A Phase 3, Open-label Study Evaluating the Pharmacokinetics, Safety, and Tolerability of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 12 to Less Than 24 Months of Age
Overview
- Phase
- Phase 3
- Intervention
- IVA
- Conditions
- Cystic Fibrosis
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Enrollment
- 70
- Locations
- 19
- Primary Endpoint
- Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites
- Status
- Completed
- Last Updated
- 6 months ago
Overview
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.
Investigators
Eligibility Criteria
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
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/Exclusion criteria may apply.
Arms & Interventions
Part A
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Intervention: IVA
Part A
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Intervention: ELX/TEZ/IVA
Part B
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening with the dose(s) to be based on the outcome of Part A.
Intervention: ELX/TEZ/IVA
Part B
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening with the dose(s) to be based on the outcome of Part A.
Intervention: IVA
Outcomes
Primary Outcomes
Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites
Time Frame: Day 1 up to Day 15
Part B: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Day 1 up to Week 28
Part A: Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Day 1 up to Day 43
Secondary Outcomes
- 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)