A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Participants With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation

Completed · Phase 3

Conditions studied: Cystic Fibrosis

In brief

This is a Phase 3, 2-arm, multicenter study with an open-label ivacaftor arm and an observational arm to evaluate the safety and efficacy of long-term ivacaftor treatment in participants with cystic fibrosis (CF) who are \<24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation.

Key facts

Study ID

NCT03277196

Run by

Vertex Pharmaceuticals Incorporated

People needed

86

Starts

2017-08-16

Expected to finish

2023-10-02

Last updated by the study team

2024-10-23

Who can join

Age: any, up to 2. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Ivacaftor Arm: Participants From Study 124 (NCT02725567 ) Part B:
• Participants transitioning from Study 124 Part B must have completed the last study visit of Study 124 Part B.
• As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the informed consent form (ICF).
• Ivacaftor Arm: Participants Not From Study 124 Part B:
• Confirmed diagnosis of CF, or 2 CF-causing mutations.
• An ivacaftor- responsive CFTR mutation on at least 1 allele. Participants will be eligible in countries/regions where ivacaftor is approved for use in participants 2 years of age and older.
• As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions; and must sign the ICF.
• Observational Arm:
• Received ivacaftor treatment in Study 124 Part B and elected not to enroll or ineligible to enroll in the ivacaftor arm of Study 126.

You may not qualify if…

• Ivacaftor Arm: Participants From Study 124 Part B:
• History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the participant.
• Participants receiving commercially available ivacaftor treatment
• Ivacaftor Arm: Participants Not From Study 124 Part B:
• History of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering ivacaftor to the participant
• An acute upper or lower respiratory infection, or pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1
• Abnormal liver function at screening
• Hemoglobin <9.5 g/dL at screening
• History of solid organ or hematological transplantation
• Use of any moderate or strong inducers or inhibitors of CYP3A within 2 weeks of Day 1
• Observational Arm:
• Receiving ivacaftor treatment
• Other protocol defined Inclusion/Exclusion criteria may apply.

Where it is running

• University of Alabama at Birmingham — Birmingham, Alabama, United States
• Stanford University — Palo Alto, California, United States
• Alfred I DuPont Hospital for Children — Wilmington, Delaware, United States
• Nemours Children's Hospital — Orlando, Florida, United States
• Center for Advanced Pediatrics — Atlanta, Georgia, United States
• Northwestern University — Chicago, Illinois, United States
• Riley Hospital for Children at Indiana University Health — Indianapolis, Indiana, United States
• John Hopkins Hospital — Baltimore, Maryland, United States
• Boston Children's Hospital — Boston, Massachusetts, United States
• Childrens's Hospitals and Clinics of Minnnesota — Minneapolis, Minnesota, United States
• Children's Mercy Hospital — Kansas City, Missouri, United States
• Billings Clinic Hospital — Billings, Montana, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
• Texas Children's Hospital — Houston, Texas, United States
• Seattle Children's Hospital — Seattle, Washington, United States
• University of Wisconsin — Madison, Wisconsin, United States
• Queensland Children's Hospital — South Brisbane, Australia
• The Hospital for Sick Children — Toronto, Ontario, Canada
• Universitaetsklinikum Heidelberg, Zenter fuer Kinder-und Jugendmedizin — Heidelberg, Germany
• Children's Health Ireland at Crumlin — Dublin, Ireland
• Children's University Hospital Temple Street — Dublin, Ireland
• University Hospital Limerick — Limerick, Ireland
• Paediatric Clinical Research Facility — Edinburgh, United Kingdom
• Alder Hey Children's Alder Hey Children's NHS Foundation Trust — Liverpool, United Kingdom

Full record on ClinicalTrials.gov

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