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

Vertex Pharmaceuticals

Status and phase: Completed; Phase 4

Conditions: Cystic Fibrosis

Treatments: Drug: Placebo; Drug: LUM/IVA

Study type: Interventional

Funder types: Industry

Identifiers

NCT02875366

2016-000066-34 (EudraCT Number)

VX15-809-112

Details and patient eligibility

About

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.

Enrollment: 70 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Other

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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70 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo

LUM/IVA

Experimental group

Description:

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

Treatment:

Drug: LUM/IVA