A Study Evaluating Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis and F/MF Genotypes
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|ClinicalTrials.gov Identifier: NCT04353817|
Recruitment Status : Recruiting
First Posted : April 20, 2020
Last Update Posted : September 23, 2020
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: ELX/TEZ/IVA Drug: IVA Other: Placebo||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||108 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 3b, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects 6 Through 11 Years of Age Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)|
|Actual Study Start Date :||June 19, 2020|
|Estimated Primary Completion Date :||May 2021|
|Estimated Study Completion Date :||May 2021|
Subjects will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Fixed-dose combination (FDC) tablet.
Placebo Comparator: Placebo
Subjects will receive placebo matched to ELX/TEZ/IVA in the morning and placebo matched to IVA in the evening.
Placebo matched to ELX/TEZ/IVA.
Placebo matched to IVA.
- Absolute change in lung clearance index 2.5 (LCI2.5) [ Time Frame: From baseline through Week 24 ]
- Absolute change in sweat chloride (SwCl) [ Time Frame: From baseline through Week 24 ]
- Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From Day 1 up to Week 28 ]
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): NCT04353817
|Contact: Medical Informationfirstname.lastname@example.org|