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

Phase 3

Completed

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

Recruitment & Eligibility

Inclusion Criteria

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.

Exclusion Criteria

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:

Other protocol defined Inclusion/Exclusion criteria may apply.

Arm && Interventions

Group	Intervention	Description
Ivacaftor Arm	IVA	Participants less than (\<) 24 months of age and weighing 5 to less than (\<) 7 kilogram (kg) received 25 mg IVA every 12 hours (q12h), 7 to \<14 kg received 50 mg IVA q12h, and those weighing 14 to \<25 kg received 75 mg IVA q12h. Participants more than or equal (\>=) 24 months of age and weighing \<14 kg received 50 mg IVA q12h, and those weighing more than or equal to (\>=)14 kg received 75 mg IVA q12h in the treatment period for up to 96 weeks. Doses were determined based on safety and pharmacokinetic (PK) data from parent study, age and weight.

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEs	Day 1 up to Week 120

Secondary Outcome Measures

Name	Time	Method
Absolute Change in Sweat Chloride	From Baseline at Week 96	Sweat samples were collected using an approved collection device.

Locations (29): 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
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Chicago, Illinois, United States
Riley Hospital for Children at Indiana University Health
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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
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University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States