Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03768089
• Date of registration: 05/12/2018
• Prospective Registration: No
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis
• Scientific title: A Phase 1/2 Study of VX-121 in Healthy Subjects and in Subjects With Cystic Fibrosis
• Date of first enrolment: March 20, 2018
• Target sample size: 114
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03768089
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 1/Phase 2

Countries of recruitment

Netherlands, United Kingdom

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- Part A, B, and C: Healthy Volunteers

- Female subjects must be of non-childbearing potential

- Between the ages of 18 and 55 years, inclusive

- Body mass index (BMI) of 18.0 to 32.0 kg/m2, inclusive, and a total body weight
>50 kg

- Part D: Subjects with CF

- Heterozygous for F508del and an MF mutation (F/MF)

- FEV1 value =40% and =90% of predicted mean for age, sex, and height

- Body weight =35 kg

Key Exclusion Criteria:

- Part A, B and C: Healthy Volunteers

- Any condition possibly affecting drug absorption

- History of febrile illness or other acute illness within 5 days before the first
study drug dose

- Part D: Subjects with CF

- 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: VX-121

Drug: Matched Placebo

Drug: IVA

Drug: TEZ/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 Day 56]

Secondary Outcome(s)

Part D only: Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [Time Frame: From baseline through Day 29]

Maximum observed concentration (Cmax) of VX-121,TEZ and metabolites, IVA and metabolites [Time Frame: From Day 1 up to Day 29]

Observed pre-dose concentration (Ctrough) of VX-121, TEZ and metabolites, IVA and metabolites [Time Frame: From Day 1 up to Day 29]

Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-121, TEZ and metabolites, IVA and metabolites [Time Frame: From Day 1 up to Day 29]

Part D only: Absolute change in sweat chloride concentrations [Time Frame: From baseline through Day 29]

Secondary ID(s)

VX17-121-001

2018-000126-55

Source(s) of Monetary Support

Please refer to primary and secondary sponsors