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A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis

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ClinicalTrials.gov Identifier: NCT03559062
Recruitment Status : Completed
First Posted : June 15, 2018
Results First Posted : February 11, 2020
Last Update Posted : February 11, 2020
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This study will evaluate the efficacy of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 through 11 years, who are homozygous for the F508del mutation (F/F) or heterozygous for F508del with an eligible residual function mutation (F/RF).

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 67 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Double-blind, Parallel-group Study to Evaluate the Efficacy and Safety of Tezacaftor in Combination With Ivacaftor in Subjects Aged 6 Through 11 Years With Cystic Fibrosis, Homozygous or Heterozygous for the F508del-CFTR Mutation
Actual Study Start Date : May 17, 2018
Actual Primary Completion Date : December 21, 2018
Actual Study Completion Date : December 21, 2018

Resource links provided by the National Library of Medicine

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

Arm Intervention/treatment
Placebo
Participants with genotype F/F received placebo matched to TEZ/IVA fixed dose combination (FDC) in the morning and placebo matched to IVA in the evening for 8 weeks.
Drug: Placebo
Placebo matched to TEZ/IVA FDC

Drug: Placebo
Placebo matched to IVA

Experimental: TEZ/IVA
Participants with genotype F/F received TEZ/IVA FDC in the morning and IVA in the evening for 8 weeks. Participants with genotype F/RF received TEZ/IVA FDC and placebo matched to IVA in the morning and IVA in the evening for 8 weeks.
Drug: TEZ/IVA
Participants weighing <40 kg received TEZ 50 mg/IVA 75 mg FDC tablet and those weighing ≥40 kg received TEZ 100 mg/IVA 150 mg FDC tablet.
Other Names:
  • VX-661/VX-770
  • tezacaftor/ivacaftor fixed dose combination

Drug: IVA
Participants weighing <40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet.
Other Names:
  • VX-770
  • ivacaftor

Drug: Placebo
Placebo matched to IVA

Experimental: Ivacaftor
Participants with genotype F/RF received placebo matched to TEZ/IVA FDC in the morning and IVA in morning and evening for 8 weeks.
Drug: IVA
Participants weighing <40 kg IVA 75 mg tablet and those weighing ≥40 kg received IVA 150 mg tablet.
Other Names:
  • VX-770
  • ivacaftor

Drug: Placebo
Placebo matched to TEZ/IVA FDC




Primary Outcome Measures :
  1. Absolute Change in Lung Clearance Index 2.5 (LCI2.5) Through Week 8 [ Time Frame: From baseline through Week 8 ]
    LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.


Secondary Outcome Measures :
  1. Absolute Change in Sweat Chloride At Week 8 [ Time Frame: From baseline at Week 8 ]
    Sweat samples were collected using an approved collection device.

  2. Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 8 [ Time Frame: From baseline through Week 8 ]
    The CFQ-R is a validated participant-reported outcome measuring health-related quality of life for participants with cystic fibrosis. Respiratory domain assessed respiratory symptoms, score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life.

  3. Safety and Tolerability as Assessed Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) up to Safety Follow-up Visit [ Time Frame: From first dose of study drug up to safety follow-up visit (up to Week 12) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 11 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Homozygous for F508del or heterozygous for F508del and an RF mutation (as defined in the protocol).
  • Participants with ppFEV1 of ≥70 percentage points adjusted for age, sex, height.
  • Participants with a screening LCI2.5 result ≥7.5.
  • Participants who are able to swallow tablets.

Key Exclusion Criteria:

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

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): NCT03559062


Locations
Show Show 27 study locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] November 17, 2017
Statistical Analysis Plan  [PDF] October 31, 2018

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT03559062    
Other Study ID Numbers: VX16-661-115
2016-004479-35 ( EudraCT Number )
First Posted: June 15, 2018    Key Record Dates
Results First Posted: February 11, 2020
Last Update Posted: February 11, 2020
Last Verified: February 2020

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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