A Study to Evaluate the Safety of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03277196|
Recruitment Status : Active, not recruiting
First Posted : September 8, 2017
Last Update Posted : September 15, 2022
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|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: Ivacaftor||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||86 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 3, 2-Arm, Open-label Study to Evaluate the Safety and Pharmacodynamics of Long-term Ivacaftor Treatment in Subjects With Cystic Fibrosis Who Are Less Than 24 Months of Age at Treatment Initiation and Have an Approved Ivacaftor-Responsive Mutation|
|Actual Study Start Date :||August 16, 2017|
|Estimated Primary Completion Date :||July 3, 2024|
|Estimated Study Completion Date :||July 3, 2024|
|Experimental: Ivacaftor Arm||
Ivacaftor will be administered every 12 hours (q12h) from Day 1 through the morning dose of the Week 96 Visit.
Other Name: VX-770
|No Intervention: Observational Arm|
- Safety assessments based on the number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (up to 24 weeks after last dose) ]Number of subjects with AEs and SAEs will be reported.
- Absolute change in sweat chloride [ Time Frame: from baseline through Week 96 ]
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|Ages Eligible for Study:||0 Months to 24 Months (Child)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
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.
- Received ivacaftor treatment in Study 124 Part B and elected not to enroll or ineligible to enroll in the ivacaftor arm of Study 126.
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
- Receiving ivacaftor treatment
Other protocol defined Inclusion/Exclusion criteria may apply.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03277196
|Responsible Party:||Vertex Pharmaceuticals Incorporated|
|Other Study ID Numbers:||
2017-001379-21 ( EudraCT Number )
|First Posted:||September 8, 2017 Key Record Dates|
|Last Update Posted:||September 15, 2022|
|Last Verified:||September 2022|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Digestive System Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action