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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

Tracking Information
First Submitted Date  ICMJE June 5, 2018
First Posted Date  ICMJE June 15, 2018
Results First Submitted Date  ICMJE December 20, 2019
Results First Posted Date  ICMJE February 11, 2020
Last Update Posted Date February 11, 2020
Actual Study Start Date  ICMJE May 17, 2018
Actual Primary Completion Date December 21, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: February 4, 2020)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: June 5, 2018)
Absolute change in lung clearance index2.5 (LCI2.5) [ Time Frame: from baseline through Week 8 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: February 4, 2020)
  • Absolute Change in Sweat Chloride At Week 8 [ Time Frame: From baseline at Week 8 ]
    Sweat samples were collected using an approved collection device.
  • 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.
  • 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) ]
Original Secondary Outcome Measures  ICMJE
 (submitted: June 5, 2018)
  • Absolute change in sweat chloride [ Time Frame: from baseline at Week 8 ]
  • Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline through Week 8 ]
  • Safety and tolerability as measured by adverse events (AEs) and non-serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (16 Weeks) ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate Efficacy and Safety of TEZ/IVA in Subjects Aged 6 Through 11 Years With Cystic Fibrosis
Official Title  ICMJE 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
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).
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: 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 TEZ/IVA FDC
  • Drug: Placebo
    Placebo matched to IVA
Study Arms  ICMJE
  • 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.
    Interventions:
    • Drug: Placebo
    • Drug: Placebo
  • 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.
    Interventions:
    • Drug: TEZ/IVA
    • Drug: IVA
    • Drug: Placebo
  • 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.
    Interventions:
    • Drug: IVA
    • Drug: Placebo
Publications * Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12:CD010966. doi: 10.1002/14651858.CD010966.pub3.

*   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: February 4, 2020)
67
Original Estimated Enrollment  ICMJE
 (submitted: June 5, 2018)
65
Actual Study Completion Date  ICMJE December 21, 2018
Actual Primary Completion Date December 21, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years to 11 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Belgium,   Denmark,   France,   Germany,   Ireland,   Poland,   Switzerland,   United Kingdom
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03559062
Other Study ID Numbers  ICMJE VX16-661-115
2016-004479-35 ( EudraCT Number )
Has Data Monitoring Committee Yes
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 Not Provided
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 February 2020

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