Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis (CF) Participants 2 Years and Older

NCT05153317 | Completed Phase 3 FDA Drug

• 2 other identifiers: VX20-445-1122020-002239-31
• Study Type: interventional
• Target: 71
• Locations: 5 countries
• Sites: 22

Brief Summary

The purpose of this study is to evaluate the long term safety, tolerability, efficacy and pharmacodynamics of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) in CF participants 2 years of age and older.

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 Day 1 up to Week 196

Secondary Outcomes (2)

• Absolute Change in Sweat Chloride (SwCl)

  From Baseline up to Week 192

• Absolute Change in Lung Clearance Index (LCI) 2.5

  From Baseline up to Week 192

Study Arms (1)

ELX/TEZ/IVA

EXPERIMENTAL

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Drug: ELX/TEZ/IVA; Drug: IVA

Interventions

ELX/TEZ/IVA DRUG

Fixed-dose combination granules or tablets for oral administration.

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

ELX/TEZ/IVA

IVA DRUG

Granules or tablets for oral administration.

Also known as: VX-770, ivacaftor

ELX/TEZ/IVA

Eligibility Criteria

• Age: 2 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 the parent study (VX20-445-111 Part B, NCT04537793), or had study drug interruption(s) in the parent study, but did not permanently discontinue study drug, and completed study visits up to the last scheduled visit of the Treatment Period of the parent study

You may not qualify if:

• History of study drug intolerance in the parent study
• History of poor compliance with ELX/TEZ/IVA and/or procedures in the parent study

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead

Study Sites (22)

Banner University of Arizona Medical Center

Tucson, Arizona, 85724, United States

Location

Stanford University

Palo Alto, California, 94304, United States

Location

Children's Hospital of Colorado

Aurora, Colorado, 80045, United States

Location

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

Riley Hospital for Children at Indiana University Health

Indianapolis, Indiana, 46202, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States

Location

The Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Washington University School of Medicine / St. Louis Children's Hospital

St Louis, Missouri, 63110, United States

Location

NC TraCS Institute - CTRC University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, 15224, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Telethon Kids Institute

Nedlands, Australia

Location

The Royal Children's Hospital

Parkville, Australia

Location

Queensland Children's Hospital

South Brisbane, Australia

Location

The Hospital for Sick Children

Toronto, Canada

Location

British Columbia Children's Hospital

Vancouver, Canada

Location

Charite Paediatric Pulmonology Department

Berlin, Germany

Location

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

Essen, Germany

Location

Alder Hey Children's NHS Foundation Trust

Liverpool, United Kingdom

Location

Royal Brompton Hospital

London, United Kingdom

Location

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

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: November 29, 2021
• First Posted: December 10, 2021
• Study Start: January 17, 2022
• Primary Completion: January 29, 2026
• Study Completion: January 29, 2026
• Last Updated: March 3, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (13); DE (2); AU (3); GB (2); CA (2)