A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy of Lumacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis Who Have an A455E-CFTR Mutation

Vertex Pharmaceuticals Incorporated2 sites in 1 country20 target enrollmentJanuary 31, 2017

ConditionsCystic Fibrosis

InterventionsLUM/IVA Placebo

DrugsLUM/IVA Placebo

Overview

Phase: Phase 2
Intervention: LUM/IVA
Conditions: Cystic Fibrosis
Sponsor: Vertex Pharmaceuticals Incorporated
Enrollment: 20
Locations: 2
Primary Endpoint: Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 8
Status: Completed
Last Updated: 7 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter, crossover study that will evaluate the efficacy of LUM/IVA in subjects with CF 12 years of age and older who have at least one A455E mutation.

Registry

clinicaltrials.gov

Start Date

January 31, 2017

End Date

October 4, 2017

Last Updated

7 years ago

Study Type

Interventional

Study Design

Crossover

Sex

All

Investigators

Sponsor

Vertex Pharmaceuticals Incorporated

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Male or female with confirmed diagnosis of CF
•All subjects must have an A455E mutation on at least 1 CFTR allele; no more than 10 subjects may have an F508del mutation on 1 CFTR allele.
•Forced expiratory volume in one second (FEV1) ≥30% of predicted and ≤90% of predicted at the Screening Visit, based on the Global Lung Function Initiative (GLI)-2012 multi ethnic all-age reference equations.
•Stable CF disease as judged by the investigator.
•Willing to remain on a stable medication regimen for CF from 4 weeks before Day 1 through the Follow up Visit.

Exclusion Criteria

•History of any comorbidity reviewed at the Screening Visit 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.
•A G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutation on at least one CFTR allele.
•Pregnant or breastfeeding.
•Any abnormal laboratory values at the Screening Visit.
•History of cataract/lens opacity, or evidence of cataract/lens opacity determined to be clinically significant by the ophthalmologist or optometrist during the ophthalmologic examination at the Screening Visit.
•Use of strong inhibitors or strong inducers of CYP3A, including consumption of certain herbal medications and certain fruit and fruit juices, within 14 days before Day 1
•Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements