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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

NCT ID: NCT04545515

Last Updated: 2024-05-08

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

120 participants

Study Classification

INTERVENTIONAL

Study Start Date

2021-01-11

Study Completion Date

2023-03-24

Brief Summary

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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.

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Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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ELX/TEZ/IVA

Participants 6 to less than \<12 year of age and weighing \<30 kilogram (kg) at Day 1 received ELX 100 milligram (mg)/TEZ 50 mg /IVA 75 mg as fixed dose combination (FDC) tablets in the morning and IVA as mono tablet in the evening and those weighing more than or equal to (≥) 30 kg at Day 1 received ELX 200 mg/TEZ 100 mg /IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 96 weeks. Doses were adjusted upward with subsequent changes in weight. Participants ≥12 years age at Day 1 received ELX 200 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA as mono tablet in the evening for 96 weeks.

Group Type EXPERIMENTAL

ELX/TEZ/IVA

Intervention Type DRUG

Fixed dose combination (FDC) tablets for oral administration.

IVA

Intervention Type DRUG

Tablet for oral administration.

Interventions

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ELX/TEZ/IVA

Fixed dose combination (FDC) tablets for oral administration.

Intervention Type DRUG

IVA

Tablet for oral administration.

Intervention Type DRUG

Other Intervention Names

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VX-445/VX-661/VX-770 elexacaftor/tezacaftor/ivacaftor VX-770 ivacaftor

Eligibility Criteria

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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

Exclusion Criteria

* History of study drug intolerance in the parent study
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Telethon Kids Institute

Nedlands, , Australia

Site Status

Queensland Children's Hospital

South Brisbane, , Australia

Site Status

The Children's Hospital at Westmead

Westmead, , Australia

Site Status

McGill University Health Centre, Glen Site, Montreal Children's Hospital

Montreal, , Canada

Site Status

The Hospital for Sick Children

Toronto, , Canada

Site Status

British Columbia Children's Hospital

Vancouver, , Canada

Site Status

Juliane Marie Center, Rigshospitalet

Copenhagen, , Denmark

Site Status

Groupe Hospitaler Pellegrin, CHU De Bordeaux

Bordeaux, , France

Site Status

CHU Lyon - Hopital Femme Mere-Enfant

Bron, , France

Site Status

Hopital Necker, Enfants Malades

Paris, , France

Site Status

Hopital Robert Debre

Paris, , France

Site Status

Centre de Perharidy

Roscoff, , France

Site Status

Charite Paediatric Pulmonology Department

Berlin, , Germany

Site Status

Universitaetsklinkum Koeln, CF-Studienzentrum

Cologne, , Germany

Site Status

Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie

Essen, , Germany

Site Status

Johann Wolfgang Goethe University

Frankfurt, , Germany

Site Status

Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin

Giessen, , Germany

Site Status

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status

Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin

Heidelberg, , Germany

Site Status

Hadassah University Hospital Mount Scopus

Jerusalem, , Israel

Site Status

Schneider Children's Medical Center of Israel

Petach Tikvah, , Israel

Site Status

Universitair Medisch Centrum Groningen

Groningen, , Netherlands

Site Status

Erasmus Medical Center / Sophia Children's Hospital

Rotterdam, , Netherlands

Site Status

Hospital Universitari Vall d Hebron

Barcelona, , Spain

Site Status

Hospital Virgen de la Arrixaca

Murcia, , Spain

Site Status

Inselspital - Universitaetsspital Bern

Bern, , Switzerland

Site Status

Kinderspital Zuerich

Zurich, , Switzerland

Site Status

University Hospitals Bristol and Weston NHS Foundation Trust, Bristol Royal Hospital

Bristol, , United Kingdom

Site Status

Children's Hospital of Wales

Cardiff, , United Kingdom

Site Status

Royal Hospital for Sick Children

Edinburgh, , United Kingdom

Site Status

Alder Hey Children's NHS Foundation Trust

Liverpool, , United Kingdom

Site Status

Great Ormond Street Hospital for Sick Children

London, , United Kingdom

Site Status

Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital

London, , United Kingdom

Site Status

Southampton General Hospital

Southampton, , United Kingdom

Site Status

Countries

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Australia Canada Denmark France Germany Israel Netherlands Spain Switzerland United Kingdom

References

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Heneghan M, 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. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.

Reference Type DERIVED
PMID: 37983082 (View on PubMed)

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.

Reference Type DERIVED
PMID: 33331662 (View on PubMed)

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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2020-001404-42

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX20-445-119

Identifier Type: -

Identifier Source: org_study_id

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