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Completed

NCT Number: NCT05882357

Evaluation of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis (CF) Participants 12 to Less Than 24 Months of Age

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.

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Key information

Conditions

Cystic Fibrosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Lung Diseases Pancreatic Diseases Respiratory Tract Diseases

Age range

12 month–24 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Telethon Kids Institute, Nedlands, Australia

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Key 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

Key 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.

Treatment and study plan

ELX/TEZ/IVA

Drug

Fixed-dose combination granules for oral administration.

Other names: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor

IVA

Drug

Granules for oral administration

Other names: VX-770, ivacaftor

Primary outcomes

  1. Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites

    Time frame: Day 1 up to Day 15

  2. 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

  3. 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

Secondary outcomes

  1. Part B: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites

    Time frame: Day 15 up to Week 16

  2. Part B: Absolute Change in Sweat Chloride (SwCl)

    Time frame: From Baseline Through Week 24

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2025
Study completion
2025
First posted
May 31, 2023
Registry last updated
Oct 6, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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