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Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older

Phase 3
Conditions
Cystic Fibrosis
Interventions
Registration Number
NCT06460506
Lead Sponsor
Vertex Pharmaceuticals Incorporated
Brief Summary

The purpose of the study is to evaluate the long-term safety, tolerability, efficacy, and pharmacodynamics (PD) of elexacaftor (ELX)/tezacaftor (TEZ)/ivacaftor (IVA).

Detailed Description

Not available

Recruitment & Eligibility

Status
ENROLLING_BY_INVITATION
Sex
All
Target Recruitment
50
Inclusion Criteria
  • Completed study drug treatment in the parent study VX22-445-122 Part B (NCT05882357) OR had study drug interruption(s) in the parent study but did not permanently discontinue study drug and completed study visits up to the last scheduled visit of the Treatment Period of the parent study

Key

Exclusion Criteria
  • History of drug intolerance in the parent study that would pose an additional risk to the participant
  • Current participation in an investigational drug trial other than the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
ELX/TEZ/IVAIVAParticipants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
ELX/TEZ/IVAELX/TEZ/IVAParticipants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Primary Outcome Measures
NameTimeMethod
Safety and Tolerability as Assessed by Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs)Day 1 up to Week 100
Secondary Outcome Measures
NameTimeMethod
Absolute Change in Sweat Chloride (SwCl)From Baseline through Week 96

Trial Locations

Locations (17)

The Kids Research Institute Australia

🇦🇺

Nedlands, Australia

The Royal Children's Hospital

🇦🇺

Parkville, Australia

Queensland Children's Hospital

🇦🇺

South Brisbane, 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

Kinderklinik III, Abt. fur Pneumologie

🇩🇪

Essen, Germany

Erasmus Medical Center

🇳🇱

Rotterdam, Netherlands

Inselspital - Universitaetsspital Bern

🇨🇭

Bern, Switzerland

Kinderspital Zurich

🇨🇭

Zurich, Switzerland

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, United Kingdom

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