Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

• Register: ClinicalTrials.gov
• Last refreshed on: 30 January 2023
• Main ID: NCT04058366
• Date of registration: 14/08/2019
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy
• Scientific title: A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
• Date of first enrolment: December 5, 2019
• Target sample size: 251
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT04058366
• 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

Australia, Belgium, Canada, Denmark, France, Germany, Ireland, Italy, Netherlands, Spain, United Kingdom, United States

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- Completed study drug treatment in parent study (VX18-445-104); 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

Key Exclusion Criteria:

- History of study drug intolerance in parent study

Other protocol defined Inclusion/Exclusion criteria may apply



Age minimum: 12 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)

Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [Time Frame: From Baseline up to Week 100]

Secondary Outcome(s)

Absolute change in BMI z-score [Time Frame: From Baseline up to Week 96]

Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [Time Frame: From Baseline up to Week 96]

Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [Time Frame: From Baseline up to Week 96]

Absolute change in body mass index (BMI) [Time Frame: From Baseline up to Week 96]

Absolute change in body weight [Time Frame: From Baseline up to Week 96]

Absolute change in sweat chloride (SwCl) [Time Frame: From Baseline up to Week 96]

Secondary ID(s)

2019-000833-37

VX18-445-110

Source(s) of Monetary Support

Please refer to primary and secondary sponsors