A Phase 3 Study Evaluating the Safety, Tolerability, and Pharmacokinetics of Elexacaftor/Tezacaftor/Ivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 2 Through 5 Years of Age
Overview
- Phase
- Phase 3
- Intervention
- ELX/TEZ/IVA
- Conditions
- Cystic Fibrosis
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Enrollment
- 83
- Locations
- 22
- Primary Endpoint
- Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
- Status
- Completed
- Last Updated
- 2 years ago
Overview
Brief Summary
This study will evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA) triple combination therapy in CF subjects 2 through 5 years of age.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Homozygous for the F508del mutation or heterozygous for F508del and a minimal function (MF) mutation (F/F or F/MF genotypes)
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
Part A: ELX/TEZ/IVA
Participants weighing greater than or equal to (\>=)14 kilograms (kg) at screening received elexacaftor (ELX) 100 milligrams (mg) once daily (qd)/tezacaftor (TEZ) 50 mg qd/ivacaftor (IVA) 75 mg every 12 hours (q12h) in the treatment period for 15 days.
Intervention: ELX/TEZ/IVA
Part A: ELX/TEZ/IVA
Participants weighing greater than or equal to (\>=)14 kilograms (kg) at screening received elexacaftor (ELX) 100 milligrams (mg) once daily (qd)/tezacaftor (TEZ) 50 mg qd/ivacaftor (IVA) 75 mg every 12 hours (q12h) in the treatment period for 15 days.
Intervention: IVA
Part B: ELX/TEZ/IVA
Participants weighing (\>=)14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h. Participants weighing (\>=)10 kg to less than (\<)14 kg received ELX 80 mg qd/TEZ 40 mg qd/IVA 60 mg once every morning (qAM) and 59.5 mg once every evening (qPM) in the treatment period for 24 weeks.
Intervention: ELX/TEZ/IVA
Part B: ELX/TEZ/IVA
Participants weighing (\>=)14 kg at screening received ELX 100 mg qd/TEZ 50 mg qd/IVA 75 mg q12h. Participants weighing (\>=)10 kg to less than (\<)14 kg received ELX 80 mg qd/TEZ 40 mg qd/IVA 60 mg once every morning (qAM) and 59.5 mg once every evening (qPM) in the treatment period for 24 weeks.
Intervention: IVA
Outcomes
Primary Outcomes
Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites
Time Frame: From Day 1 through Day 15
Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame: From Day 1 up to Day 43
Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame: From Day 1 up to Week 28
Secondary Outcomes
- Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites(From Day 1 through Week 16)
- Part B: Absolute Change in Sweat Chloride (SwCl)(From Baseline through Week 24)
- Part B: Absolute Change in Lung Clearance Index 2.5 (LCI 2.5)(From Baseline through Week 24)