivacaftor
Placebo
Placebo
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

TEZ/IVA

Placebo

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Full Information

First Posted
March 31, 2016
Last Updated
October 22, 2019
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT02730208
Brief Title
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
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
Study Type
Interventional

2. Study Status

Record Verification Date
October 2019
Overall Recruitment Status
Completed
Study Start Date
September 2016 (Actual)
Primary Completion Date
July 2018 (Actual)
Study Completion Date
July 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
41 (Actual)

8. Arms, Groups, and Interventions

Arm Title
TEZ/IVA
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
Tezacaftor/Ivacaftor
Other Intervention Name(s)
TEZ/IVA; VX-661/VX-770
Intervention Description
TEZ 100 mg/IVA 150 mg fixed-dose combination tablet.
Intervention Type
Drug
Intervention Name(s)
Ivacaftor
Other Intervention Name(s)
VX-770; IVA
Intervention Description
IVA 150 mg tablet.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo matched to TEZ/IVA fixed-dose combination tablet.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo matched to IVA tablet.
Primary Outcome Measure Information:
Title
Absolute Change in Total Brody/CF-CT Score
Description
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.
Time Frame
From Baseline at Week 72
Secondary Outcome Measure Information:
Title
Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
Day 1 up to Week 76

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Facility Information:
City
Chermside
Country
Australia
City
Melbourne
Country
Australia
City
Nedlands
Country
Australia
City
New Lambton Heights
Country
Australia
City
Parkville SIC
Country
Australia
City
Randwick
Country
Australia
City
South Brisbane
Country
Australia
City
Subiaco
Country
Australia
City
Westmead
Country
Australia

12. IPD Sharing Statement

Learn more about this trial

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

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