Clinical trial NCT02390219: Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Advanced Lung Disease
Official title: A Phase 3b, Open-Label Study to Evaluate Lumacaftor and Ivacaftor Combination Therapy in Subjects 12 Years and Older With Cystic Fibrosis and Advanced Lung Disease, Homozygous for the F508del-CFTR Mutation
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Advanced Lung Disease
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2015-03-17
Enrollment: 46
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Treatment Emergent Adverse Events (AEs) or Serious Adverse Events (SAEs)
timeFrame: Day 1 up to Week 28
Secondary outcomes:
measure: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24
timeFrame: Baseline, Up to Week 24
measure: Absolute Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) Up to Week 24
timeFrame: Baseline, Up to Week 24
measure: Duration For Which Participants Received Intravenous (IV) Antibiotics
timeFrame: Baseline through Week 24
measure: Number of Hospitalizations
timeFrame: Baseline through Week 24
measure: Absolute Change From Baseline in Sweat Chloride at Average of Day 15 and Week 4
timeFrame: Baseline, Day 15 and Week 4
measure: Absolute Change From Baseline in Cystic Fibrosis Questionnaire - Revised (CFQ-R) Respiratory Domain Score Through Week 24
timeFrame: Baseline, Through Week 24
Other outcomes:
Locations:
city: Denver
country: United States
state: Colorado
city: Tampa
country: United States
state: Florida
city: Chicago
country: United States
state: Illinois
city: Saint Louis
country: United States
state: Missouri
city: Pittsburgh
country: United States
state: Pennsylvania
city: Houston
country: United States
state: Texas
Study officials:
References:
citation: 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.