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Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del

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ClinicalTrials.gov Identifier: NCT05331183
Recruitment Status : Active, not recruiting
First Posted : April 15, 2022
Last Update Posted : May 7, 2024
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE April 8, 2022
First Posted Date  ICMJE April 15, 2022
Last Update Posted Date May 7, 2024
Actual Study Start Date  ICMJE November 23, 2022
Estimated Primary Completion Date April 2027   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 3, 2024)		 Parts A and B: 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 196 ]
Original Primary Outcome Measures  ICMJE
 (submitted: April 8, 2022)		 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 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 3, 2024)
  • Part A: Absolute Change in Percent Predicted Forced Expiratory Volume in 1 second (ppFEV1) [ Time Frame: From Baseline up to Week 96 ]
  • Part A: Absolute Change in Sweat Chloride (SwCl) [ Time Frame: From Baseline up to Week 96 ]
  • Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score [ Time Frame: From Baseline up to Week 96 ]
  • Part A: Absolute Change in Body Mass Index (BMI) [ Time Frame: From Baseline up to Week 96 ]
  • Part A: Absolute Change in Weight [ Time Frame: From Baseline up to Week 96 ]
  • Part A: Number of Pulmonary Exacerbations (PEx) [ Time Frame: From Baseline up to Week 96 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: April 8, 2022)
  • 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 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study to Evaluate Elexacaftor/Tezacaftor/Ivacaftor (ELX/TEZ/IVA) Long-term Safety and Efficacy in Subjects Without F508del
Official Title  ICMJE 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
Brief Summary This study will evaluate the long-term safety, efficacy and pharmacodynamics of ELX/TEZ/IVA in participants with cystic fibrosis (CF) with at least 1 non-F508del ELX/TEZ/IVA-responsive CF transmembrane conductance regulator (CFTR) gene mutation.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • 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
    Tablets for oral administration.
    Other Names:
    • VX-770
    • ivacaftor
Study Arms  ICMJE Experimental: ELX/TEZ/IVA

Part A: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for 96 weeks.

Part B: Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening for an additional 96 weeks.

Interventions:
  • Drug: ELX/TEZ/IVA
  • Drug: IVA
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: August 3, 2023)		 297
Original Estimated Enrollment  ICMJE
 (submitted: April 8, 2022)		 270
Estimated Study Completion Date  ICMJE April 2027
Estimated Primary Completion Date April 2027   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Part A: Completed study drug treatment in parent study or had study drug interruption(s) in parent study but completed study visits up to the last scheduled visit of the treatment period in the parent study
  • Part B: Completed study drug treatment in Part A or had study drug interruption(s) in Part A but completed study visits up to the last scheduled visit of the treatment period of Part A

Key Exclusion Criteria:

  • History of study drug intolerance in the parent study

Other protocol defined Inclusion/Exclusion criteria may apply.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   Belgium,   Canada,   Czechia,   France,   Germany,   Hungary,   Italy,   Netherlands,   Norway,   Poland,   Portugal,   Spain,   Sweden,   Switzerland
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05331183
Other Study ID Numbers  ICMJE VX21-445-125
2021-005914-33 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
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
Current Responsible Party Vertex Pharmaceuticals Incorporated
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date May 2024

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP