A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)
NCT ID: NCT03150719
Last Updated: 2019-09-12
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
98 participants
INTERVENTIONAL
2017-05-24
2018-08-09
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
Participants received placebo matched to TEZ/IVA fixed-dose combination tablet orally once daily in the morning followed by placebo matched to IVA tablet orally once daily in the evening for 56 days.
Placebo
Placebo matched to TEZ/IVA fixed-dose combination tablet.
Placebo
Placebo matched to IVA tablet.
TEZ/IVA
Participants received TEZ 100 milligram (mg)/IVA 150 mg fixed-dose combination tablet orally once daily in the morning and IVA 150 mg tablet orally once daily in the evening for 56 days.
Tezacaftor/Ivacaftor
TEZ 100 mg/IVA 150 mg fixed-dose combination tablet.
Ivacaftor
IVA 150 mg tablet.
Interventions
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Tezacaftor/Ivacaftor
TEZ 100 mg/IVA 150 mg fixed-dose combination tablet.
Ivacaftor
IVA 150 mg tablet.
Placebo
Placebo matched to TEZ/IVA fixed-dose combination tablet.
Placebo
Placebo matched to IVA tablet.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Prior discontinuation of Orkambi, with at least 1 respiratory sign or symptom considered related to therapy.
* Resolution or stabilization of qualifying event(s) \>28 days prior to Screening.
* Discontinuation of Orkambi therapy must have occurred within approximately 12 weeks from the first dose of Orkambi.
* Homozygous for F508del mutation in the CFTR gene as documented in the subject's medical record. If genotype documentation is not available in the medical record, genotyping will be performed during screening.
* FEV1 ≥25% and ≤90% of predicted normal for age, sex, and height.
* Stable CF disease as judged by the investigator.
Exclusion Criteria
* Recent rapid or progressive deterioration in respiratory status.
* Receiving continuous oxygen at \>2L/min or on face-mask ventilation.
* Any protocol-defined exclusionary laboratory values at Screening.
* Child-Pugh Class B or C hepatic impairment.
* An acute upper or lower respiratory infection, pulmonary exacerbation, or change in therapy for pulmonary disease within 28 days before Day 1.
* Documentation of colonization with organisms associated with a more rapid decline in pulmonary status.
* History of lung transplantation since most recent initiation of Orkambi.
* History of alcohol or drug abuse in the past year as deemed by the investigator.
* Participation in an investigational drug study or use of a CFTR modulator within 28 days or 5 terminal half-lives of the investigational drug or modulator (whichever is longer).
* Use of restricted medications or foods within the specified window before the first dose of study drug, or an anticipated need or use of restricted medication or foods after the first dose of study drug.
* Pregnant or nursing females: Females of child-bearing potential must have a negative pregnancy test at Screening and Day 1.
12 Years
ALL
No
Sponsors
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Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Locations
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University of Alabama at Birmingham
Birmingham, Alabama, United States
Phoenix Children's Hospital
Phoenix, Arizona, United States
UCSF - Fresno, Community Regional Medical Center
Fresno, California, United States
Miller Children's Hospital / Long Beach Memorial
Long Beach, California, United States
Children's Hospital Los Angeles
Los Angeles, California, United States
Rady Children's Hospital
San Diego, California, United States
National Jewish Health
Denver, Colorado, United States
Yale New Haven Hospital
New Haven, Connecticut, United States
Central Florida Pulmonary Group
Orlando, Florida, United States
Arnold Palmer Hospital
Orlando, Florida, United States
Tampa General Hospital Cardiac and Lung Transplant Clinic
Tampa, Florida, United States
Children's Speciality Services at North Druid Hills
Atlanta, Georgia, United States
St. Luke's CF Center of Idaho
Boise, Idaho, United States
Advocate Children's Hospital - Park Ridge / North Suburban Pulmonary and Critical Care Consultants
Niles, Illinois, United States
Southern Illinois University
Springfield, Illinois, United States
Riley Hospital for Children at Indiana University Health
Indianapolis, Indiana, United States
The University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Kentucky Clinic
Lexington, Kentucky, United States
Tulane Medical Center
New Orleans, Louisiana, United States
Massachusetts General Hospital Cystic Fibrosis Center
Boston, Massachusetts, United States
Boston Children's Hospital
Boston, Massachusetts, United States
University of Mississippi Medical Center
Jackson, Mississippi, United States
Children's Mercy Hospital
Kansas City, Missouri, United States
Billings Clinic
Billings, Montana, United States
Mount Sinai Beth Israel
New York, New York, United States
New York Medical College
Valhalla, New York, United States
Duke University Medical Center
Durham, North Carolina, United States
Wake Forest University Baptist Medical Center
Winston-Salem, North Carolina, United States
Cleveland Clinic Foundation
Cleveland, Ohio, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
Santiago Reyes, M.D.
Oklahoma City, Oklahoma, United States
Drexel University College of Medicine/ Drexel Adult Cystic Fibrosis Center
Philadelphia, Pennsylvania, United States
Medical University of South Carolina
Charleston, South Carolina, United States
Vanderbilt University Medical Center
Nashville, Tennessee, United States
The University of Tennessee Medical Center
Nashville, Tennessee, United States
Dell Children's Medical Center of Central Texas
Austin, Texas, United States
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, United States
University of Utah / Primary Children's Medical Center
Salt Lake City, Utah, United States
Children's Hospital of the King's Daughters
Norfolk, Virginia, United States
Children's Hospital of Richmond at VCU, Children's Pavilion
Richmond, Virginia, United States
Centre Hospitalier Intercommunal de Créteil
Créteil, , France
Centre Hospitalier Universitaire De Grenoble - Hopital Michallon
Grenoble, , France
CHU de Montpellier - Hopital Arnaud de Villeneuve
Montpellier, , France
Centre Hospitalier Universitaire De Nantes
Nantes, , France
Centre hospitalier universitaire de Nice, Pulmonology
Nice, , France
Hopital Cochin
Paris, , France
Centre Hospitalier Universitaire - Hopitaux de Rouen
Rouen, , France
Charite Paediatric Pulmonology Department
Berlin, , Germany
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
Essen, , Germany
Klinikum Innenstadt, University of Munich
München, , Germany
Pneumologische Praxis Pasing
München, , Germany
Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin
Tübingen, , Germany
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2017-000540-18
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX16-661-114
Identifier Type: -
Identifier Source: org_study_id
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