A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-659/TEZ/IVA Triple Combination Therapy in Cystic Fibrosis Subjects 6 Through 11 Years of Age
Trial Snapshot
- Phase
- Phase 3
- Status
- Terminated
- Enrollment
- 18
- Locations
- 6
- Primary Endpoint
- Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, and IVA
Study Overview
Brief Summary
This study will evaluate the pharmacokinetics (PK), safety, tolerability, efficacy, and pharmacodynamic effect of VX-659, tezacaftor (TEZ), and ivacaftor (IVA) when dosed in triple combination (TC) in Cystic Fibrosis (CF) subjects with F/F or F/MF genotypes.
The study was discontinued after completion of Part A due to Sponsor's discretion.
Study Design
- Study Type
- Interventional
- Intervention Model
- Sequential
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 6 Years to 11 Years (Child)
- Sex
- All
- Accepts Healthy Volunteers
- No
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.
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.
Arms & Interventions
VX-659/TEZ/IVA
Participants who received VX-659 120 milligram (mg)/TEZ 50 mg/ IVA 75 mg as fixed-dose combination (FDC) in the morning and IVA 75 mg as a mono tablet in the evening in the triple combination (TC) treatment period.
Intervention: VX-659/TEZ/IVA (Drug)
VX-659/TEZ/IVA
Participants who received VX-659 120 milligram (mg)/TEZ 50 mg/ IVA 75 mg as fixed-dose combination (FDC) in the morning and IVA 75 mg as a mono tablet in the evening in the triple combination (TC) treatment period.
Intervention: IVA (Drug)
Outcomes
Primary Outcomes
Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, and IVA
Time Frame: Day 8 and Day 15
Maximum Observed Concentration (Cmax) of VX-659, TEZ, and IVA
Time Frame: Day 1 and Day 15
Area Under the Concentration Versus Time Curve From Time 0 to 6 Hours (AUC0-6h) of VX-659, TEZ, and IVA
Time Frame: Day 1 and Day 15
Secondary Outcomes
- Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)(From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to 6 weeks))
- Observed Pre-Dose Concentration (Ctrough) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)(Day 8 and Day 15)
- Maximum Observed Concentration (Cmax) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)(Day 1 and Day 15)
- Area Under the Concentration Versus Time Curve From Time 0 to 6 Hours (AUC0-6h) of TEZ Metabolite (M1-TEZ), and IVA Metabolite (M1-IVA)(Day 1 and Day 15)