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

• Register: ClinicalTrials.gov
• Last refreshed on: 20 February 2023
• Main ID: NCT03525574
• Date of registration: 02/05/2018
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A 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 Homozygous or Heterozygous for the F508del Mutation
• Date of first enrolment: October 9, 2018
• Target sample size: 507
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03525574
• 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, Austria, Belgium, Canada, Czechia, France, Germany, Greece, Italy, Netherlands, Sweden, United Kingdom, United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Completed study drug treatment in a parent study; or had study drug interruption(s) in
a parent study but completed study visits up to the last scheduled visit of the
Treatment Period in the parent study.

Exclusion Criteria:

- History of drug intolerance in a parent study that would pose an additional risk to
the subject in the opinion of the investigator.

- Current participation in an investigational drug trial (other than a 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: IVA

Drug: VX-445

Drug: TEZ

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 through safety follow-up (up to 196 weeks)]

Secondary Outcome(s)

Absolute change in sweat chloride (SwCl) [Time Frame: from baseline through last dose of study drug (up to 192 weeks)]

Absolute change in BMI z-score [Time Frame: from baseline through last dose of study drug (up to 192 weeks)]

Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [Time Frame: from baseline through last dose of study drug (up to 192 weeks)]

Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) [Time Frame: from baseline through last dose of study drug (up to 192 weeks)]

Absolute change in body weight [Time Frame: from baseline through last dose of study drug (up to 192 weeks)]

Time to first PEx [Time Frame: from baseline through last dose of study drug (up to 192 weeks)]

Absolute change in body mass index (BMI) [Time Frame: from baseline through last dose of study drug (up to 192 weeks)]

Number of pulmonary exacerbations (PEx) [Time Frame: from baseline through last dose of study drug (up to 192 weeks)]

Secondary ID(s)

2018-000185-11

VX17-445-105

Source(s) of Monetary Support

Please refer to primary and secondary sponsors