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A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation (TRANSPORT)

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01807949
First received: March 4, 2013
Last updated: July 23, 2014
Last verified: July 2014
  Purpose

The purpose of this study is to evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in persons 12 years and older with Cystic Fibrosis who are homozygous for the F508del mutation.


Condition Intervention Phase
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Drug: Ivacaftor
Drug: Lumacaftor
Drug: Ivacaftor Placebo
Drug: Lumacaftor Placebo
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

Resource links provided by NLM:


Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Relative change in percent predicted FEV1 [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in body mass index (BMI) [ Time Frame: From Baseline through Week 24 ] [ Designated as safety issue: No ]
  • Number of pulmonary exacerbations [ Time Frame: Through Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in Cystic Fibrosis Questionnaire - Revised (CFQ R) respiratory domain score [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in BMI z-score [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in body weight [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Time-to-first pulmonary exacerbation [ Time Frame: Through Week 24 ] [ Designated as safety issue: No ]
  • Event of having at least 1 pulmonary exacerbation [ Time Frame: Through Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in EuroQol 3 Level (EQ 5D 3L) score [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in Treatment Satisfaction Questionnaire for Medication (TSQM) domains [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values (hematology, serum chemistry, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), vital signs, and pulse oximetry [ Time Frame: From Baseline and up to 28 Weeks ] [ Designated as safety issue: Yes ]
  • PK parameters of lumacaftor, M28 lumacaftor, ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: Baseline and Week 16 ] [ Designated as safety issue: No ]

Enrollment: 563
Study Start Date: March 2013
Study Completion Date: April 2014
Primary Completion Date: April 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Treatment Arm 1
Subjects randomized to study drug will take 600 mg of lumacaftor once daily (qd) and 250 mg of ivacaftor every 12 hours (q12h)
Drug: Ivacaftor
Other Name: VX-770
Drug: Lumacaftor
Other Name: VX-809
Experimental: Treatment Arm 2
Subjects randomized to study drug will take 400 mg of lumacaftor every 12 hours (q12h) and 250 mg of ivacaftor every 12 hours (q12h)
Drug: Ivacaftor
Other Name: VX-770
Drug: Lumacaftor
Other Name: VX-809
Experimental: Treatment Arm 3
Subjects randomized to placebo will remain on placebo through 24 weeks. Subjects will be given tablets that match both lumacaftor and ivacaftor and will follow the same dosing regimen as subjects receiving the study drug
Drug: Ivacaftor Placebo Drug: Lumacaftor Placebo

  Eligibility

Ages Eligible for Study:   12 Years to 65 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Males and females, aged 12 years or older on the date of informed consent or, where appropriate, date of assent
  • Confirmed diagnosis of CF
  • Homozygous for the F508del CFTR mutation
  • FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height
  • Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the Safety Follow up Visit

Exclusion Criteria:

  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before first dose of study drug
  • 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 , including consumption of certain herbal medications (e.g., St. John's Wort) and certain fruit and fruit juices within 14 days before Day 1 of dosing
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01807949

  Hide Study Locations
Locations
United States, California
Oakland, California, United States
Sacramento, California, United States
United States, Colorado
Aurora, Colorado, United States
United States, Connecticut
Hartford, Connecticut, United States
New Haven, Connecticut, United States
United States, Florida
Jacksonville, Florida, United States
Miami, Florida, United States
United States, Indiana
Indianapolis, Indiana, United States
United States, Iowa
Iowa City, Iowa, United States
United States, Kansas
Kansas City, Kansas, United States
United States, Kentucky
Lexington, Kentucky, United States
United States, Maine
Portland, Maine, United States
United States, Massachusetts
Worcester, Massachusetts, United States
United States, Michigan
Detroit, Michigan, United States
Grand Rapids, Michigan, United States
United States, Minnesota
Minneapolis, Minnesota, United States
United States, Mississippi
Jackson, Mississippi, United States
United States, Missouri
St. Louis, Missouri, United States
United States, Nebraska
Omaha, Nebraska, United States
United States, New Jersey
Morristown, New Jersey, United States
New Brunswick, New Jersey, United States
United States, New Mexico
Alburquerque, New Mexico, United States
United States, New York
Albany, New York, United States
Buffalo, New York, United States
New York, New York, United States
Rochester, New York, United States
Syracuse, New York, United States
United States, Ohio
Cleveland, Ohio, United States
Columbus, Ohio, United States
Toledo, Ohio, United States
United States, Oklahoma
Oklahoma City, Oklahoma, United States
United States, Pennsylvania
Philadelphia, Pennsylvania, United States
Pittsburgh, Pennsylvania, United States
United States, South Carolina
Charleston, South Carolina, United States
United States, South Dakota
Sioux Falls, South Dakota, United States
United States, Tennessee
Memphis, Tennessee, United States
Nashville, Tennessee, United States
United States, Texas
Dallas, Texas, United States
Fort Worth, Texas, United States
Houston, Texas, United States
San Antonio, Texas, United States
United States, Utah
Salt Lake City, Utah, United States
United States, Virginia
Richmond, Virginia, United States
United States, Washington
Seattle, Washington, United States
Spokane, Washington, United States
United States, Wisconsin
Milwaukee, Wisconsin, United States
Australia, Queensland
Brisbane, Queensland, Australia
Chermside, Queensland, Australia
Herston, Queensland, Australia
South Brisbane, Queensland, Australia
Australia, Western Australia
Nedlands, Western Australia, Australia
Subiaco, Western Australia, Australia
Austria
Innsbruck, Austria
Wels, Austria
Belgium
Bruxelles, Belgium
Gent, Belgium
Leuven, Belgium
Liège, Belgium
Canada, Alberta
Calgary, Alberta, Canada
Edmonton, Alberta, Canada
Canada, British Columbia
Vancouver, British Columbia, Canada
Canada, Quebec
Montreal, Quebec, Canada
Denmark
København Ø, Denmark
France
Marseille, Bouches-du-Rhône, France
Toulouse cedex 9, Haute Garonne, France
Montpellier cedex 5, Herault, France
Lille, Nord, France
Bordeaux Cedex, France
Paris, France
Germany
Muenchen, Bayern, Germany
Frankfurt, Hessen, Germany
Giessen, Hessen, Germany
Hannover, Niederachsen, Germany
Bochum, Nordrhein Westfalen, Germany
Jena, Thueringen, Germany
Spain
Barcelona, Spain
Valencia, Spain
United Kingdom
Bristol, Avon, United Kingdom
London, Greater London, United Kingdom
Liverpool, Merseyside, United Kingdom
Newcastle upon Tyne, Tyne & Wear, United Kingdom
Leeds, West Yorkshire, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  More Information

No publications provided

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01807949     History of Changes
Other Study ID Numbers: VX12-809-104
Study First Received: March 4, 2013
Last Updated: July 23, 2014
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Spain: Ministry of Health and Consumption
Belgium: Federal Agency for Medicinal Products and Health Products
Denmark: Danish Medicines Agency
Austria: Austrian Medicines and Medical Devices Agency
Germany: Federal Institute for Drugs and Medical Devices
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Australia: Department of Health and Ageing Therapeutic Goods Administration

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Digestive System Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases

ClinicalTrials.gov processed this record on November 20, 2014