A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
NCT ID: NCT04353817
Last Updated: 2022-07-26
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
121 participants
INTERVENTIONAL
2020-06-19
2021-05-17
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Placebo
Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks.
Placebo (matched to ELX/TEZ/IVA)
Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning.
Placebo (matched to IVA)
Placebo matched to IVA for oral administration qd in the evening.
ELX/TEZ/IVA
Participants weighing less than (\<) 30 kilograms (kg) at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg every 12 hours (q12h) and participants weighing greater than equals to (\>=) 30 kg at screening received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period for 24 weeks.
ELX/TEZ/IVA
Fixed-dose combination tablet for oral administration qd in the morning.
IVA
Tablet for oral administration qd in the evening.
Interventions
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ELX/TEZ/IVA
Fixed-dose combination tablet for oral administration qd in the morning.
IVA
Tablet for oral administration qd in the evening.
Placebo (matched to ELX/TEZ/IVA)
Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning.
Placebo (matched to IVA)
Placebo matched to IVA for oral administration qd in the evening.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Forced expiratory volume in 1 second (FEV1) value greater than equal to(≥) 70%
Exclusion Criteria
* Lung infection with organisms associated with a more rapid decline in pulmonary status
* Solid organ or hematological transplantation
6 Years
11 Years
ALL
No
Sponsors
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Vertex Pharmaceuticals Incorporated
INDUSTRY
Responsible Party
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Locations
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Telethon Kids Institute
Nedlands, , Australia
Queensland Children's Hospital
South Brisbane, , Australia
The Children's Hospital at Westmead
Westmead, , Australia
McGill University Health Centre, Glen Site, Montreal Children's Hospital
Montreal, , Canada
The Hospital for Sick Children
Toronto, , Canada
British Columbia Children's Hospital
Vancouver, , Canada
Juliane Marie Center, Rigshospitalet
Copenhagen, , Denmark
Groupe Hospitaler Pellegrin, CHU De Bordeaux
Bordeaux, , France
CHU Lyon - Hopital Femme Mere-Enfant
Bron, , France
Hopital Necker, Enfants Malades
Paris, , France
Hopital Robert Debre
Paris, , France
Centre de Perharidy
Roscoff, , France
Charite Paediatric Pulmonology Department
Berlin, , Germany
Universitaetsklinkum Koeln, CF-Studienzentrum
Cologne, , Germany
Universitätsklinikum Essen
Essen, , Germany
Johann Wolfgang Goethe University
Frankfurt, , Germany
Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
Giessen, , Germany
Medizinische Hochschule Hannover
Hanover, , Germany
Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
Heidelberg, , Germany
Hadassah University Hospital Mount Scopus
Jerusalem, , Israel
Schneider Children's Medical Center
Petah Tikva, , Israel
Universitair Medisch Centrum Groningen
Groningen, , Netherlands
Erasmus Medical Center / Sophia Children's Hospital
Rotterdam, , Netherlands
Hospital Universitari Vall d Hebron
Barcelona, , Spain
Hospital Virgen de la Arrixaca
Murcia, , Spain
Inselspital - Universitaetsspital Bern
Bern, , Switzerland
Kinderspital Zuerich
Zurich, , Switzerland
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
Bristol, , United Kingdom
Children's Hospital of Wales
Cardiff, , United Kingdom
Royal Hospital for Sick Children
Edinburgh, , United Kingdom
Alder Hey Children's NHS Foundation Trust
Liverpool, , United Kingdom
Great Ormond Street Hospital for Sick Children
London, , United Kingdom
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
London, , United Kingdom
Southampton General Hospital
Southampton, , United Kingdom
Countries
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Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2019-003554-86
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
VX19-445-116
Identifier Type: -
Identifier Source: org_study_id
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