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A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)

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ClinicalTrials.gov Identifier: NCT03525444
Recruitment Status : Completed
First Posted : May 15, 2018
Last Update Posted : May 27, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE May 2, 2018
First Posted Date  ICMJE May 15, 2018
Last Update Posted Date May 27, 2019
Actual Study Start Date  ICMJE June 15, 2018
Actual Primary Completion Date April 24, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 2, 2018)
Absolute change in percent predicted forced expiratory volume in 1 second (ppFEV1) [ Time Frame: from baseline at Week 4 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT03525444 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: May 2, 2018)
  • Absolute change in ppFEV1 [ Time Frame: from baseline through Week 24 ]
  • Number of pulmonary exacerbations (PEx) [ Time Frame: from baseline through Week 24 ]
  • Absolute change in body mass index (BMI) [ Time Frame: from baseline at Week 24 ]
  • Absolute change in sweat chloride (SwCl) [ Time Frame: from baseline at Week 4 and through Week 24 ]
  • Absolute change in Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score [ Time Frame: from baseline at Week 4 and through Week 24 ]
  • Time-to-first PEx [ Time Frame: through Week 24 ]
  • Absolute change in BMI z-score [ Time Frame: from baseline at Week 24 ]
  • Absolute change in body weight [ Time Frame: from baseline at Week 24 ]
  • Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: from baseline through 4-week safety follow-up (up to 28 weeks) ]
  • Observed pre-dose concentration (Ctrough) of VX-445, TEZ, M1-TEZ, and IVA [ Time Frame: from Day 1 through Week 16 ]
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 Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Official Title  ICMJE A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
Brief Summary This study will evaluate the efficacy of VX-445 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are heterozygous for F508del and a minimal function mutation (F/MF subjects).
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: VX-445
    Fixed dose combination (FDC) tablet (VX-445/TEZ/IVA)
  • Drug: TEZ
    FDC tablet (VX-445/TEZ/IVA)
    Other Name: tezacaftor; VX-661
  • Drug: IVA
    FDC tablet (VX-445/TEZ/IVA)
    Other Name: ivacaftor; VX-770
  • Drug: Matched Placebos
    Placebo will be used as a comparator.
Study Arms  ICMJE
  • Experimental: 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
    Interventions:
    • Drug: VX-445
    • Drug: TEZ
    • Drug: IVA
  • Placebo Comparator: Placebo
    Intervention: Drug: Matched Placebos
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 Completed
Actual Enrollment  ICMJE
 (submitted: December 3, 2018)
405
Original Estimated Enrollment  ICMJE
 (submitted: May 2, 2018)
360
Actual Study Completion Date  ICMJE April 24, 2019
Actual Primary Completion Date April 24, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Heterozygous for the F508del mutation (F/MF)
  • Forced expiratory volume in 1 second (FEV1) value ≥40% and ≤90% of predicted mean for age, sex, and height

Key Exclusion Criteria:

  • Clinically significant cirrhosis with or without portal hypertension
  • Lung infection 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 12 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,   Austria,   Belgium,   Canada,   Czechia,   France,   Germany,   Greece,   Italy,   Netherlands,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03525444
Other Study ID Numbers  ICMJE VX17-445-102
2018-000183-28 ( 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 May 2019

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