Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03691779
• Date of registration: 28/09/2018
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Evaluation of VX 445/TEZ/IVA in Cystic Fibrosis Subjects 6 Through 11 Years of Age
• Scientific title: A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-445/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of Age
• Date of first enrolment: October 2, 2018
• Target sample size: 66
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03691779
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Ireland, United Kingdom, United States

Key inclusion & exclusion criteria

Key Inclusion Criteria:

- Homozygous or heterozygous for F508del mutation (F/F or F/MF genotypes)

- Forced expiratory volume in 1 second (FEV1) value =40% of predicted mean for age, sex,
and height.

Key Exclusion Criteria:

- Clinically significant cirrhosis with or without portal hypertension

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

- Solid organ or hematological transplantation.

Other protocol defined Inclusion/Exclusion criteria may apply.



Age minimum: 6 Years
Age maximum: 11 Years
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: IVA

Drug: VX-445

Drug: TEZ

Primary Outcome(s)

Part B: Safety and tolerability as assessed by number of subjects with adverse events and serious adverse events [Time Frame: from baseline through safety follow-up (28 Weeks)]

Part A: Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-445, TEZ, and IVA [Time Frame: Day 1 through 15]

Part A: Maximum Observed Concentration (Cmax) of VX-445, TEZ, and IVA [Time Frame: Day 1 through 15]

Part A: Observed pre-dose concentration (Ctrough) of VX-445, TEZ, and IVA [Time Frame: Day 1 through 15]

Secondary Outcome(s)

Part A: Area under the concentration versus time curve during a dosing interval (AUCtau) of VX-445, TEZ, and IVA metabolites [Time Frame: from Day 1 through 15]

Part B: Absolute change in body mass index (BMI) and BMI for age-z-score [Time Frame: from baseline at Week 24]

Part B: Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain score [Time Frame: from baseline through Week 24]

Part A: 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 (28 Weeks)]

Part B: Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [Time Frame: from baseline through Week 24]

Part A: Maximum observed concentration (Cmax) of VX-445, TEZ, and IVA metabolites [Time Frame: from Day 1 through 15]

Part B: Absolute change in sweat chloride [Time Frame: from baseline through Week 24]

Part B: Number of CF Related Hospitalizations [Time Frame: from baseline through Week 24]

Parts B: Ctrough of VX-445, TEZ, IVA, and IVA metabolites [Time Frame: Day 1 through Week 24]

Part B: Absolute change in height and height for age-z-score [Time Frame: from baseline at Week 24]

Part B: Absolute change in weight and weight for age-z-score [Time Frame: from baseline at Week 24]

Part B: Number of Pulmonary Exacerbations (PEx) [Time Frame: from baseline through Week 24]

Part A: Observed pre-dose concentration (Ctrough) of VX-445, TEZ, and IVA metabolites [Time Frame: from Day 1 through 15]

Part B: Absolute change in lung clearance index2.5 (LCI2.5) [Time Frame: from baseline through Week 24]

Part B: Absolute change in the Modified Facial Hedonic Scale [Time Frame: from baseline at Week 24]

Secondary ID(s)

VX18-445-106

2018-001695-38

Source(s) of Monetary Support

Please refer to primary and secondary sponsors