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A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03559062
Recruitment Status : Completed
First Posted : June 15, 2018
Last Update Posted : January 29, 2019
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in subjects with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 69 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Actual Study Start Date : May 17, 2018
Actual Primary Completion Date : December 21, 2018
Actual Study Completion Date : December 21, 2018

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Experimental: F/F Subjects: TEZ + IVA Drug: TEZ/IVA
Fixed-dose combination (FDC) tablet (TEZ/IVA)
Other Name: tezacaftor/ivacaftor

Experimental: F/F Subjects: Placebo Drug: Placebo
Matching placebo.

Experimental: F/RF Subjects: TEZ + IVA Drug: TEZ/IVA
Fixed-dose combination (FDC) tablet (TEZ/IVA)
Other Name: tezacaftor/ivacaftor

Experimental: F/RF Subjects: IVA + Placebo Drug: IVA
IVA tablet
Other Name: ivacaftor; VX-770

Drug: Placebo
Matching placebo.

Primary Outcome Measures :
  1. Absolute change in lung clearance index2.5 (LCI2.5) [ Time Frame: from baseline through Week 8 ]

Secondary Outcome Measures :
  1. Absolute change in sweat chloride [ Time Frame: from baseline at Week 8 ]
  2. Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline through Week 8 ]
  3. Safety and tolerability as measured by adverse events (AEs) and non-serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (16 Weeks) ]

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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Ages Eligible for Study:   6 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

  • Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
  • Subjects with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
  • Subjects with a screening LCI2.5 result ≥7.5.
  • Subjects who are able to swallow tablets.

Key Exclusion Criteria:

  • Clinically significant cirrhosis with or without portal hypertension.
  • Colonization with organisms associated with a more rapid decline in pulmonary status.
  • Solid organ or hematological transplantation.

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 identifier (NCT number): NCT03559062

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Hunter Medical Research Institute (HMRI)
New Lambton Heights, Australia
Princess Margaret Hospital for Children
Perth, Australia
Lady Cilento Children's Hospital
South Brisbane, Australia
The Children's Hospital at Westmead
Westmead, Australia
Universitair Ziekenhuis Brussel - Campus Jette
Brussels, Belgium
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
Leuven, Belgium
University of Copenhagen Rigshospitalet
Copenhagen, Denmark
Groupe Hospitalier Pellegrin - Hôpital des Enfants
Bordeaux Cedex, France
Hôpital Necker - Enfants Malades
Paris, France
Universitaetsklinikum Essen
Essen, Germany
Klinikum der Johann Wolfgang Goethe-Universitaet
Frankfurt, Germany
Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
Giessen, Germany
Medizinische Hochschule Hannover
Hannover, Germany
Universitaetsklinikum Heidelberg
Heidelberg, Germany
Universitaetsklinikum Jena
Jena, Germany
Universitaetsklinikum Koeln
Koeln, Germany
Universitaetsklinikum Tuebingen
Tuebingen, Germany
Our Lady's Children's Hospital
Dublin, Ireland
University Hospital Limerick
Limerick, Ireland
Klinika Mukowiscydozy, Oddział Chorób Płuc SZP ZOZ
Dziekanow Lesny, Poland
Inselspital - Universitaetsspital Bern
Bern, Switzerland
Kinderspital Zuerich
Zuerich, Switzerland
United Kingdom
Royal Hospital for Sick Children
Edinburgh, United Kingdom
Leeds General Infirmary
Leeds, United Kingdom
Royal Brompton Hospital
London, United Kingdom
Nottingham University Hospital City Campus
Nottingham, United Kingdom
Southampton General Hospital
Southampton, United Kingdom
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated

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Responsible Party: Vertex Pharmaceuticals Incorporated Identifier: NCT03559062     History of Changes
Other Study ID Numbers: VX16-661-115
2016-004479-35 ( EudraCT Number )
First Posted: June 15, 2018    Key Record Dates
Last Update Posted: January 29, 2019
Last Verified: January 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
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action