A Study to Evaluate Safety, Efficacy, and Tolerability of TEZ/IVA in Orkambi® (Lumacaftor/Ivacaftor) -Experienced Subjects With Cystic Fibrosis (CF)

Description

Study VX16-661-114 (Study 114) is a Phase 3b, randomized, double-blind, placebo-controlled, parallel group, multicenter study in subjects aged 12 years and older with CF who are homozygous for the F508del mutation on the CFTR gene and who discontinued treatment with Orkambi due to respiratory symptoms considered related to treatment. This study is designed to evaluate the safety and efficacy of TEZ/IVA.

Study Start Date

April, 13 2017

Estimated Completion Date

June 2018

Interventions

  • Drug: IVA
  • Drug: Placebo
  • Drug: TEZ/IVA

Study ID

Vertex Pharmaceuticals Incorporated -- VX16-661-114

Status

Recruiting

Trial ID

NCT03150719

Study Type

Interventional

Trial Phase

Phase 3

Enrollment Quota

90

Sponsor

Vertex Pharmaceuticals Incorporated

Inclusion Criteria

  • Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures.
  • 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 <8 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.
  • Other protocol defined inclusion criteria could apply.

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 subject.
  • 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.
  • Other protocol defined exclusion criteria could apply.

Gender

All

Ages

12 Years and older

Accepts Healthy Volunteers

No

Study Locations and Contact Information (91)

