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

NCT ID: NCT02390219

Last Updated: 2017-12-06

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

46 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-03-31

Study Completion Date

2016-10-31

Brief Summary

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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

Detailed Description

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Conditions

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Cystic Fibrosis Advanced Lung Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Lumacaftor/Ivacaftor combination

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

Group Type EXPERIMENTAL

Lumacaftor

Intervention Type DRUG

Ivacaftor

Intervention Type DRUG

Interventions

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Lumacaftor

Intervention Type DRUG

Ivacaftor

Intervention Type DRUG

Other Intervention Names

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VX-809 VX-770

Eligibility Criteria

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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.
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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Denver, Colorado, United States

Site Status

Tampa, Florida, United States

Site Status

Chicago, Illinois, United States

Site Status

St Louis, Missouri, United States

Site Status

Pittsburgh, Pennsylvania, United States

Site Status

Houston, Texas, United States

Site Status

Countries

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United States

References

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Taylor-Cousar JL, Jain M, Barto TL, Haddad T, Atkinson J, Tian S, Tang R, Marigowda G, Waltz D, Pilewski J; VX14-809-106 Investigator Group. Lumacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease homozygous for F508del-CFTR. J Cyst Fibros. 2018 Mar;17(2):228-235. doi: 10.1016/j.jcf.2017.09.012. Epub 2017 Nov 8.

Reference Type DERIVED
PMID: 29126871 (View on PubMed)

Other Identifiers

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VX14-809-106

Identifier Type: -

Identifier Source: org_study_id