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
A Phase 3b, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
2 other identifiers
interventional
121
10 countries
34
Brief Summary
This study will evaluate the efficacy and safety of elexacaftor (ELX) / tezacaftor (TEZ) / ivacaftor (IVA) triple combination (TC) in subjects 6 through 11 years of age with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function (MF) mutation (F/MF genotypes).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Jun 2020
Shorter than P25 for phase_3
34 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 16, 2020
CompletedFirst Posted
Study publicly available on registry
April 20, 2020
CompletedStudy Start
First participant enrolled
June 19, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 17, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
May 17, 2021
CompletedResults Posted
Study results publicly available
July 26, 2022
CompletedJuly 26, 2022
July 1, 2022
11 months
April 16, 2020
May 16, 2022
July 1, 2022
Conditions
Outcome Measures
Primary Outcomes (1)
Absolute Change in Lung Clearance Index 2.5 (LCI2.5)
The LCI2.5 index is the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting values and is calculated by dividing the sum of exhaled tidal breaths (cumulative exhaled volume (CEV)) by simultaneously measured functional residual capacity (FRC). An LCI of 7.5 and below is normal; values greater than 7.5 are abnormal. LCI is able to detect abnormalities in lung function earlier than more traditional modalities such as spirometry.
From Baseline Through Week 24
Secondary Outcomes (2)
Absolute Change in Sweat Chloride (SwCl)
From Baseline Through Week 24
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Day 1 up to Week 28
Study Arms (2)
Placebo
PLACEBO COMPARATORParticipants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks.
ELX/TEZ/IVA
EXPERIMENTALParticipants 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.
Interventions
Fixed-dose combination tablet for oral administration qd in the morning.
Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning.
Placebo matched to IVA for oral administration qd in the evening.
Eligibility Criteria
You may qualify if:
- Heterozygous for the F508del mutation (F/MF)
- Forced expiratory volume in 1 second (FEV1) value greater than equal to(≥) 70%
You may not qualify if:
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
- Solid organ or hematological transplantation
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (34)
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
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 16, 2020
First Posted
April 20, 2020
Study Start
June 19, 2020
Primary Completion
May 17, 2021
Study Completion
May 17, 2021
Last Updated
July 26, 2022
Results First Posted
July 26, 2022
Record last verified: 2022-07
Data Sharing
- IPD Sharing
- Will not share
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing