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

NCT04545515 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: VX20-445-1192020-001404-42
• Study Type: interventional
• Target: 120
• Locations: 10 countries
• Sites: 34

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Absolute Change From Parent Study Baseline in Sweat Chloride (SwCl)

  From Parent Study Baseline to Week 96

• Absolute Change From Parent Study Baseline in Lung Clearance Index 2.5 (LCI2.5)

  From Parent Study Baseline to Week 96

Study Arms (1)

ELX/TEZ/IVA

EXPERIMENTAL

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.

Drug: ELX/TEZ/IVA; Drug: IVA

Interventions

ELX/TEZ/IVA DRUG

Fixed dose combination (FDC) tablets for oral administration.

Also known as: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor

ELX/TEZ/IVA

IVA DRUG

Tablet for oral administration.

Also known as: VX-770, ivacaftor

ELX/TEZ/IVA

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• History of study drug intolerance in the parent study

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (34)

Telethon Kids Institute

Nedlands, Australia

Location

Queensland Children's Hospital

South Brisbane, Australia

Location

The Children's Hospital at Westmead

Westmead, Australia

Location

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

Montreal, Canada

Location

The Hospital for Sick Children

Toronto, Canada

Location

British Columbia Children's Hospital

Vancouver, Canada

Location

Juliane Marie Center, Rigshospitalet

Copenhagen, Denmark

Location

Groupe Hospitaler Pellegrin, CHU De Bordeaux

Bordeaux, France

Location

CHU Lyon - Hopital Femme Mere-Enfant

Bron, France

Location

Hopital Necker, Enfants Malades

Paris, France

Location

Hopital Robert Debre

Paris, France

Location

Centre de Perharidy

Roscoff, France

Location

Charite Paediatric Pulmonology Department

Berlin, Germany

Location

Universitaetsklinkum Koeln, CF-Studienzentrum

Cologne, Germany

Location

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

Essen, Germany

Location

Johann Wolfgang Goethe University

Frankfurt, Germany

Location

Justus-Liebig-Universität Gießen Zentrum fur Kinderheilkunde und Jugendmedizin

Giessen, Germany

Location

Medizinische Hochschule Hannover

Hanover, Germany

Location

Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin

Heidelberg, Germany

Location

Hadassah University Hospital Mount Scopus

Jerusalem, Israel

Location

Schneider Children's Medical Center of Israel

Petach Tikvah, Israel

Location

Universitair Medisch Centrum Groningen

Groningen, Netherlands

Location

Erasmus Medical Center / Sophia Children's Hospital

Rotterdam, Netherlands

Location

Hospital Universitari Vall d Hebron

Barcelona, Spain

Location

Hospital Virgen de la Arrixaca

Murcia, Spain

Location

Inselspital - Universitaetsspital Bern

Bern, Switzerland

Location

Kinderspital Zuerich

Zurich, Switzerland

Location

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

Bristol, United Kingdom

Location

Children's Hospital of Wales

Cardiff, United Kingdom

Location

Royal Hospital for Sick Children

Edinburgh, United Kingdom

Location

Alder Hey Children's NHS Foundation Trust

Liverpool, United Kingdom

Location

Great Ormond Street Hospital for Sick Children

London, United Kingdom

Location

Royal Brompton & Harefield NHS Foundation Trust, Royal Brompton Hospital

London, United Kingdom

Location

Southampton General Hospital

Southampton, United Kingdom

Location

Related Publications (2)

• 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.

  PMID: 37983082 DERIVED

• 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.

  PMID: 33331662 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

elexacaftor, ivacaftor, tezacaftor drug combination; ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Medical Monitor
• Organization: Vertex Pharmaceuticals Incorporated

medicalinfo@vrtx.com

617-341-6777

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 3, 2020
• First Posted: September 11, 2020
• Study Start: January 11, 2021
• Primary Completion: March 24, 2023
• Study Completion: March 24, 2023
• Last Updated: May 8, 2024
• Results First Posted: May 8, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

ES (2); CH (2); CA (3); AU (3); DE (7); GB (7); NL (2); FR (5); IL (2); DK (1)