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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
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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Brief Summary:
This study will evaluate the efficacy, pharmacodynamics (PD) and safety of ELX/TEZ/IVA in participants 6 years of age and older with a non-F508del ELX/TEZ/IVA-responsive cystic fibrosis transmembrane conductance regulator gene (CFTR) mutation.

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 Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 307 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor

Arms and Interventions
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Arm Intervention/treatment
Experimental: ELX/TEZ/IVA
Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.
 Drug: ELX/TEZ/IVA
Fixed-dose combination (FDC) tablets for oral administration.
Other Names:
  • VX-445/VX-661/VX-770
  • elexacaftor/tezacaftor/ivacaftor

Drug: IVA
Tablet for oral administration.
Other Names:
  • VX-770
  • ivacaftor
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.


Outcome Measures
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Primary Outcome Measures :
  1. Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [ Time Frame: From Baseline Through Week 24 ]

Secondary Outcome Measures :
  1. Absolute Change in Sweat Chloride (SwCl) [ Time Frame: From Baseline Through Week 24 ]
  2. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [ Time Frame: From Baseline Through Week 24 ]
  3. Absolute Change in Body Mass Index (BMI) [ Time Frame: From Baseline at Week 24 ]
  4. Absolute Change in Weight [ Time Frame: From Baseline at Week 24 ]
  5. Number of Pulmonary Exacerbations (PEx) [ Time Frame: From Baseline Through Week 24 ]
  6. 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 ]

Eligibility Criteria
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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
Criteria

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.

Contacts and Locations
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Information from the National Library of Medicine

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


Locations
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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT05274269    
Other Study ID Numbers: VX21-445-124
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

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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
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
 Genetic Diseases, Inborn
Infant, Newborn, Diseases
Ivacaftor
Elexacaftor
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action