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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

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ELX/TEZ/IVA
IVA
Placebo (matched to ELX/TEZ/IVA)
Placebo (matched to IVA)
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

6 Years - 11 Years (Child)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • Heterozygous for the F508del mutation (F/MF)
  • Forced expiratory volume in 1 second (FEV1) value greater than equal to(≥) 70%

Key Exclusion Criteria:

  • 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

Other protocol defined Inclusion/Exclusion criteria may apply

Sites / Locations

  • Telethon Kids Institute
  • Queensland Children's Hospital
  • The Children's Hospital at Westmead
  • McGill University Health Centre, Glen Site, Montreal Children's Hospital
  • The Hospital for Sick Children
  • British Columbia Children's Hospital
  • Juliane Marie Center, Rigshospitalet
  • Groupe Hospitaler Pellegrin, CHU De Bordeaux
  • CHU Lyon - Hopital Femme Mere-Enfant
  • Hopital Necker, Enfants Malades
  • Hopital Robert Debre
  • Centre de Perharidy
  • Charite Paediatric Pulmonology Department
  • Universitätsklinikum Essen
  • Johann Wolfgang Goethe University
  • Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
  • Medizinische Hochschule Hannover
  • Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
  • Universitaetsklinkum Koeln, CF-Studienzentrum
  • Hadassah University Hospital Mount Scopus
  • Schneider Children's Medical Center
  • Universitair Medisch Centrum Groningen
  • Erasmus Medical Center / Sophia Children's Hospital
  • Hospital Universitari Vall d Hebron
  • Hospital Virgen de la Arrixaca
  • Inselspital - Universitaetsspital Bern
  • Kinderspital Zuerich
  • University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
  • Children's Hospital of Wales
  • Royal Hospital for Sick Children
  • Alder Hey Children's NHS Foundation Trust
  • Great Ormond Street Hospital for Sick Children
  • Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
  • Southampton General Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Placebo Comparator

Experimental

Arm Label

Placebo

ELX/TEZ/IVA

Arm Description

Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks.

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.

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Absolute Change in Sweat Chloride (SwCl)
Sweat samples were collected using an approved collection device.
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Full Information

First Posted
April 16, 2020
Last Updated
July 1, 2022
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04353817
Brief Title
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
Official Title
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)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2022
Overall Recruitment Status
Completed
Study Start Date
June 19, 2020 (Actual)
Primary Completion Date
May 17, 2021 (Actual)
Study Completion Date
May 17, 2021 (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
Product Manufactured in and Exported from the U.S.
Yes

5. Study Description

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).

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
121 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Participants received placebo matched to ELX/TEZ/IVA and placebo matched to IVA in the treatment period for 24 weeks.
Arm Title
ELX/TEZ/IVA
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
ELX/TEZ/IVA
Other Intervention Name(s)
VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Intervention Description
Fixed-dose combination tablet for oral administration qd in the morning.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
Tablet for oral administration qd in the evening.
Intervention Type
Other
Intervention Name(s)
Placebo (matched to ELX/TEZ/IVA)
Intervention Description
Placebo matched to ELX/TEZ/IVA for oral administration once daily (qd) in the morning.
Intervention Type
Other
Intervention Name(s)
Placebo (matched to IVA)
Intervention Description
Placebo matched to IVA for oral administration qd in the evening.
Primary Outcome Measure Information:
Title
Absolute Change in Lung Clearance Index 2.5 (LCI2.5)
Description
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.
Time Frame
From Baseline Through Week 24
Secondary Outcome Measure Information:
Title
Absolute Change in Sweat Chloride (SwCl)
Description
Sweat samples were collected using an approved collection device.
Time Frame
From Baseline Through Week 24
Title
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame
Day 1 up to Week 28

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: Heterozygous for the F508del mutation (F/MF) Forced expiratory volume in 1 second (FEV1) value greater than equal to(≥) 70% Key Exclusion Criteria: 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 Other protocol defined Inclusion/Exclusion criteria may apply
Facility Information:
Facility Name
Telethon Kids Institute
City
Nedlands
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
The Children's Hospital at Westmead
City
Westmead
Country
Australia
Facility Name
McGill University Health Centre, Glen Site, Montreal Children's Hospital
City
Montréal
Country
Canada
Facility Name
The Hospital for Sick Children
City
Toronto
Country
Canada
Facility Name
British Columbia Children's Hospital
City
Vancouver
Country
Canada
Facility Name
Juliane Marie Center, Rigshospitalet
City
Copenhagen
Country
Denmark
Facility Name
Groupe Hospitaler Pellegrin, CHU De Bordeaux
City
Bordeaux cedex
Country
France
Facility Name
CHU Lyon - Hopital Femme Mere-Enfant
City
Bron Cedex
Country
France
Facility Name
Hopital Necker, Enfants Malades
City
Paris Cedex 15
Country
France
Facility Name
Hopital Robert Debre
City
Paris
Country
France
Facility Name
Centre de Perharidy
City
Roscoff cedex
Country
France
Facility Name
Charite Paediatric Pulmonology Department
City
Berlin
Country
Germany
Facility Name
Universitätsklinikum Essen
City
Essen
Country
Germany
Facility Name
Johann Wolfgang Goethe University
City
Frankfurt
Country
Germany
Facility Name
Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
City
Gießen
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
Country
Germany
Facility Name
Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
City
Heidelberg
Country
Germany
Facility Name
Universitaetsklinkum Koeln, CF-Studienzentrum
City
Koeln
Country
Germany
Facility Name
Hadassah University Hospital Mount Scopus
City
Jerusalem
Country
Israel
Facility Name
Schneider Children's Medical Center
City
Petah Tikva
Country
Israel
Facility Name
Universitair Medisch Centrum Groningen
City
Groningen
Country
Netherlands
Facility Name
Erasmus Medical Center / Sophia Children's Hospital
City
Rotterdam
Country
Netherlands
Facility Name
Hospital Universitari Vall d Hebron
City
Barcelona
Country
Spain
Facility Name
Hospital Virgen de la Arrixaca
City
Murcia
Country
Spain
Facility Name
Inselspital - Universitaetsspital Bern
City
Bern
Country
Switzerland
Facility Name
Kinderspital Zuerich
City
Zurich
Country
Switzerland
Facility Name
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
City
Bristol
Country
United Kingdom
Facility Name
Children's Hospital of Wales
City
Cardiff
Country
United Kingdom
Facility Name
Royal Hospital for Sick Children
City
Edinburgh
Country
United Kingdom
Facility Name
Alder Hey Children's NHS Foundation Trust
City
Liverpool
Country
United Kingdom
Facility Name
Great Ormond Street Hospital for Sick Children
City
London
Country
United Kingdom
Facility Name
Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
City
London
Country
United Kingdom
Facility Name
Southampton General Hospital
City
Southampton
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
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

Learn more about this trial

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

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