NCT05882357

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
70

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jun 2023

Geographic Reach
7 countries

19 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 19, 2023

Completed
12 days until next milestone

First Posted

Study publicly available on registry

May 31, 2023

Completed
27 days until next milestone

Study Start

First participant enrolled

June 27, 2023

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 4, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

September 4, 2025

Completed
Last Updated

October 6, 2025

Status Verified

October 1, 2025

Enrollment Period

2.2 years

First QC Date

May 19, 2023

Last Update Submit

October 3, 2025

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (3)

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

    Day 1 up to Day 15

  • 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

  • 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 Outcomes (2)

  • 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

Study Arms (2)

Part A

EXPERIMENTAL

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Drug: ELX/TEZ/IVADrug: IVA

Part B

EXPERIMENTAL

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.

Drug: ELX/TEZ/IVADrug: IVA

Interventions

ELX/TEZ/IVADRUG

Fixed-dose combination granules for oral administration.

Also known as: VX-445/VX-661/VX-770, elexacaftor/tezacaftor/ivacaftor
Part APart B
IVADRUG

Granules for oral administration

Also known as: VX-770, ivacaftor
Part APart B

Eligibility Criteria

Age12 Months - 24 Months
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Participants who have at least 1 F508del mutation in the CF transmembrane conductance regulator (CFTR) gene or another ELX/TEZ/IVA-responsive CFTR mutation

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (19)

Telethon Kids Institute

Nedlands, Australia

Location

The Royal Children's Hospital

Parkville, Australia

Location

Queensland Children's Hospital

South Brisbane, Australia

Location

The Children's Hospital at Westmead

Westmead, Australia

Location

The Hospital for Sick Children

Toronto, Canada

Location

British Columbia Children's Hospital

Vancouver, Canada

Location

Juliane Marie Center, Rigshospitalet

Copenhagen, Denmark

Location

Charite Paediatric Pulmonology Department

Berlin, Germany

Location

Kinderklinik III, Abt. fur Pneumologie

Essen, Germany

Location

Medizinische Hochschule Hannover

Hanover, Germany

Location

Erasmus Medical Center / Sophia Children's Hospital

Rotterdam, Netherlands

Location

Inselspital - Universitaetsspital Bern

Bern, Switzerland

Location

Kinderspital Zuerich

Zurich, Switzerland

Location

Children and Young Adults Research Unit

Cardiff, United Kingdom

Location

Leeds General Infirmary

Leeds, United Kingdom

Location

Alder Hey Children's NHS Foundation Trust

Liverpool, United Kingdom

Location

Great Ormond Street Hospital for Children

London, United Kingdom

Location

Royal Brompton Hospital

London, United Kingdom

Location

Southampton General Hospital

Southampton, United Kingdom

Location

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

elexacaftor, ivacaftor, tezacaftor drug combinationivacaftor

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 19, 2023

First Posted

May 31, 2023

Study Start

June 27, 2023

Primary Completion

September 4, 2025

Study Completion

September 4, 2025

Last Updated

October 6, 2025

Record last verified: 2025-10

Data Sharing

IPD Sharing
Will not share

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

Locations

DK(1)NL(1)DE(3)CA(2)AU(4)GB(6)CH(2)