Clinical trial NCT04545515: 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
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
First posted: 2020-09-11
Enrollment: 120
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: 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

Primary outcomes:
measure: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: From Baseline up to Week 100
Secondary outcomes:
measure: Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)
timeFrame: From Parent Study Baseline to Week 96
measure: Absolute Change From Parent Study Baseline in Lung Clearance Index 2.5 (LCI2.5)
timeFrame: From Parent Study Baseline to Week 96
Other outcomes:
Locations:
city: Nedlands
country: Australia
facility: Telethon Kids Institute
city: South Brisbane
country: Australia
facility: Queensland Children's Hospital
city: Westmead
country: Australia
facility: The Children's Hospital at Westmead
city: Montreal
country: Canada
facility: McGill University Health Centre, Glen Site, Montreal Children's Hospital
city: Toronto
country: Canada
facility: The Hospital for Sick Children
city: Vancouver
country: Canada
facility: British Columbia Children's Hospital
city: Copenhagen
country: Denmark
facility: Juliane Marie Center, Rigshospitalet
city: Bordeaux cedex
country: France
facility: Groupe Hospitaler Pellegrin, CHU De Bordeaux
city: Bron Cedex
country: France
facility: CHU Lyon - Hopital Femme Mere-Enfant
city: Paris Cedex 15
country: France
facility: Hopital Necker, Enfants Malades
city: Paris
country: France
facility: Hopital Robert Debre
city: Roscoff cedex
country: France
facility: Centre de Perharidy
city: Berlin
country: Germany
facility: Charite Paediatric Pulmonology Department
city: Essen
country: Germany
facility: Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie
city: Frankfurt
country: Germany
facility: Johann Wolfgang Goethe University
city: Gießen
country: Germany
facility: Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Heidelberg
country: Germany
facility: Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin
city: Koeln
country: Germany
facility: Universitaetsklinkum Koeln, CF-Studienzentrum
city: Jerusalem
country: Israel
facility: Hadassah University Hospital Mount Scopus
city: Petach Tikvah
country: Israel
facility: Schneider Children's Medical Center of Israel
city: Groningen
country: Netherlands
facility: Universitair Medisch Centrum Groningen
city: Rotterdam
country: Netherlands
facility: Erasmus Medical Center / Sophia Children's Hospital
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d Hebron
city: Murcia
country: Spain
facility: Hospital Virgen de la Arrixaca
city: Bern
country: Switzerland
facility: Inselspital - Universitaetsspital Bern
city: Zurich
country: Switzerland
facility: Kinderspital Zuerich
city: Bristol
country: United Kingdom
facility: University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital
city: Cardiff
country: United Kingdom
facility: Children's Hospital of Wales
city: Edinburgh
country: United Kingdom
facility: Royal Hospital for Sick Children
city: Liverpool
country: United Kingdom
facility: Alder Hey Children's NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Sick Children
city: London
country: United Kingdom
facility: Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
Study officials:
References:
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.