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

• Register: ClinicalTrials.gov
• Last refreshed on: 21 April 2025
• Main ID: NCT05331183
• Date of registration: 08/04/2022
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
• Scientific title: A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes
• Date of first enrolment: November 23, 2022
• Target sample size: 297
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT05331183
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Austria, Belgium, Canada, Czechia, France, Germany, Hungary, Italy, Netherlands, Norway, Poland, Portugal, Spain, Sweden, Switzerland

Key inclusion & exclusion criteria

Key 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.


Age minimum: 6 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: ELX/TEZ/IVA

Drug: IVA

Primary Outcome(s)

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

Secondary Outcome(s)

Part A: Absolute Change in Weight [Time Frame: From Baseline up to Week 96]

Part A: Absolute Change in Body Mass Index (BMI) [Time Frame: From Baseline up to Week 96]

Part A: Number of Pulmonary Exacerbations (PEx) [Time Frame: From Baseline up to Week 96]

Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [Time Frame: From Baseline up to Week 96]

Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1) [Time Frame: From Baseline up to Week 96]

Part A: Absolute Change in Sweat Chloride (SwCl) [Time Frame: From Baseline up to Week 96]

Secondary ID(s)

2021-005914-33

VX21-445-125

2024-515637-14-00

Source(s) of Monetary Support

Please refer to primary and secondary sponsors