Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age
- Registration Number
- NCT05882357
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 70
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
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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Part A IVA Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening. Part B IVA 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. Part A ELX/TEZ/IVA Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening. Part B ELX/TEZ/IVA 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.
- Primary Outcome Measures
Name Time Method Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites 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) 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) Day 1 up to Day 43
- Secondary Outcome Measures
Name Time Method 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
Trial Locations
- Locations (19)
Erasmus Medical Center / Sophia Children's Hospital
🇳🇱Rotterdam, Netherlands
Inselspital - Universitaetsspital Bern
🇨🇭Bern, Switzerland
Kinderspital Zuerich
🇨🇭Zurich, Switzerland
Children and Young Adults Research Unit
🇬🇧Cardiff, United Kingdom
Kinderklinik III, Abt. fur Pneumologie
🇩🇪Essen, Germany
Medizinische Hochschule Hannover
🇩🇪Hannover, Germany
Telethon Kids Institute
🇦🇺Nedlands, Australia
The Royal Children's Hospital
🇦🇺Parkville, Australia
Queensland Children's Hospital
🇦🇺South Brisbane, Australia
The Children's Hospital at Westmead
🇦🇺Westmead, Australia
The Hospital for Sick Children
🇨🇦Toronto, Canada
British Columbia Children's Hospital
🇨🇦Vancouver, Canada
Juliane Marie Center, Rigshospitalet
🇩🇰Copenhagen, Denmark
Charite Paediatric Pulmonology Department
🇩🇪Berlin, Germany
Leeds General Infirmary
🇬🇧Leeds, United Kingdom
Alder Hey Children's NHS Foundation Trust
🇬🇧Liverpool, United Kingdom
Great Ormond Street Hospital for Children
🇬🇧London, United Kingdom
Royal Brompton Hospital
🇬🇧London, United Kingdom
Southampton General Hospital
🇬🇧Southampton, United Kingdom