Current Environment:

Summary

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 subjects with cystic fibrosis (CF) who are <24 months of age at treatment initiation and have an approved Ivacaftor-Responsive mutation

Conditions

Cystic Fibrosis

Recruitment Status

Enrolling by invitation

Eligibility Criteria

Inclusion Criteria:

Ivacaftor Arm: Subjects From Study 124 (NCT02725567 ) Part B:

Subjects 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: Subjects 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. Subjects will be eligible in countries/regions where ivacaftor is approved for use in subjects 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.

Exclusion Criteria:

Ivacaftor Arm: Subjects 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 subject.
Subjects receiving commercially available ivacaftor treatment

Ivacaftor Arm: Subjects 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 subject
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.

Intervention

Intervention Type

Intervention Name

Drug

Ivacaftor

Phase

Phase 3

Gender

All

Minimum Age

N/A

Maximum Age

24 Months

Download Date

January 11, 2022

Principal Investigator

N/A

Primary Contact Information

For more information on this trial, visit clinicaltrials.gov.

Contact

For more information and to contact the study team: