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 : Enrolling by invitation
First Posted : September 8, 2017
Last Update Posted : July 7, 2021
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: Ivacaftor||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||75 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 ]
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