Study Location Distance Name Phone Email
Massachusetts General Hospital Cystic Fibrosis Center - Boston, Massachusetts 2.8 miles None None None
Massachusetts General Hospital Cystic Fibrosis Center - Boston, Massachusetts 2.8 miles None None None
Massachusetts General Hospital Cystic Fibrosis Center - Boston, Massachusetts 2.8 miles None None None
Massachusetts General Hospital Cystic Fibrosis Center - Boston, Massachusetts 2.8 miles None None None
Boston Childrens Hospital - Boston, Massachusetts 4.5 miles None None None
Boston Childrens Hospital - Boston, Massachusetts 4.5 miles None None None
Boston Childrens Hospital - Boston, Massachusetts 4.5 miles None None None
Boston Childrens Hospital - Boston, Massachusetts 4.5 miles None None None
Yale New Haven Hospital - New Haven, Connecticut 119.2 miles None None None
Yale New Haven Hospital - New Haven, Connecticut 119.2 miles None None None
Yale New Haven Hospital - New Haven, Connecticut 119.2 miles None None None
New York Medical College - Valhalla, New York 164.0 miles None None None
New York Medical College - Valhalla, New York 164.0 miles None None None
New York Medical College - Valhalla, New York 164.0 miles None None None
New York Medical College - Valhalla, New York 164.0 miles None None None
Drexel University College of Medicine Drexel Adult Cystic Fibrosis Center - Philadelphia, Pennsylvania 269.5 miles None None None
Drexel University College of Medicine Drexel Adult Cystic Fibrosis Center - Philadelphia, Pennsylvania 269.5 miles None None None
Drexel University College of Medicine Drexel Adult Cystic Fibrosis Center - Philadelphia, Pennsylvania 269.5 miles None None None
Drexel University College of Medicine Drexel Adult Cystic Fibrosis Center - Philadelphia, Pennsylvania 269.5 miles None None None
Childrens Hospital of the Kings Daughters - Norfolk, Virginia 471.1 miles None None None
Childrens Hospital of the Kings Daughters - Norfolk, Virginia 471.1 miles None None None
Childrens Hospital of the Kings Daughters - Norfolk, Virginia 471.1 miles None None None
Childrens Hospital of the Kings Daughters - Norfolk, Virginia 471.1 miles None None None
Childrens Hospital of Richmond at VCU Childrens Pavilion - Richmond, Virginia 473.4 miles None None None
Childrens Hospital of Richmond at VCU Childrens Pavilion - Richmond, Virginia 473.4 miles None None None
Childrens Hospital of Richmond at VCU Childrens Pavilion - Richmond, Virginia 473.4 miles None None None
Childrens Hospital of Richmond at VCU Childrens Pavilion - Richmond, Virginia 473.4 miles None None None
Kentucky Clinic - Lexington, Kentucky 769.1 miles None None None
Kentucky Clinic - Lexington, Kentucky 769.1 miles None None None
Kentucky Clinic - Lexington, Kentucky 769.1 miles None None None
Kentucky Clinic - Lexington, Kentucky 769.1 miles None None None
Riley Hospital for Children at Indiana University Health - Indianapolis, Indiana 805.0 miles None None None
Riley Hospital for Children at Indiana University Health - Indianapolis, Indiana 805.0 miles None None None
Riley Hospital for Children at Indiana University Health - Indianapolis, Indiana 805.0 miles None None None
Riley Hospital for Children at Indiana University Health - Indianapolis, Indiana 805.0 miles None None None
Medical University of South Carolina - Charleston, South Carolina 821.2 miles None None None
Medical University of South Carolina - Charleston, South Carolina 821.2 miles None None None
Medical University of South Carolina - Charleston, South Carolina 821.2 miles None None None
Medical University of South Carolina - Charleston, South Carolina 821.2 miles None None None
Advocate Childrens Hospital Park Ridge North Suburban Pulmonary and Critical Care Consultants - Niles, Illinois 856.2 miles None None None
Advocate Childrens Hospital Park Ridge North Suburban Pulmonary and Critical Care Consultants - Niles, Illinois 856.2 miles None None None
Advocate Childrens Hospital Park Ridge North Suburban Pulmonary and Critical Care Consultants - Niles, Illinois 856.2 miles None None None
Advocate Childrens Hospital Park Ridge North Suburban Pulmonary and Critical Care Consultants - Niles, Illinois 856.2 miles None None None
Southern Illinois University - Springfield, Illinois 982.1 miles None None None
Southern Illinois University - Springfield, Illinois 982.1 miles None None None
Southern Illinois University - Springfield, Illinois 982.1 miles None None None
Southern Illinois University - Springfield, Illinois 982.1 miles None None None
The University of Iowa Hospitals and Clinics - Iowa City, Iowa 1,050.4 miles None None None
The University of Iowa Hospitals and Clinics - Iowa City, Iowa 1,050.4 miles None None None
The University of Iowa Hospitals and Clinics - Iowa City, Iowa 1,050.4 miles None None None
The University of Iowa Hospitals and Clinics - Iowa City, Iowa 1,050.4 miles None None None
University of Alabama at Birmingham - Birmingham, Alabama 1,051.7 miles None None None
Central Florida Pulmonary Group - Orlando, Florida 1,115.8 miles None None None
Central Florida Pulmonary Group - Orlando, Florida 1,115.8 miles None None None
Central Florida Pulmonary Group - Orlando, Florida 1,115.8 miles None None None
Central Florida Pulmonary Group - Orlando, Florida 1,115.8 miles None None None
Arnold Palmer Hospital - Orlando, Florida 1,118.6 miles None None None
Arnold Palmer Hospital - Orlando, Florida 1,118.6 miles None None None
Arnold Palmer Hospital - Orlando, Florida 1,118.6 miles None None None
Arnold Palmer Hospital - Orlando, Florida 1,118.6 miles None None None
Tampa General Hospital Cardiac and Lung Transplant Clinic - Tampa, Florida 1,186.3 miles None None None
Tampa General Hospital Cardiac and Lung Transplant Clinic - Tampa, Florida 1,186.3 miles None None None
Tampa General Hospital Cardiac and Lung Transplant Clinic - Tampa, Florida 1,186.3 miles None None None
Tampa General Hospital Cardiac and Lung Transplant Clinic - Tampa, Florida 1,186.3 miles None None None
Childrens Mercy Hospital - Kansas City, Missouri 1,249.3 miles None None None
Childrens Mercy Hospital - Kansas City, Missouri 1,249.3 miles None None None
Childrens Mercy Hospital - Kansas City, Missouri 1,249.3 miles None None None
Childrens Mercy Hospital - Kansas City, Missouri 1,249.3 miles None None None
Tulane Medical Center - New Orleans, Louisiana 1,360.0 miles None None None
Tulane Medical Center - New Orleans, Louisiana 1,360.0 miles None None None
Tulane Medical Center - New Orleans, Louisiana 1,360.0 miles None None None
Tulane Medical Center - New Orleans, Louisiana 1,360.0 miles None None None
Santiago Reyes MD - Oklahoma City, Oklahoma 1,495.7 miles None None None
Santiago Reyes MD - Oklahoma City, Oklahoma 1,495.7 miles None None None
Santiago Reyes MD - Oklahoma City, Oklahoma 1,495.7 miles None None None
Santiago Reyes MD - Oklahoma City, Oklahoma 1,495.7 miles None None None
Dell Childrens Medical Center of Central Texas - Austin, Texas 1,691.8 miles None None None
Dell Childrens Medical Center of Central Texas - Austin, Texas 1,691.8 miles None None None
Dell Childrens Medical Center of Central Texas - Austin, Texas 1,691.8 miles None None None
Dell Childrens Medical Center of Central Texas - Austin, Texas 1,691.8 miles None None None
National Jewish Health - Denver, Colorado 1,766.1 miles None None None
National Jewish Health - Denver, Colorado 1,766.1 miles None None None
National Jewish Health - Denver, Colorado 1,766.1 miles None None None
St Lukes CF Center of Idaho - Boise, Idaho 2,256.8 miles None None None
St Lukes CF Center of Idaho - Boise, Idaho 2,256.8 miles None None None
St Lukes CF Center of Idaho - Boise, Idaho 2,256.8 miles None None None
St Lukes CF Center of Idaho - Boise, Idaho 2,256.8 miles None None None
Childrens Hospital Los Angeles - Los Angeles, California 2,595.3 miles None None None
Childrens Hospital Los Angeles - Los Angeles, California 2,595.3 miles None None None
Childrens Hospital Los Angeles - Los Angeles, California 2,595.3 miles None None None
Childrens Hospital Los Angeles - Los Angeles, California 2,595.3 miles None None None

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