Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
|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: NCT05331183|
Recruitment Status : Active, not recruiting
First Posted : April 15, 2022
Last Update Posted : August 4, 2023
|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: ELX/TEZ/IVA Drug: IVA||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||297 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes|
|Actual Study Start Date :||November 23, 2022|
|Estimated Primary Completion Date :||April 2025|
|Estimated Study Completion Date :||April 2025|
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Fixed-dose combination (FDC) tablets for oral administration.
Tablets for oral administration.
- Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Day 1 up to Week 100 ]
- Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1) [ Time Frame: From Baseline up to Week 96 ]
- Absolute Change in Sweat Chloride (SwCl) [ Time Frame: From Baseline up to Week 96 ]
- Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [ Time Frame: From Baseline up to Week 96 ]
- Absolute Change in Body Mass Index (BMI) [ Time Frame: From Baseline up to Week 96 ]
- Absolute Change in Weight [ Time Frame: From Baseline up to Week 96 ]
- Number of Pulmonary Exacerbations (PEx) [ Time Frame: From Baseline up to 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): NCT05331183