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

Cystic Fibrosis Clinical Trial

Official 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05882357
• Other study ID #: VX22-445-122
• Secondary ID: 2023-503230-49-0
• Status: Recruiting
• Phase: Phase 3
• Start date: June 27, 2023
• Est. completion date: October 2025

Study information

• Verified date: May 2024
• Source: Vertex Pharmaceuticals Incorporated
• Contact: Medical Information
• Phone: 617-341-6777
• Email: medicalinfo[@]vrtx.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 64
• Est. completion date: October 2025
• Est. primary completion date: October 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Months to 24 Months

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• ELX/TEZ/IVA
Fixed-dose combination granules for oral administration.
• IVA
Granules for oral administration

Locations

Country	Name	City	State
Australia	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
Canada	The Hospital for Sick Children	Toronto
Canada	British Columbia Children's Hospital	Vancouver
Switzerland	Inselspital - Universitaetsspital Bern	Bern
Switzerland	Kinderspital Zuerich	Zurich
United Kingdom	Children and Young Adults Research Unit	Cardiff
United Kingdom	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

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Countries where clinical trial is conducted

Australia,  Canada,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Part A: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites		Day 1 up to Day 15
Primary	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
Primary	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
Secondary	Part B: Observed Pre-dose Concentration (Ctrough) of ELX, TEZ, IVA, and their Relevant Metabolites		Day 15 up to Week 16
Secondary	Part B: Absolute Change in Sweat Chloride (SwCl)		From Baseline Through Week 24