Evaluation of Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) in Cystic Fibrosis Subjects Without an F508del Mutation
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|ClinicalTrials.gov Identifier: NCT05274269|
Recruitment Status : Active, not recruiting
First Posted : March 10, 2022
Last Update Posted : February 8, 2023
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|Condition or disease||Intervention/treatment||Phase|
|Cystic Fibrosis||Drug: ELX/TEZ/IVA Drug: IVA Other: Placebo (matched to ELX/TEZ/IVA) Other: Placebo (matched to IVA)||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||307 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|Official Title:||A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR Mutation|
|Actual Study Start Date :||May 9, 2022|
|Estimated Primary Completion Date :||April 2023|
|Estimated Study Completion Date :||May 2023|
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
Fixed-dose combination (FDC) tablets for oral administration.
Tablet for oral administration.
Placebo Comparator: Placebo
Participants will receive placebo matched to ELX/TEZ/IVA in the morning and placebo matched to IVA in the evening.
Other: Placebo (matched to ELX/TEZ/IVA)
Placebo matched to ELX/TEZ/IVA for oral administration.
Other: Placebo (matched to IVA)
Placebo matched to IVA for oral administration.
- Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [ Time Frame: From Baseline Through Week 24 ]
- Absolute Change in Sweat Chloride (SwCl) [ Time Frame: From Baseline Through Week 24 ]
- Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [ Time Frame: From Baseline Through Week 24 ]
- Absolute Change in Body Mass Index (BMI) [ Time Frame: From Baseline at Week 24 ]
- Absolute Change in Weight [ Time Frame: From Baseline at Week 24 ]
- Number of Pulmonary Exacerbations (PEx) [ Time Frame: From Baseline Through Week 24 ]
- 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 28 ]
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|Ages Eligible for Study:||6 Years and older (Child, Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
Key Inclusion Criteria:
- Participant has a qualifying ELX/TEZ/IVA-responsive CFTR mutation and does not have an exclusionary CFTR mutation
- Forced expiratory volume in 1 second (FEV1) value >=40% and <=100% of predicted mean for age, sex, and height
Key Exclusion Criteria:
- History of solid organ or hematological transplantation
- Clinically significant cirrhosis with or without portal hypertension
- Lung infection with organisms associated with a more rapid decline in pulmonary status
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): NCT05274269
|Responsible Party:||Vertex Pharmaceuticals Incorporated|
|Other Study ID Numbers:||
2021-005320-38 ( EudraCT Number )
|First Posted:||March 10, 2022 Key Record Dates|
|Last Update Posted:||February 8, 2023|
|Last Verified:||February 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||No|
|Plan Description:||Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
|Product Manufactured in and Exported from the U.S.:||Yes|
Digestive System Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action