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

NCT ID: NCT05882357

Last Updated: 2025-10-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

70 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-06-27

Study Completion Date

2025-09-04

Brief Summary

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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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Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Part A

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

Group Type EXPERIMENTAL

ELX/TEZ/IVA

Intervention Type DRUG

Fixed-dose combination granules for oral administration.

IVA

Intervention Type DRUG

Granules for oral administration

Part B

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.

Group Type EXPERIMENTAL

ELX/TEZ/IVA

Intervention Type DRUG

Fixed-dose combination granules for oral administration.

IVA

Intervention Type DRUG

Granules for oral administration

Interventions

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ELX/TEZ/IVA

Fixed-dose combination granules for oral administration.

Intervention Type DRUG

IVA

Granules for oral administration

Intervention Type DRUG

Other Intervention Names

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VX-445/VX-661/VX-770 elexacaftor/tezacaftor/ivacaftor VX-770 ivacaftor

Eligibility Criteria

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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

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
Minimum Eligible Age

12 Months

Maximum Eligible Age

24 Months

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Telethon Kids Institute

Nedlands, , Australia

Site Status

The Royal Children's Hospital

Parkville, , Australia

Site Status

Queensland Children's Hospital

South Brisbane, , Australia

Site Status

The Children's Hospital at Westmead

Westmead, , Australia

Site Status

The Hospital for Sick Children

Toronto, , Canada

Site Status

British Columbia Children's Hospital

Vancouver, , Canada

Site Status

Juliane Marie Center, Rigshospitalet

Copenhagen, , Denmark

Site Status

Charite Paediatric Pulmonology Department

Berlin, , Germany

Site Status

Kinderklinik III, Abt. fur Pneumologie

Essen, , Germany

Site Status

Medizinische Hochschule Hannover

Hanover, , Germany

Site Status

Erasmus Medical Center / Sophia Children's Hospital

Rotterdam, , Netherlands

Site Status

Inselspital - Universitaetsspital Bern

Bern, , Switzerland

Site Status

Kinderspital Zuerich

Zurich, , Switzerland

Site Status

Children and Young Adults Research Unit

Cardiff, , United Kingdom

Site Status

Leeds General Infirmary

Leeds, , United Kingdom

Site Status

Alder Hey Children's NHS Foundation Trust

Liverpool, , United Kingdom

Site Status

Great Ormond Street Hospital for Children

London, , United Kingdom

Site Status

Royal Brompton Hospital

London, , United Kingdom

Site Status

Southampton General Hospital

Southampton, , United Kingdom

Site Status

Countries

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Australia Canada Denmark Germany Netherlands Switzerland United Kingdom

Other Identifiers

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2023-503230-49-00

Identifier Type: OTHER

Identifier Source: secondary_id

VX22-445-122

Identifier Type: -

Identifier Source: org_study_id

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