Cystic Fibrosis
Conditions
Brief summary
Part A: PK parameters of VX-121, TEZ, D-IVA, and relevant metabolites, Part A: Safety and tolerability as determined by adverse events (AEs), clinical laboratory values, standard 12 lead ECGs, vital signs, and pulse oximetry, Part B: Safety and tolerability as determined by AEs, clinical laboratory values, standard 12-lead ECGs, vital signs, and pulse oximetry
Detailed description
Part B (ID 1-11): Absolute change in SwCl from baseline through Week 24, PK parameters of VX 121, TEZ, D-IVA, and relevant metabolites, Drug acceptability assessment using Modified Facial Hedonic Scale, Absolute change in ppFEV1 from baseline through Week 24, Number of PEx and CF-related hospitalizations through Week 24, Absolute change in CFQ-R RD score from baseline through Week 24, Absolute change in BMI and BMI-for-age z-score from baseline at Week 24, Absolute change in weight and weight-for-age z-score from baseline at Week 24, Absolute change in height and height-for-age z-score from baseline at Week 24, Proportion of subjects with SwCl <60 mmol/L through Week 24, Proportion of subjects with SwCl <30 mmol/L through Week 24
Interventions
Sponsors
Vertex Pharmaceuticals Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: PK parameters of VX-121, TEZ, D-IVA, and relevant metabolites, Part A: Safety and tolerability as determined by adverse events (AEs), clinical laboratory values, standard 12 lead ECGs, vital signs, and pulse oximetry, Part B: Safety and tolerability as determined by AEs, clinical laboratory values, standard 12-lead ECGs, vital signs, and pulse oximetry | — |
Secondary
| Measure | Time frame |
|---|---|
| Part B (ID 1-11): Absolute change in SwCl from baseline through Week 24, PK parameters of VX 121, TEZ, D-IVA, and relevant metabolites, Drug acceptability assessment using Modified Facial Hedonic Scale, Absolute change in ppFEV1 from baseline through Week 24, Number of PEx and CF-related hospitalizations through Week 24, Absolute change in CFQ-R RD score from baseline through Week 24, Absolute change in BMI and BMI-for-age z-score from baseline at Week 24, Absolute change in weight and weight-for-age z-score from baseline at Week 24, Absolute change in height and height-for-age z-score from baseline at Week 24, Proportion of subjects with SwCl <60 mmol/L through Week 24, Proportion of subjects with SwCl <30 mmol/L through Week 24 | — |
Countries
France, Germany, Netherlands, Sweden
Outcome results
None listed