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A Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03525574
Recruitment Status : Active, not recruiting
First Posted : May 15, 2018
Last Update Posted : September 17, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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Brief Summary:
This study will evaluate the long-term safety and tolerability of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the F508del mutation

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: VX-445 Drug: TEZ Drug: 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)
Actual Enrollment : 507 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-label Study Evaluating the Long-term Safety and Efficacy of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous or Heterozygous for the F508del Mutation
Actual Study Start Date : October 9, 2018
Estimated Primary Completion Date : June 2021
Estimated Study Completion Date : June 2021

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: Open-label Triple Combination

Subjects will receive 200 mg VX-445/ 100 mg TEZ/ 150 mg IVA as FDC tablets in the morning and 150 mg IVA as mono tablet in the evening.

Parent studies are Phase 3 Vertex studies investigating VX-445 in combination with TEZ and IVA. This includes Studies VX17-445-102 and VX17-445-103.

 Drug: VX-445
Fixed-dose combination (FDC) tablets (VX-445/TEZ/IVA)

Drug: TEZ
FDC tablets (VX-445/TEZ/IVA)
Other Name: tezacaftor; VX-661

Drug: IVA
FDC tablets (VX-445/TEZ/IVA)
Other Name: ivacaftor; VX-770

Drug: IVA
IVA tablet
Other Name: ivacaftor; VX-770


Outcome Measures
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Primary Outcome Measures :
  1. Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (up to 100 weeks) ]

Secondary Outcome Measures :
  1. Absolute change from baseline in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  2. Absolute change in sweat chloride (SwCl) [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  3. Number of pulmonary exacerbations (PEx) [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  4. Time to first PEx [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  5. Absolute change in body mass index (BMI) [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  6. Absolute change in BMI z-score [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  7. Absolute change in body weight [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]
  8. Absolute change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline through last dose of study drug (up to 96 weeks) ]

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

Inclusion Criteria:

  • Completed study drug treatment in a parent study; or had study drug interruption(s) in a parent study but completed study visits up to the last scheduled visit of the Treatment Period in the parent study.

Exclusion Criteria:

  • History of drug intolerance in a parent study that would pose an additional risk to the subject in the opinion of the investigator.
  • Current participation in an investigational drug trial (other than a parent study)

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): NCT03525574


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: NCT03525574    
Other Study ID Numbers: VX17-445-105
2018-000185-11 ( EudraCT Number )
First Posted: May 15, 2018    Key Record Dates
Last Update Posted: September 17, 2019
Last Verified: September 2019

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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