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A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Subjects With an F508del CFTR Mutation

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: NCT03537651
Recruitment Status : Active, not recruiting
First Posted : May 25, 2018
Results First Posted : November 26, 2021
Last Update Posted : November 26, 2021
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This study will evaluate the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in subjects with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del mutation.

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 130 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 3, Open-label, Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Tezacaftor in Combination With Ivacaftor in Subjects With Cystic Fibrosis Aged 6 Years and Older, Homozygous or Heterozygous for the F508del-CFTR Mutation
Actual Study Start Date : April 25, 2018
Actual Primary Completion Date : October 28, 2020
Estimated Study Completion Date : September 2022

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Experimental: TEZ/IVA
TEZ 50 mg once daily (qd)/IVA 75 mg every 12 hours (q12h) or TEZ 100 mg qd/IVA 150 mg q12h based on body weight for participants aged 6 through 11 years at enrollment and TEZ 100 mg qd/IVA 150 mg q12h for participants aged >=12 years at enrollment. Doses were adjusted upward for changes in body weight and/or age.
Drug: TEZ/IVA
Fixed-dose combination tablet for oral administration.
Other Names:
  • VX-661/VX-770
  • tezacaftor/ivacaftor

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




Primary Outcome Measures :
  1. Part A: Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Part A: Day 1 up to Week 100 ]

Secondary Outcome Measures :
  1. Part A: Absolute Change in Lung Clearance Index2.5 (LCI2.5) for 115/116 FAS (TEZ/IVA Group) [ Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116) ]
    LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

  2. Part A: Absolute Change in LCI2.5 for 113B/116 LCI FAS [ Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116) ]
    LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.

  3. Part A: Absolute Change in Sweat Chloride for 115/116 FAS (TEZ/IVA Group) [ Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116) ]
    Sweat samples were collected using an approved collection device.

  4. Part A: Absolute Change in SwCl for 113B/116 FAS [ Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116) ]
    Sweat samples were collected using an approved collection device.

  5. Part A: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score for 115/116 FAS (TEZ/IVA Group) [ Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116) ]
    The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

  6. Part A: Absolute Change in CFQ-R Respiratory Domain Score for 113B/116 FAS [ Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116) ]
    The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with CF. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

  7. Part A: Absolute Change in Body Mass Index (BMI) for 115/116 FAS (TEZ/IVA Group) [ Time Frame: From Parent Study 115 Baseline at Week 96 (Study 116) ]
    BMI was defined as weight in kilograms (kg) divided by squared height in meters (m^2).

  8. Part A: Absolute Change in BMI for 113B/116 FAS [ Time Frame: From Parent Study 113B Baseline at Week 96 (Study 116) ]
    BMI was defined as weight in kg divided by m^2.



Information from the National Library of Medicine

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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

Inclusion Criteria:

  • Completed the Week 24 Visit in Study 113 Part B or the Week 8 Visit in Study 115.
  • Eligible CFTR Mutation.

Exclusion Criteria:

  • Pregnant and nursing females.
  • History of poor compliance with study drug and/or procedures in a previous study as deemed by the investigator.
  • Ongoing participation in another study with investigational drug.

Other protocol defined Inclusion/Exclusion criteria may apply.


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): NCT03537651


Locations
Show Show 55 study locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] November 8, 2019
Statistical Analysis Plan  [PDF] August 19, 2020

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03537651    
Other Study ID Numbers: VX17-661-116
2017-002968-40 ( EudraCT Number )
First Posted: May 25, 2018    Key Record Dates
Results First Posted: November 26, 2021
Last Update Posted: November 26, 2021
Last Verified: October 2021

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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
Chloride Channel Agonists
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action