Clinical trial NCT03460990: A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
Official title: A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2018-03-09
Enrollment: 116
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F).


Eligibility criteria: Key Inclusion Criteria:

* Homozygous for the F508del mutation (F/F)
* Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

* Clinically significant cirrhosis with or without portal hypertension
* Lung infection with organisms associated with a more rapid decline in pulmonary status
* Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply

Primary outcomes:
measure: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)
timeFrame: From Baseline at Week 4
Secondary outcomes:
measure: Absolute Change in Sweat Chloride (SwCl)
timeFrame: From Baseline at Week 4
measure: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score
timeFrame: From Baseline at Week 4
measure: Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to Week 8)
measure: Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, TEZ Metabolite (M1-TEZ), and IVA
timeFrame: From Day 1 and Week 4
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: New Haven
country: United States
facility: Yale New Haven Medical Center
state: Connecticut
city: Miami
country: United States
facility: University of Miami/Miller School of Medicine
state: Florida
city: Boise
country: United States
facility: St. Luke's CF Center of Idaho
state: Idaho
city: Niles
country: United States
facility: Advocate Children's Hospital- Park Ridge/ North Suburban Pulmonary and Critical Care Consultants
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Clinical Research Center, IU Health University Hospital
state: Indiana
city: Iowa City
country: United States
facility: The University of Iowa Hospitals and Clinics
state: Iowa
city: Baltimore
country: United States
facility: The Johns Hopkins Hospital
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Grand Rapids
country: United States
facility: Helen DeVos Children's Hospital CF Center
state: Michigan
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Saint Louis
country: United States
facility: Washington University School of Medicine / St. Louis Children's Hospital
state: Missouri
city: New Brunswick
country: United States
facility: Rutgers-Robert Wood Johnson Medical School
state: New Jersey
city: Albany
country: United States
facility: Albany Medical College
state: New York
city: New Hyde Park
country: United States
facility: Northwell Health, Long Island Jewish Medical Center
state: New York
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Syracuse
country: United States
facility: SUNY Upstate Medical University
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Oklahoma City
country: United States
facility: Respiratory Diseases of Children and Adolescents
state: Oklahoma
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Pittsburgh
country: United States
facility: Children's Hospital of Pittsburgh of University of Pittsburgh Medical Center
state: Pennsylvania
city: Sioux Falls
country: United States
facility: Sanford Research/ USD
state: South Dakota
city: Knoxville
country: United States
facility: University of Tennessee Medical Center-Adult Cystic Fibrosis Clinic
state: Tennessee
city: Memphis
country: United States
facility: Children's Foundation Research Center/ Le Bonheur Children's Hospital
state: Tennessee
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah/ Primary Children's Medical Center
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Melbourne
country: Australia
facility: The Alfred Hospital
state: Victoria
city: Adelaide
country: Australia
facility: Royal Adelaide Hospital
city: Chermside
country: Australia
facility: Prince Charles Hospital
city: Nedlands
country: Australia
facility: Institute for Respiratory Health Inc./ Sir Charles Gairdner Hospital
city: New Lambton Heights
country: Australia
facility: John Hunter Hospital & Hunter Medical Research Institute
city: Berlin
country: Germany
facility: Charite Paediatric Pulmonology Department
city: Muenchen
country: Germany
facility: Pneumologische Praxis Pasing
city: Dublin
country: Ireland
facility: Cork University Hospital
city: Dublin
country: Ireland
facility: Our Lady's Children's Hospital
city: Dublin
country: Ireland
facility: St. Vincent's University Hospital
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d Hebron
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron Servicio de Broncoscopia
city: Sevilla
country: Spain
facility: Hospital Universitario Virgen del Rocio
city: Cambridge
country: United Kingdom
facility: Papworth Hospital NHS Foundation Trust, Papworth Everard
city: Liverpool
country: United Kingdom
facility: Liverpool Heart and Chest Hospital
city: London
country: United Kingdom
facility: Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
city: Manchester
country: United Kingdom
facility: Wythenshawe Hospital
city: Penarth
country: United Kingdom
facility: University Hospital Llandough
Study officials:
References: