Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

Phase 3

Active, not recruiting

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

• Registration Number: NCT05331183
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator (CFTR) gene mutation.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-04-08
• Study First Submitted QC: 2022-04-08
• Study First Posted: 2022-04-15
• Study Start: 2022-11-23
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-10
• Last Update Posted: 2025-04-15
• Primary Completion: 2027-04-06
• Study Completion: 2027-04-06

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 297

Inclusion Criteria

Part A: Completed study drug treatment in parent study 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

Part B: Completed study drug treatment in Part A or had study drug interruption(s) in Part A but completed study visits up to the last scheduled visit of the treatment period of Part A

Key

Exclusion Criteria

History of study drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ELX/TEZ/IVA	ELX/TEZ/IVA	Part A: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for 96 weeks. Part B: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for an additional 96 weeks.
ELX/TEZ/IVA	IVA	Part A: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for 96 weeks. Part B: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for an additional 96 weeks.

Primary Outcome Measures

Name	Time	Method
Parts A and B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)	Day 1 up to Week 196

Secondary Outcome Measures

Name	Time	Method
Part A: Absolute Change in Weight	From Baseline up to Week 96
Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score	From Baseline up to Week 96
Part A: Absolute Change in Body Mass Index (BMI)	From Baseline up to Week 96
Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1)	From Baseline up to Week 96
Part A: Number of Pulmonary Exacerbations (PEx)	From Baseline up to Week 96
Part A: Absolute Change in Sweat Chloride (SwCl)	From Baseline up to Week 96

Trial Locations

Locations (81)

Medizinische Universität Innsbruck; 🇦🇹 Innsbruck, Austria

Cliniques Universitaires de Bruxelles Hopital Erasme; 🇧🇪 Brussels, Belgium

Universitair Ziekenhuis Brussel - Campus Jette; 🇧🇪 Brussels, Belgium

Universitair Ziekenhuis Antwerpen (UZA) - Antwerp University Hospital; 🇧🇪 Edegem, Belgium

Universitair Ziekenhuis Gent; 🇧🇪 Gent, Belgium

Universitaire Ziekenhuizen Leuven - Campus Gasthuisberg; 🇧🇪 Leuven, Belgium

Cliniques Universitaires Saint-Luc; 🇧🇪 Woluwe-Saint-Lambert, Belgium

University of Alberta Hospital, Edmonton Clinic; 🇨🇦 Edmonton, Canada

Centre Hospitalier de l'Universite de Montreal (CHUM) Hotel-Dieu; 🇨🇦 Montreal, Canada

McGill University Health Centre, Glen Site, Montreal Children's Hospital; 🇨🇦 Montreal, Canada

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