Cystic Fibrosis
Conditions
Brief summary
The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
Interventions
DRUGVX-121/TEZ/D-IVA
Fixed-dose combination for oral administration.
Sponsors
Vertex Pharmaceuticals Incorporated
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
1 Years to 11 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: * Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene Key
Exclusion criteria
* History of solid organ, hematological transplantation, or cancer * Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15) * Lung infection with organisms associated with a more rapid decline in pulmonary status Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites | From Day 1 up to Day 22 |
| Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to Day 50 |
| Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) | From Day 1 up to Week 28 |
Secondary
| Measure | Time frame |
|---|---|
| Part B: Absolute Change in Sweat Chloride (SwCl) | From Baseline Through Week 24 |
| Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites | From Day 1 up to Week 16 |
| Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale | At Day 1 and Week 24 |
| Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1) | From Baseline Through Week 24 |
| Part B: Number of Pulmonary Exacerbation (PEx) | From Baseline Through Week 24 |
| Part B: Number of CF-Related Hospitalizations | From Baseline Through Week 24 |
| Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score | From Baseline Through Week 24 |
| Part B: Absolute Change in Body Mass Index (BMI) | From Baseline at Week 24 |
| Part B: Absolute Change in BMI-for-age Z-score | From Baseline at Week 24 |
| Part B: Absolute Change in Weight | From Baseline at Week 24 |
| Part B: Absolute Change in Weight-for-age Z-score | From Baseline at Week 24 |
| Part B: Absolute Change in Weight-for-length | From Baseline at Week 24 |
| Part B: Absolute Change in Weight-for-length Z-score | From Baseline at Week 24 |
| Part B: Absolute Change in Height | From Baseline at Week 24 |
| Part B: Absolute Change in Height-for-age Z-score | From Baseline at Week 24 |
| Part B: Absolute Change in Length | From Baseline at Week 24 |
| Part B: Absolute Change in Length-for-age Z-score | From Baseline at Week 24 |
| Part B: Proportion of Participants With SwCl <60 millimole per liter (mmol/L) | From Baseline Through Week 24 |
| Part B: Proportion of Participants With SwCl <30 mmol/L | From Baseline Through Week 24 |
Countries
Australia, Canada, France, Germany, Netherlands, New Zealand, Sweden, Switzerland, United Kingdom, United States
Outcome results
None listed