A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del

Cystic Fibrosis Clinical Trial

Official title:

A Phase 3b, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04105972
• Other study ID #: VX18-445-109
• Secondary ID: 2019-001735-31
• Status: Completed
• Phase: Phase 3
• Start date: October 3, 2019
• Est. completion date: July 24, 2020

Study information

• Verified date: July 2021
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This study will evaluate the efficacy, safety, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for F508del.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 176
• Est. completion date: July 24, 2020
• Est. primary completion date: July 24, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Key Inclusion Criteria:

• Homozygous for the F508del mutation (F/F)
• Forced expiratory volume in 1 second (FEV1) value =40% and =90% of predicted mean for age, sex, and height

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• ELX/TEZ/IVA
FDC tablet for oral administration.
• TEZ/IVA
Fixed-dose combination (FDC) tablet for oral administration.
• IVA
Mono tablet for oral administration.

Locations

Country	Name	City	State
Australia	The Prince Charles Hospital	Chermside
Australia	Institute for Respiratory Health	Nedlands
Australia	Perth Children's Hospital	Nedlands
Australia	John Hunter Hospital & Hunter Medical Research Institute and John Hunter Children's Hospital	New Lambton
Australia	The Royal Children's Hospital	Parkville, VIC
Australia	Queensland Children's Hospital	South Brisbane
Belgium	Universitair Ziekenhuis Brussel - Campus Jette	Brussels
Belgium	UZ Antwerpen	Edegem
Belgium	Universitair Ziekenhuis Gent	Gent
Belgium	Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg	Leuven
Germany	Charite Paediatric Pulmonology Department	Berlin
Germany	Ruhrlandklinik Westdeutsches Lungenzentrum am Klinikum Essen	Essen
Germany	Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie	Essen
Germany	Johann Wolfgang Goethe University	Frankfurt
Germany	Mukeviszidose-Zentrum am Universitatsklinikum Jena, Klinik fuer Kinder- und Jugendmedizin	Jena
Germany	Universitaetsklinkum Koeln, CF-Studienzentrum	Koeln
Germany	Pneumologisches Studienzentrum Muenchen-West	Muenchen
Germany	Klinikum Innenstadt, University of Munich	München
United Kingdom	Belfast City Hospital	Belfast
United Kingdom	University Hospitals Birmingham NHS Foundation Trust	Birmingham
United Kingdom	University Hospitals Bristol NHS Foundation Trust, Bristol Royal Hospital	Bristol
United Kingdom	Papworth Hospital NHS Foundation Trust, Papworth Everard	Cambridge
United Kingdom	Western General Hospital	Edinburgh
United Kingdom	Royal Devon and Exeter NHS Foundation Trust, Royal Devon and Exeter Hospital	Exeter
United Kingdom	Clinical Research Facility, Queen Elizabeth University Hospital	Glasgow
United Kingdom	St. James University Hospital	Leeds
United Kingdom	Leeds General Infirmary	Leeds, West Yorkshire
United Kingdom	Alder Hey Children's Alder Hey Children's NHS Foundation Trust	Liverpool
United Kingdom	Great Ormond Street Hospital for Sick Children	London
United Kingdom	London and St Bartholomew's Hospital	London
United Kingdom	The University Hospital of South Manchester	Manchester
United Kingdom	The Newcastle upon Tyne Hospitals NHS Foundation Trust, The Royal Victoria Infirmary	Newcastle Upon Tyne
United Kingdom	Nottingham University Hospitals NHS Trust, Queens Medical Center	Nottingham
United Kingdom	All Wales Adult Cystic Fibrosis Centre, University Hospital Llandough	Penarth
United Kingdom	Southampton General Hospital	Southampton

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

Australia,  Belgium,  Germany,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute Change in CF Questionnaire-Revised (CFQ-R) Respiratory Domain Score	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.	From Baseline Through Week 24
Secondary	Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1)	FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.	From Baseline Through Week 24
Secondary	Absolute Change in Sweat Chloride (SwCl)	Sweat samples were collected using an approved collection device.	From Baseline Through Week 24
Secondary	Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)		From Day 1 in the Treatment Period up to 28 Days After Last Dose of Study Drug or to the Completion of Study Participation Date, Whichever Occurs First (up to Week 28)