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

Phase 3

Active, not recruiting

• Conditions: Cystic Fibrosis
• Interventions: Drug: ELX/TEZ/IVA; Drug: IVA

• Registration Number: NCT05153317
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2021-11-29
• Study First Submitted QC: 2021-11-29
• Study First Posted: 2021-12-10
• Study Start: 2022-01-17
• Status Verified: 2024-03
• Last Update Submitted: 2024-08-20
• Last Update Posted: 2024-08-21
• Primary Completion: 2026-04
• Study Completion: 2026-04

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 71

Inclusion Criteria

• 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

Key

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Exclusion Criteria

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

Other protocol defined Inclusion/Exclusion criteria may apply

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (22)

Ann & Robert H. Lurie Children's Hospital of Chicago; 🇺🇸 Chicago, Illinois, United States

UPMC Children's Hospital of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States

Riley Hospital for Children at Indiana University Health; 🇺🇸 Indianapolis, Indiana, United States

Banner University of Arizona Medical Center; 🇺🇸 Tucson, Arizona, United States

British Columbia Children's Hospital; 🇨🇦 Vancouver, Canada

NC TraCS Institute - CTRC University of North Carolina at Chapel Hill; 🇺🇸 Chapel Hill, North Carolina, United States

Seattle Children's Hospital; 🇺🇸 Seattle, Washington, United States

Telethon Kids Institute; 🇦🇺 Nedlands, Australia

Queensland Children's Hospital; 🇦🇺 South Brisbane, Australia

The Royal Children's Hospital; 🇦🇺 Parkville, Australia

Alder Hey Children's NHS Foundation Trust; 🇬🇧 Liverpool, United Kingdom

Stanford University; 🇺🇸 Palo Alto, California, United States

Washington University School of Medicine / St. Louis Children's Hospital; 🇺🇸 Saint Louis, Missouri, United States

Children's Hospital of Colorado; 🇺🇸 Aurora, Colorado, United States

Charite Paediatric Pulmonology Department; 🇩🇪 Berlin, Germany

The Children's Mercy Hospital; 🇺🇸 Kansas City, Missouri, United States

The Hospital for Sick Children; 🇨🇦 Toronto, Canada

Universitatsklinikum Essen (AoR), Kinderklinik III, Abt. fur Pneumologie; 🇩🇪 Essen, Germany

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States

Royal Brompton Hospital; 🇬🇧 London, United Kingdom