A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03447262
• Date of registration: 21/02/2018
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
• Scientific title: A Phase 3, Open-label Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
• Date of first enrolment: July 13, 2018
• Target sample size: 484
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03447262
• 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, Canada, Denmark, Germany, Ireland, Israel, Poland, Spain, Switzerland, 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: VX-659

Drug: TEZ

Drug: IVA

Primary Outcome(s)

Safety and tolerability of long-term treatment with VX-659 in TC with TEZ and IVA based on adverse events (AEs) [Time Frame: from baseline through safety follow-up (up to 100 weeks)]

Secondary Outcome(s)

Absolute change from baseline in ppFEV1 [Time Frame: from baseline through last dose of study drug (up to 96 weeks)]

Absolute change in body mass index (BMI) [Time Frame: from baseline through last dose of study drug (up to 96 weeks)]

Number of pulmonary exacerbations (PEx) [Time Frame: from baseline through last dose of study drug (up to 96 weeks)]

Absolute change in BMI z-score [Time Frame: from baseline through last dose of study drug (up to 96 weeks)]

Absolute change in sweat chloride (SwCl) [Time Frame: from baseline through last dose of study drug (up to 96 weeks)]

Absolute change from baseline in CFQ-R respiratory domain score [Time Frame: from baseline through last dose of study drug (up to 96 weeks)]

Absolute change in body weight [Time Frame: from baseline through last dose of study drug (up to 96 weeks)]

Time to first PEx [Time Frame: from baseline through last dose of study drug (up to 96 weeks)]

Secondary ID(s)

VX17-659-105

2017-004134-29

Source(s) of Monetary Support

Please refer to primary and secondary sponsors