A Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis (CF) Particpants 6 Years and Older and F/MF Genotypes

Cystic Fibrosis Clinical Trial

Official title:

A Phase 3b Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor Combination Therapy in Cystic Fibrosis Subjects Ages 6 Years and Older Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04545515
• Other study ID #: VX20-445-119
• Secondary ID: 2020-001404-42
• Status: Completed
• Phase: Phase 3
• Start date: January 11, 2021
• Est. completion date: March 24, 2023

Study information

• Verified date: May 2024
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The study evaluates the long-term safety and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination (TC) in participants with CF who are 6 years of age and older with F/MF genotypes.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 120
• Est. completion date: March 24, 2023
• Est. primary completion date: March 24, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

Key Inclusion Criteria:

• Completed study drug treatment in parent study (VX19-445-116, NCT04353817), 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 the parent study Other protocol defined Inclusion/Exclusion criteria may apply

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• ELX/TEZ/IVA
Fixed dose combination (FDC) tablets for oral administration.
• IVA
Tablet for oral administration.

Locations

Country	Name	City	State
Australia	Telethon Kids Institute	Nedlands
Australia	Queensland Children's Hospital	South Brisbane
Australia	The Children's Hospital at Westmead	Westmead
Canada	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
Denmark	Juliane Marie Center, Rigshospitalet	Copenhagen
France	Groupe Hospitaler Pellegrin, CHU De Bordeaux	Bordeaux cedex
France	CHU Lyon - Hopital Femme Mere-Enfant	Bron Cedex
France	Hopital Robert Debre	Paris
France	Hopital Necker, Enfants Malades	Paris Cedex 15
France	Centre de Perharidy	Roscoff cedex
Germany	Charite Paediatric Pulmonology Department	Berlin
Germany	Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie	Essen
Germany	Johann Wolfgang Goethe University	Frankfurt
Germany	Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin	Gießen
Germany	Medizinische Hochschule Hannover	Hannover
Germany	Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin	Heidelberg
Germany	Universitaetsklinkum Koeln, CF-Studienzentrum	Koeln
Israel	Hadassah University Hospital Mount Scopus	Jerusalem
Israel	Schneider Children's Medical Center of Israel	Petach Tikvah
Netherlands	Universitair Medisch Centrum Groningen	Groningen
Netherlands	Erasmus Medical Center / Sophia Children's Hospital	Rotterdam
Spain	Hospital Universitari Vall d Hebron	Barcelona
Spain	Hospital Virgen de la Arrixaca	Murcia
Switzerland	Inselspital - Universitaetsspital Bern	Bern
Switzerland	Kinderspital Zuerich	Zurich
United Kingdom	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

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

Australia,  Canada,  Denmark,  France,  Germany,  Israel,  Netherlands,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)		From Baseline up to Week 100
Secondary	Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)	Sweat samples were collected using an approved collection device.	From Parent Study Baseline to Week 96
Secondary	Absolute Change From Parent Study Baseline 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 Parent Study Baseline to Week 96