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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
Last Update Posted : March 1, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE May 15, 2018
First Posted Date  ICMJE May 25, 2018
Last Update Posted Date March 1, 2019
Actual Study Start Date  ICMJE April 25, 2018
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 24, 2018)
Safety and tolerability of long-term TEZ/IVA treatment based on adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through safety follow-up (up to 28 days after Last Dose at Week 96) ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03537651 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 24, 2018)
  • Absolute change in lung clearance index2.5 (LCI2.5) [ Time Frame: from baseline through 96 weeks ]
  • Absolute change in sweat chloride [ Time Frame: from baseline through 96 weeks ]
  • Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline through 96 weeks ]
  • Absolute change in body mass index (BMI) [ Time Frame: from baseline through 96 weeks ]
Original Secondary Outcome Measures  ICMJE Same as current
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 the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Subjects With an F508del CFTR Mutation
Official Title  ICMJE 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
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE
  • Drug: TEZ
    Fixed-dose Combination (FDC) tablet (TEZ/IVA)
    Other Name: tezacaftor; VX-661
  • Drug: IVA
    FDC tablet (TEZ/IVA)
    Other Name: ivacaftor; VX-770
Study Arms  ICMJE Experimental: TEZ + IVA

Subjects <40 kg will receive 50 mg TEZ/ 75 mg IVA as a FDC tablet in the morning and 75 mg IVA as a mono tablet in the evening.

Subjects >=40 kg will receive 100 mg TEZ/ 150 mg IVA FDC tablet in the morning and 150 mg IVA as a mono tablet in the evening.

Interventions:
  • Drug: TEZ
  • Drug: IVA
Publications * Not Provided

*   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 Active, not recruiting
Actual Enrollment  ICMJE
 (submitted: February 28, 2019)
130
Original Estimated Enrollment  ICMJE
 (submitted: May 24, 2018)
121
Estimated Study Completion Date  ICMJE December 2020
Estimated Primary Completion Date December 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Australia,   Belgium,   Canada,   Denmark,   France,   Germany,   Ireland,   Poland,   Switzerland,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03537651
Other Study ID Numbers  ICMJE VX17-661-116
2017-002968-40 ( 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 2019

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