Cystic Fibrosis
Conditions
Brief summary
This study will evaluate the pharmacokinetics (PK), safety, tolerability, pharmacodynamics (PD), and efficacy of ELX/TEZ/IVA in CF subjects 12 to less than (\<) 24 months of age.
Interventions
DRUGELX/TEZ/IVA
Fixed-dose combination granules for oral administration.
DRUGIVA
Granules 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
12 Months to 24 Months
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: Participants who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or another ELX/TEZ/IVA-responsive CFTR mutation Key
Exclusion criteria
History of any illness or any clinical condition that, in the opinion of the investigator, might either confound the results of the study or pose an additional risk in administering study drug(s) to the participant Other protocol defined Inclusion/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites | Day 1 up to Day 15 |
| Part A: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Day 43 |
| Part B: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) | Day 1 up to Week 28 |
Secondary
| Measure | Time frame |
|---|---|
| Part B: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites | Day 15 up to Week 16 |
| Part B: Absolute Change in Sweat Chloride (SwCl) | From Baseline Through Week 24 |
Countries
Australia, Canada, Denmark, Germany, Netherlands, Switzerland, United Kingdom
Outcome results
None listed