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A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Subjects With an F508del CFTR Mutation

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ClinicalTrials.gov Identifier: NCT03537651
Recruitment Status : Active, not recruiting
First Posted : May 25, 2018
Last Update Posted : March 1, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This study will evaluate the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in subjects with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del mutation.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: TEZ Drug: IVA Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 130 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation
Actual Study Start Date : April 25, 2018
Estimated Primary Completion Date : December 2020
Estimated Study Completion Date : December 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arm Intervention/treatment
Experimental: TEZ + IVA

Subjects <40 kg will receive 50 mg TEZ/ 75 mg IVA as a FDC tablet in the morning and 75 mg IVA as a mono tablet in the evening.

Subjects >=40 kg will receive 100 mg TEZ/ 150 mg IVA FDC tablet in the morning and 150 mg IVA as a mono tablet in the evening.

Drug: TEZ
Fixed-dose Combination (FDC) tablet (TEZ/IVA)
Other Name: tezacaftor; VX-661

Drug: IVA
FDC tablet (TEZ/IVA)
Other Name: ivacaftor; VX-770




Primary Outcome Measures :
  1. Safety and tolerability of long-term TEZ/IVA treatment based on adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (up to 28 days after Last Dose at Week 96) ]

Secondary Outcome Measures :
  1. Absolute change in lung clearance index2.5 (LCI2.5) [ Time Frame: from baseline through 96 weeks ]
  2. Absolute change in sweat chloride [ Time Frame: from baseline through 96 weeks ]
  3. Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline through 96 weeks ]
  4. Absolute change in body mass index (BMI) [ Time Frame: from baseline through 96 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Completed the Week 24 Visit in Study 113 Part B or the Week 8 Visit in Study 115.
  • Eligible CFTR Mutation.

Exclusion Criteria:

  • Pregnant and nursing females.
  • History of poor compliance with study drug and/or procedures in a previous study as deemed by the investigator.
  • Ongoing participation in another study with investigational drug.

Other protocol defined Inclusion/Exclusion criteria may apply.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03537651


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Locations
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United States, Alabama
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35233
United States, Alaska
Providence Alaska Medical Center
Anchorage, Alaska, United States, 99508
United States, Arkansas
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
United States, California
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
United States, Delaware
Nemours/ Alfred I. duPont Hospital for Children
Wilmington, Delaware, United States, 19803
United States, Florida
Johns Hopkins All Children's Hospital Outpatient Care Center
Saint Petersburg, Florida, United States, 33701
United States, Georgia
Children's Speciality Services at North Druid Hills
Atlanta, Georgia, United States, 30324
United States, Idaho
St. Luke's CF Center of Idaho
Boise, Idaho, United States, 83702
United States, Indiana
Riley Hospital for Children Indiana University Health
Indianapolis, Indiana, United States, 46202
United States, Massachusetts
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
United States, Minnesota
Children's Hospital & Clinics of Minnesota
Minneapolis, Minnesota, United States, 55404
United States, Missouri
The Children's Mercy Hospital
Kansas City, Missouri, United States, 64108
United States, New Hampshire
Dartmouth Hitchcock Medical Center
Manchester, New Hampshire, United States, 03756
United States, New York
UBMD Pediatrics/ CF Center of Western New York
Buffalo, New York, United States, 14203
Columbia University Medical Center
New York, New York, United States, 10032
SUNY Upstate Medical University
Syracuse, New York, United States, 13202
United States, North Carolina
Wake Forest Baptist Health
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
Rainbow Babies and Children's Hospital/University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44106
United States, Pennsylvania
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
United States, South Carolina
Medical University of South Carolina (MUSC)
Charleston, South Carolina, United States, 29425
United States, South Dakota
Sanford Children's Speciality Clinic
Sioux Falls, South Dakota, United States, 57105
United States, Texas
Austin Children's Chest Associates
Austin, Texas, United States, 78723
Cook Children's Medical Center
Fort Worth, Texas, United States, 76104
Baylor College of Medicine
Houston, Texas, United States, 77030
United States, Virginia
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23507
United States, Washington
Seattle Children's Hospital
Seattle, Washington, United States, 98105
United States, Wisconsin
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States, 53226
Australia
Perth Children's Hospital
Nedlands, Australia
John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital
New Lambton, Australia
Lady Cilento Children's Hospital
South Brisbane, Australia
The Children's Hospital at Westmead
Westmead, Australia
Belgium
Universitair Ziekenhuis Brussel - Campus Jette
Brussels, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
Canada, British Columbia
British Columbia's Children's Hospital
Vancouver, British Columbia, Canada
Canada, Ontario
The Hospital for Sick Children
Toronto, Ontario, Canada
Canada, Quebec
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal, Quebec, Canada
Denmark
Juliane Marie Center, Rigshopitalet
Copenhagen, Denmark
France
Groupe Hospitaler Pellegrin, CHU De Bordeaux
Bordeaux cedex, France
Hopital Necker, Enfants Malades
Paris Cedex 15, France
Germany
Universitätsklinikum Essen
Essen, Germany
Clinic of J.W. Goethe University
Frankfurt, Germany
Justus-Leibig-Universitat Zentrum fur Kinderheilkunde und Jugendmedizin
Giessen, Germany
Medizinische Hochschule Hannover
Hannover, Germany
Universitaetsklinikum Jena, Mukoviszidose-Zentrum
Jena, Germany
Universitaetsklinkum Koeln, CF-Studienzentrum
Koeln, Germany
Universitaetsklinikum Tuebingen Klinik fuer Kinder- und Jugendmedizin
Tuebingen, Germany
Ireland
Our Lady's Children's Hospital
Dublin, Ireland
University Hospital Limerick
Limerick, Ireland
Poland
Klinika Mukowiscydozy IMD Oddozial Chorob Pluc Szpzoz IM. Dzieci WarszaWY
Lomianki, Poland
Switzerland
Inselspital - Universitaetsspital Bern
Bern, Switzerland
Kinderspital Zuerich
Zürich, Switzerland
United Kingdom
Leeds General Infirmary
Leeds, United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
London, United Kingdom
Southampton General Hospital
Southampton, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03537651     History of Changes
Other Study ID Numbers: VX17-661-116
2017-002968-40 ( EudraCT Number )
First Posted: May 25, 2018    Key Record Dates
Last Update Posted: March 1, 2019
Last Verified: February 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action