Back to resultsA Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
Primary Purpose
Cystic Fibrosis
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
TEZ/IVA
IVA
Placebo
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Cystic Fibrosis
Eligibility Criteria
Key Inclusion Criteria:
- Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
- Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
- Participants with a screening LCI2.5 result ≥7.5.
- Participants 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.
Sites / Locations
- Hunter Medical Research Institute (HMRI)
- Princess Margaret Hospital for Children
- Lady Cilento Children's Hospital
- The Children's Hospital at Westmead
- Universitair Ziekenhuis Brussel - Campus Jette
- Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
- University of Copenhagen Rigshospitalet
- Groupe Hospitalier Pellegrin - Hôpital des Enfants
- Hôpital Necker - Enfants Malades
- Universitaetsklinikum Essen
- Klinikum der Johann Wolfgang Goethe-Universitaet
- Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
- Medizinische Hochschule Hannover
- Universitaetsklinikum Heidelberg
- Universitaetsklinikum Jena
- Universitaetsklinikum Koeln
- Universitaetsklinikum Tuebingen
- Our Lady's Children's Hospital
- University Hospital Limerick
- Klinika Mukowiscydozy, Oddział Chorób Płuc SZP ZOZ
- Inselspital - Universitaetsspital Bern
- Kinderspital Zuerich
- Royal Hospital for Sick Children
- Leeds General Infirmary
- Royal Brompton Hospital
- Nottingham University Hospital City Campus
- Southampton General Hospital
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Other
Experimental
Experimental
Arm Label
Placebo
TEZ/IVA
Ivacaftor
Arm Description
Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks.
Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.
Outcomes
Primary Outcome Measures
Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8
LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
Secondary Outcome Measures
Absolute Change in Sweat Chloride At Week 8
Sweat samples were collected using an approved collection device.
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit
Full Information
NCT ID
NCT03559062
First Posted
June 5, 2018
Last Updated
February 4, 2020
Sponsor
Vertex Pharmaceuticals Incorporated
1. Study Identification
Unique Protocol Identification Number
NCT03559062
Brief Title
A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
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
Study Type
Interventional
2. Study Status
Record Verification Date
February 2020
Overall Recruitment Status
Completed
Study Start Date
May 17, 2018 (Actual)
Primary Completion Date
December 21, 2018 (Actual)
Study Completion Date
December 21, 2018 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants 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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
67 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Placebo
Arm Type
Other
Arm Description
Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks.
Arm Title
TEZ/IVA
Arm Type
Experimental
Arm Description
Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
Arm Title
Ivacaftor
Arm Type
Experimental
Arm Description
Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.
Intervention Type
Drug
Intervention Name(s)
TEZ/IVA
Other Intervention Name(s)
VX-661/VX-770, tezacaftor/ivacaftor fixed dose combination
Intervention Description
Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing ≥40 kg received TEZ 100 mg/IVA 150 mg FDC tablet.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
Participants weighing <40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo matched to TEZ/IVA FDC
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo matched to IVA
Primary Outcome Measure Information:
Title
Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8
Description
LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.
Time Frame
From baseline through Week 8
Secondary Outcome Measure Information:
Title
Absolute Change in Sweat Chloride At Week 8
Description
Sweat samples were collected using an approved collection device.
Time Frame
From baseline at Week 8
Title
Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8
Description
The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.
Time Frame
From baseline through Week 8
Title
Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit
Time Frame
From first dose of study drug up to safety follow-up visit (up to Week 12)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
Participants with a screening LCI2.5 result ≥7.5.
Participants 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.
Facility Information:
Facility Name
Hunter Medical Research Institute (HMRI)
City
New Lambton Heights
Country
Australia
Facility Name
Princess Margaret Hospital for Children
City
Perth
Country
Australia
Facility Name
Lady Cilento Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
The Children's Hospital at Westmead
City
Westmead
Country
Australia
Facility Name
Universitair Ziekenhuis Brussel - Campus Jette
City
Brussels
Country
Belgium
Facility Name
Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg
City
Leuven
Country
Belgium
Facility Name
University of Copenhagen Rigshospitalet
City
Copenhagen
Country
Denmark
Facility Name
Groupe Hospitalier Pellegrin - Hôpital des Enfants
City
Bordeaux Cedex
Country
France
Facility Name
Hôpital Necker - Enfants Malades
City
Paris
Country
France
Facility Name
Universitaetsklinikum Essen
City
Essen
Country
Germany
Facility Name
Klinikum der Johann Wolfgang Goethe-Universitaet
City
Frankfurt
Country
Germany
Facility Name
Universitaetsklinikum Giessen und Marburg GmbH Standort Giessen
City
Giessen
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
Country
Germany
Facility Name
Universitaetsklinikum Heidelberg
City
Heidelberg
Country
Germany
Facility Name
Universitaetsklinikum Jena
City
Jena
Country
Germany
Facility Name
Universitaetsklinikum Koeln
City
Koeln
Country
Germany
Facility Name
Universitaetsklinikum Tuebingen
City
Tuebingen
Country
Germany
Facility Name
Our Lady's Children's Hospital
City
Dublin
Country
Ireland
Facility Name
University Hospital Limerick
City
Limerick
Country
Ireland
Facility Name
Klinika Mukowiscydozy, Oddział Chorób Płuc SZP ZOZ
City
Dziekanow Lesny
Country
Poland
Facility Name
Inselspital - Universitaetsspital Bern
City
Bern
Country
Switzerland
Facility Name
Kinderspital Zuerich
City
Zuerich
Country
Switzerland
Facility Name
Royal Hospital for Sick Children
City
Edinburgh
Country
United Kingdom
Facility Name
Leeds General Infirmary
City
Leeds
Country
United Kingdom
Facility Name
Royal Brompton Hospital
City
London
Country
United Kingdom
Facility Name
Nottingham University Hospital City Campus
City
Nottingham
Country
United Kingdom
Facility Name
Southampton General Hospital
City
Southampton
Country
United Kingdom
12. IPD Sharing Statement
Citations:
PubMed Identifier
33331662
Citation
Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.
Results Reference
derived
Learn more about this trial
A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
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