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A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)

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ClinicalTrials.gov Identifier: NCT04058353
Recruitment Status : Completed
First Posted : August 15, 2019
Last Update Posted : July 13, 2020
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE August 14, 2019
First Posted Date  ICMJE August 15, 2019
Last Update Posted Date July 13, 2020
Actual Study Start Date  ICMJE August 28, 2019
Actual Primary Completion Date June 12, 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 14, 2019)
Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) for ELX/TEZ/IVA group [ Time Frame: From Baseline through Week 8 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 14, 2019)
  • Absolute change in sweat chloride (SwCl) for ELX/TEZ/IVA group [ Time Frame: From Baseline through Week 8 ]
  • Absolute change in ppFEV1 for ELX/TEZ/IVA group compared to the control group [ Time Frame: From Baseline through Week 8 ]
  • Absolute change in SwCl for ELX/TEZ/IVA group compared to the control group [ Time Frame: From Baseline through Week 8 ]
  • Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score for ELX/TEZ/IVA group [ Time Frame: From Baseline through Week 8 ]
  • Absolute change in CFQ-R respiratory domain score for ELX/TEZ/IVA group compared to the control group [ Time Frame: From Baseline through Week 8 ]
  • Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From Baseline up to Week 12 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase 3 Study of VX-445 Combination Therapy in Cystic Fibrosis (CF) Subjects Heterozygous for F508del and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
Official Title  ICMJE A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Gating or Residual Function Mutation (F/G and F/RF Genotypes)
Brief Summary This study will evaluate the efficacy, safety and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a gating or residual function mutation (F/G and F/RF genotypes).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: ELX/TEZ/IVA
    Fixed-dose combination (FDC) tablet for oral administration
    Other Names:
    • VX-445/VX-661/VX-770
    • elexacaftor/tezacaftor/ivacaftor
  • Drug: IVA
    150-mg tablet for oral administration
    Other Names:
    • VX-770
    • ivacaftor
  • Drug: TEZ/IVA
    FDC tablet for oral administration
    Other Names:
    • VX-661/VX-770
    • tezacaftor/ivacaftor
Study Arms  ICMJE
  • Experimental: Triple Combination Arm
    Subjects will receive ELX/TEZ/IVA TC in the morning and IVA as mono tablet in the evening.
    Interventions:
    • Drug: ELX/TEZ/IVA
    • Drug: IVA
  • Active Comparator: Control Arm
    Subjects will receive either IVA as mono tablet OR TEZ/IVA as FDC in the morning and IVA as mono tablet in the evening.
    Interventions:
    • Drug: IVA
    • Drug: TEZ/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 Completed
Actual Enrollment  ICMJE
 (submitted: May 14, 2020)
271
Original Estimated Enrollment  ICMJE
 (submitted: August 14, 2019)
250
Actual Study Completion Date  ICMJE June 12, 2020
Actual Primary Completion Date June 12, 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Subject has a confirmed diagnosis of CF and is heterozygous for F508del and either a gating or residual function mutation (F/G and F/RF genotypes)
  • Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection with organisms associated with a more rapid decline in pulmonary status
  • Solid organ or hematological transplantation

Other protocol defined Inclusion/Exclusion criteria may apply

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 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 Australia,   Belgium,   Canada,   Denmark,   France,   Germany,   Ireland,   Israel,   Italy,   Netherlands,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04058353
Other Study ID Numbers  ICMJE VX18-445-104
2018-002835-76 ( 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
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
Responsible Party Vertex Pharmaceuticals Incorporated
Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date July 2020

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