A Study of the Effects of Lumacaftor/Ivacaftor (LUM/IVA) on Exercise Tolerance in Subjects With Cystic Fibrosis (CF), Homozygous for the F508del-CFTR Mutation

NCT ID: NCT02875366

Last Updated: 2019-06-17

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

70 participants

Study Classification

INTERVENTIONAL

Study Start Date

2016-09-30

Study Completion Date

2017-10-31

Brief Summary

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This is a Phase 4, randomized, double-blind, placebo-controlled, parallel-group study in subjects aged 12 years and older with CF who are homozygous for the F508del-CFTR mutation. This study is designed to evaluate the effect of LUM/IVA on exercise tolerance in subjects with CF, homozygous for the F508del-CFTR mutation.

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

OTHER

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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Placebo

Placebo matched to LUM/IVA fixed-dose combination tablet orally every 12 hours (q12h) for 24 weeks.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

LUM/IVA

LUM 400 milligram (mg)/IVA 250 mg fixed-dose combination tablet orally q12h for 24 weeks.

Group Type EXPERIMENTAL

LUM/IVA

Intervention Type DRUG

Interventions

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LUM/IVA

Intervention Type DRUG

Placebo

Intervention Type DRUG

Other Intervention Names

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Lumacaftor/Ivacaftor

Eligibility Criteria

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Inclusion Criteria

* Homozygous for the F508del-CFTR mutation
* Confirmed diagnosis of CF defined as a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis
* Stable CF disease as judged by the investigator
* Forced expiratory volume in 1 second (FEV1) at least 40% and not greater than 90% of predicted

Exclusion Criteria

* History of any comorbidity that might confound the results of the study, interfere with the use of cardiopulmonary exercise tests (CPETs) as an assessment, or pose an additional risk in administering study drug to the subject
* Any previous exposure to LUM or IVA
* History of cardiac arrhythmia, ischemic heart disease, congestive heart failure, or other clinically significant cardiac condition, or medical condition requiring chronic use of a beta blocker, non-dihydropyridine calcium channel blocker, or other cardiac medication known to affect exercise tolerance
* History of solid organ or hematological transplantation
* For subjects under 18 years of age at Screening, except those who have had bilateral lens removal, selected findings on a screening ophthalmologic examination will be exclusionary
* Using or expected to require any concomitant medication that is prohibited in this study
* History of alcohol or drug abuse, as deemed by the investigator, in the past year, including but not limited to cannabis, cocaine, and opiates
* Participation in an investigational drug study within 30 days before the Screening Visit
* Pregnant or nursing females; males with a female partner who is pregnant or nursing
* Colonization with organisms associated with a more rapid decline in pulmonary status
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Melbourne, Victoria, Australia

Site Status

Parkville, Victoria, Australia

Site Status

Adelaide, , Australia

Site Status

Camperdown, , Australia

Site Status

Clayton, , Australia

Site Status

Nedlands, , Australia

Site Status

New Lambton Heights, , Australia

Site Status

Randwick, , Australia

Site Status

South Brisbane, , Australia

Site Status

Subiaco, , Australia

Site Status

Westmead, , Australia

Site Status

Edinburgh, , United Kingdom

Site Status

Countries

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Australia United Kingdom

References

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Wilson J, You X, Ellis M, Urquhart DS, Jha L, Duncan M, Tian S, Harris RA, Kotsimbos T, Keating D. VO2max as an exercise tolerance endpoint in people with cystic fibrosis: Lessons from a lumacaftor/ivacaftor trial. J Cyst Fibros. 2021 May;20(3):499-505. doi: 10.1016/j.jcf.2020.12.006. Epub 2020 Dec 24.

Reference Type DERIVED
PMID: 33358691 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2016-000066-34

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX15-809-112

Identifier Type: -

Identifier Source: org_study_id

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