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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 : Recruiting
First Posted : August 15, 2019
Last Update Posted : February 17, 2020
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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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).

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: ELX/TEZ/IVA Drug: IVA Drug: TEZ/IVA Phase 3

Expanded Access : An investigational treatment associated with this study has been approved for sale to the public.   More info ...

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 250 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : August 28, 2019
Estimated Primary Completion Date : October 2020
Estimated Study Completion Date : October 2020

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arms and Interventions
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Arm Intervention/treatment
Experimental: Triple Combination Arm
Subjects will receive ELX/TEZ/IVA TC in the morning and IVA as mono tablet in the evening.
 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
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.
 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


Outcome Measures
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Primary Outcome Measures :
  1. Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) for ELX/TEZ/IVA group [ Time Frame: From Baseline through Week 8 ]

Secondary Outcome Measures :
  1. Absolute change in sweat chloride (SwCl) for ELX/TEZ/IVA group [ Time Frame: From Baseline through Week 8 ]
  2. Absolute change in ppFEV1 for ELX/TEZ/IVA group compared to the control group [ Time Frame: From Baseline through Week 8 ]
  3. Absolute change in SwCl for ELX/TEZ/IVA group compared to the control group [ Time Frame: From Baseline through Week 8 ]
  4. 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 ]
  5. 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 ]
  6. 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 ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

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): NCT04058353


Contacts
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Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com

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: NCT04058353    
Other Study ID Numbers: VX18-445-104
2018-002835-76 ( EudraCT Number )
First Posted: August 15, 2019    Key Record Dates
Last Update Posted: February 17, 2020
Last Verified: December 2019
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
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
VX-445
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action