Clinical trial NCT02070744: Study to Evaluate Safety and Efficacy of VX-661 in Combination With Ivacaftor in Subjects With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation With an Open-Label Expansion
Official title: A Phase 2, Randomized, Multicenter, Double Blind, Placebo Controlled Study to Evaluate Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of VX-661 in Combination With Ivacaftor for 12 Weeks in Subjects With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation With an Open-Label Extension
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Cystic Fibrosis
Keywords: CF
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2014-02-25
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The objective of this study was to evaluate the safety and efficacy of VX-661in combination with ivacaftor in participants with cystic fibrosis (CF) who are homozygous for F508del cystic fibrosis transmembrane conductance regulator (CFTR) mutation


Eligibility criteria: Inclusion Criteria:

* Homozygous for the F508del CFTR mutation
* FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height
* Stable CF disease as judged by the investigator

Exclusion Criteria:

* 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
* Pregnant and nursing females: Females of childbearing potential must have a negative pregnancy test at screening and Day 1 of the PC Phase and Day -7 or Day 1 of the OLE Phase (whichever was applicable)
* Sexually active participants of reproductive potential who are not willing to follow the contraception requirements
* The participant or a close relative of the participant is the investigator or sub investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study.

Primary outcomes:
measure: PC Phase: Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Baseline (PC Phase) up to 112 days
measure: OLE Phase: Number of Participants With Treatment-Emergent AEs and SAEs
timeFrame: Baseline (OLE Phase) up to 364 days
Secondary outcomes:
measure: PC Phase: Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 12
timeFrame: Baseline (PC Phase), Through Week 12
measure: OLE Phase: Absolute Change From Baseline in Percent Predicted FEV1 Through Week 40
timeFrame: Baseline (OLE Phase), Through Week 40
measure: PC Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 12
timeFrame: Baseline (PC Phase), Through Week 12
measure: OLE Phase: Relative Change From Baseline in Percent Predicted FEV1 Through Week 40
timeFrame: Baseline (OLE Phase), Through Week 40
measure: PC Phase: Absolute Change From Baseline in Sweat Chloride Through Week 12
timeFrame: Baseline (PC Phase), Through Week 12
measure: OLE Phase: Absolute Change From Baseline in Sweat Chloride Through Week 40
timeFrame: Baseline (OLE Phase), Through Week 40
measure: PC Phase: Absolute Change From Baseline in Body Weight at Week 12
timeFrame: Baseline (PC Phase), Week 12
measure: OLE Phase: Absolute Change From Baseline in Body Weight at Week 40
timeFrame: Baseline (OLE Phase), Week 40
measure: PC Phase: Absolute Change From Baseline Body Mass Index (BMI) at Week 12
timeFrame: Baseline (PC Phase), Week 12
measure: OLE Phase: Absolute Change From Baseline BMI at Week 40
timeFrame: Baseline (OLE Phase), Week 40
measure: PC Phase: Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 12
timeFrame: Baseline (PC Phase), Through Week 12
measure: OLE Phase: Absolute Change From Baseline in CFQ-R Respiratory Domain Score Through Week 40
timeFrame: Baseline (OLE Phase), Through Week 40
measure: PC Phase: Maximum Plasma Concentration (Cmax) of VX-661 and IVA
timeFrame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
measure: PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 24 Hours (AUC0-24h) of VX-661
timeFrame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
measure: PC Phase: Area Under the Concentration Versus Time Curve From Time 0 to 12 Hours (AUC0-12h) of IVA
timeFrame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
measure: PC Phase: Time to Reach Cmax (Tmax) of VX-661 and IVA
timeFrame: Pre-dose, 2, 3, 4, 6, 9 and 12 hours post-dose on Day 85
Other outcomes:
Locations:
city: Birmingham
country: United States
state: Alabama
city: Palo Alto
country: United States
state: California
city: Stanford
country: United States
state: California
city: Altamonte Springs
country: United States
state: Florida
city: Miami
country: United States
state: Florida
city: Orlando
country: United States
state: Florida
city: Tampa
country: United States
state: Florida
city: Boise
country: United States
state: Idaho
city: Chicago
country: United States
state: Illinois
city: Boston
country: United States
state: Massachusetts
city: New Brunswick
country: United States
state: New Jersey
city: New York
country: United States
state: New York
city: Durham
country: United States
state: North Carolina
city: Cincinnati
country: United States
state: Ohio
city: Oklahoma City
country: United States
state: Oklahoma
city: Pittsburgh
country: United States
state: Pennsylvania
city: Charleston
country: United States
state: South Carolina
city: Memphis
country: United States
state: Tennessee
city: Houston
country: United States
state: Texas
city: Burlington
country: United States
state: Vermont
city: Colchester
country: United States
state: Vermont
city: Seattle
country: United States
state: Washington
city: Milwaukee
country: United States
state: Wisconsin
Study officials:
References: