Phase 3
N=18
Evaluation of ELX/TEZ/IVA in Cystic Fibrosis (CF) Subjects 2 Through 5 Years
Cystic Fibrosis
Bottom Line
View on ClinicalTrials.gov: NCT04537793 ↗Enrolled (actual)
18
Serious AEs
2.2%
Results posted
Jun 2023
Primary outcome: Primary: Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites — 0.00; 3.44; 3.69; 0.00 micrograms per milliliter (mcg/ml)
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- ELX/TEZ/IVA (Drug); IVA (Drug)
- Age
- Pediatric · 2+ yrs
- Sex
- All
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Primary completion
- Jun 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part A: Observed Pre-dose Concentration (Ctrough) of ELX,TEZ,IVA, and Relevant Metabolites |
0.00; 3.44; 3.69; 0.00; 2.39; 2.47 | — |
| PRIMARY Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment- Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
15; 0 | — |
| PRIMARY Part B: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) |
74; 2 | — |
| SECONDARY Part B: Observed Pre-dose Plasma Concentration (Ctrough) of ELX,TEZ,IVA and Relevant Metabolites |
2.91; 3.87; 3.13; 3.56; 2.48; 3.49 | — |
| SECONDARY Part B: Absolute Change in Sweat Chloride (SwCl) |
-57.9 | — |
| SECONDARY Part B: Absolute Change in Lung Clearance Index 2.5 (LCI 2.5) |
-0.83 | — |
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.
Eligibility Criteria
Key Inclusion Criteria
- Homozygous for the F508del mutation or heterozygous for F508del and a minimal function (MF) mutation (F/F or F/MF genotypes)
Key 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
Data sourced from ClinicalTrials.gov (NCT04537793). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.