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A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
ELX/TEZ/IVA
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

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • Completed study drug treatment in parent study (VX18-445-109); or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study

Key Exclusion Criteria:

  • History of study drug intolerance in parent study

Other protocol defined Inclusion/Exclusion criteria may apply

Sites / Locations

  • The Prince Charles Hospital
  • Institute for Respiratory Health
  • Telethon Kids Institute, Perth Children's Hospital
  • The Royal Children's Hospital
  • Queensland Children's Hospital
  • Universitair Ziekenhuis Gent
  • Universitair Ziekenhuizen Leuven - Campus Gasthuisberg
  • Charite Paediatric Pulmonology Department
  • Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
  • Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
  • Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin
  • Universitaetsklinkum Koeln, CF-Studienzentrum
  • Klinikum Innenstadt, University of Munich
  • Belfast City Hospital
  • University Hospitals Birmingham NHS Foundation Trust
  • University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
  • Royal Papworth Hospital NHS Foundation Trust
  • Western General Hospital
  • Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
  • Clinical Research Facility, Queen Elizabeth University Hospital
  • Leeds General Infirmary
  • St. James University Hospital
  • Alder Hey Children's NHS Foundation Trust
  • Great Ormond Street Hospital for Sick Children
  • London and St Bartholomew's Hospital
  • The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
  • Nottingham University Hospitals NHS Trust, Queens Medical Center
  • All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
  • Southampton General Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

ELX/TEZ/IVA

Arm Description

Part A: Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks. Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 weeks.

Outcomes

Primary Outcome Measures

Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)

Secondary Outcome Measures

Full Information

First Posted
April 22, 2020
Last Updated
July 11, 2023
Sponsor
Vertex Pharmaceuticals Incorporated
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1. Study Identification

Unique Protocol Identification Number
NCT04362761
Brief Title
A Study Evaluating the Long-Term Safety of Elexacaftor Combination Therapy
Official Title
A Phase 3b Open-label Extension Study Evaluating the Safety of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
May 4, 2020 (Actual)
Primary Completion Date
December 21, 2022 (Actual)
Study Completion Date
December 21, 2022 (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

5. Study Description

Brief Summary
This study evaluate the long-term safety and tolerability of elexacaftor (ELX)/tezacaftor (TEZ)/ ivacaftor (IVA) triple combination (TC) in participants with cystic fibrosis (CF) who are homozygous for F508del.

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
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
172 (Actual)

8. Arms, Groups, and Interventions

Arm Title
ELX/TEZ/IVA
Arm Type
Experimental
Arm Description
Part A: Participants received elexacaftor (ELX) 200 milligram (mg) once daily (qd)/tezacaftor (TEZ)100 mg qd/ivacaftor (IVA)150 mg every 12 hours (q12h) in the treatment period for 48 weeks. Part B: Participants received ELX 200 mg qd/TEZ 100 mg qd/IVA 150 mg q12h in the treatment period up to 86 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 (FDC) tablet for oral administration.
Intervention Type
Drug
Intervention Name(s)
IVA
Other Intervention Name(s)
VX-770, ivacaftor
Intervention Description
Tablet for oral administration.
Primary Outcome Measure Information:
Title
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame
From Day 1 up to Week 52
Title
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame
From Day 1 up to Week 86

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Completed study drug treatment in parent study (VX18-445-109); or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study Key Exclusion Criteria: History of study drug intolerance in parent study Other protocol defined Inclusion/Exclusion criteria may apply.
Facility Information:
Facility Name
The Prince Charles Hospital
City
Chermside
Country
Australia
Facility Name
Institute for Respiratory Health
City
Nedlands
Country
Australia
Facility Name
Telethon Kids Institute, Perth Children's Hospital
City
Nedlands
Country
Australia
Facility Name
The Royal Children's Hospital
City
Parkville, VIC
Country
Australia
Facility Name
Queensland Children's Hospital
City
South Brisbane
Country
Australia
Facility Name
Universitair Ziekenhuis Gent
City
Gent
Country
Belgium
Facility Name
Universitair Ziekenhuizen Leuven - Campus Gasthuisberg
City
Leuven
Country
Belgium
Facility Name
Charite Paediatric Pulmonology Department
City
Berlin
Country
Germany
Facility Name
Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen
City
Essen
Country
Germany
Facility Name
Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
City
Essen
Country
Germany
Facility Name
Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin
City
Jena
Country
Germany
Facility Name
Universitaetsklinkum Koeln, CF-Studienzentrum
City
Koeln
Country
Germany
Facility Name
Klinikum Innenstadt, University of Munich
City
München
Country
Germany
Facility Name
Belfast City Hospital
City
Belfast
Country
United Kingdom
Facility Name
University Hospitals Birmingham NHS Foundation Trust
City
Birmingham
Country
United Kingdom
Facility Name
University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
City
Bristol
Country
United Kingdom
Facility Name
Royal Papworth Hospital NHS Foundation Trust
City
Cambridge
Country
United Kingdom
Facility Name
Western General Hospital
City
Edinburgh
Country
United Kingdom
Facility Name
Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital
City
Exeter
Country
United Kingdom
Facility Name
Clinical Research Facility, Queen Elizabeth University Hospital
City
Glasgow
Country
United Kingdom
Facility Name
Leeds General Infirmary
City
Leeds
Country
United Kingdom
Facility Name
St. James University Hospital
City
Leeds
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
London and St Bartholomew's Hospital
City
London
Country
United Kingdom
Facility Name
The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary
City
Newcastle Upon Tyne
Country
United Kingdom
Facility Name
Nottingham University Hospitals NHS Trust, Queens Medical Center
City
Nottingham
Country
United Kingdom
Facility Name
All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough
City
Penarth
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.
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 Evaluating the Long-Term Safety of Elexacaftor Combination Therapy

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