A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 30 August 2021
• Main ID: NCT03911713
• Date of registration: 09/04/2019
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Phase 2 Study to Evaluate Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
• Scientific title: A Phase 2, Randomized, Double-blind Study to Evaluate the Efficacy and Safety of VX-561 in Subjects Aged 18 Years and Older With Cystic Fibrosis
• Date of first enrolment: April 17, 2019
• Target sample size: 77
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03911713
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Australia, Belgium, Germany, Ireland, Netherlands, United Kingdom, United States

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- Must have 1 of the following 9 CFTR mutations on at least 1 allele: G551D, G178R,
S549N, S549R, G551S, G1244E, S1251N, S1255P, or G1349D

- On ivacaftor therapy

- FEV1 value =40% and =100% of predicted mean for age, sex, and height

Key Exclusion Criteria:

- History of clinically significant cirrhosis with or without portal hypertension

- History of solid organ or hematological transplantation

- Lung infection with organisms associated with a more rapid decline in pulmonary status

Other protocol defined Inclusion/Exclusion criteria may apply



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: IVA

Drug: Placebo

Drug: VX-561

Primary Outcome(s)

Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [Time Frame: From Baseline at Week 12]

Secondary Outcome(s)

Absolute change in sweat chloride concentrations [Time Frame: From Baseline at Week 12]

Maximum observed concentration (Cmax) of VX-561, IVA, and relevant metabolites [Time Frame: From Baseline up to Week 16]

Observed pre-dose concentration (Ctrough) of VX-561, IVA, and relevant metabolites [Time Frame: From Baseline up to Week 16]

Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-561, IVA, and relevant metabolites [Time Frame: From Baseline up to Week 16]

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 16]

Secondary ID(s)

2018-003970-28

VX18-561-101

Source(s) of Monetary Support

Please refer to primary and secondary sponsors