Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis; Advanced Lung Disease

Treatments: Drug: Lumacaftor; Drug: Ivacaftor

Study type: Interventional

Funder types: Industry

Identifiers

NCT02390219

VX14-809-106

Details and patient eligibility

About

The purpose of this study is to evaluate the safety and tolerability of LUM/IVA combination therapy in subjects 12 years and older with CF and advanced lung disease and who are homozygous for the F508del CFTR mutation

Enrollment: 46 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Homozygous for the F508del-CFTR mutation; historical genotype must be documented in the participant's source documents.
• Percent predicted FEV1 <40 of adjusted for age, sex, and height at Screening

Exclusion criteria

• Participant currently receiving invasive mechanical ventilation.
• 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 participant
• Any clinically significant laboratory abnormalities at screening that would interfere with the study assessments or pose an undue risk for the subject
• A 12-lead electrocardiograms (ECG) demonstrating QTcF >450 msec at Screening
• History of solid organ or hematological transplantation
• History of alcohol or drug abuse in the past year
• Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of screening.
• Use of strong inhibitors, moderate inducers, or strong inducers of CYP3A
• Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at Screening and Day 1.
• Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements
• Use of beta blockers or the equivalent at Screening.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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46 participants in 1 patient group

Lumacaftor/Ivacaftor combination

Experimental group

Description:

Lumacaftor 400 milligram (mg) and ivacaftor 250 mg combination tablet orally twice daily for 24 weeks.

Treatment:

Drug: Ivacaftor

Drug: Lumacaftor