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A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)

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ClinicalTrials.gov Identifier: NCT03460990
Recruitment Status : Completed
First Posted : March 9, 2018
Results First Posted : October 17, 2019
Last Update Posted : October 17, 2019
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE March 1, 2018
First Posted Date  ICMJE March 9, 2018
Results First Submitted Date  ICMJE September 26, 2019
Results First Posted Date  ICMJE October 17, 2019
Last Update Posted Date October 17, 2019
Actual Study Start Date  ICMJE May 1, 2018
Actual Primary Completion Date September 26, 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 26, 2019)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [ Time Frame: From Baseline at Week 4 ]
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.
Original Primary Outcome Measures  ICMJE
 (submitted: March 7, 2018)
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) [ Time Frame: From Baseline at Week 4 ]
Change History Complete list of historical versions of study NCT03460990 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 26, 2019)
  • Absolute Change in Sweat Chloride (SwCl) [ Time Frame: From Baseline at Week 4 ]
    Sweat samples were collected using an approved collection device.
  • Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score [ Time Frame: From Baseline at Week 4 ]
    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) [ Time Frame: From first dose of study drug in TC treatment period up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first (up to Week 8) ]
  • Observed Pre-Dose Concentration (Ctrough) of VX-659, TEZ, TEZ Metabolite (M1-TEZ), and IVA [ Time Frame: From Day 1 and Week 4 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: March 7, 2018)
  • Absolute change in CF Questionnaire-Revised (CFQ-R) respiratory domain score [ Time Frame: From Baseline at Week 4 ]
  • Absolute Change in Sweat Chloride (SwCl) [ Time Frame: From Baseline at Week 4 ]
  • 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 to12 weeks) ]
  • Observed pre-dose concentration (Ctrough) of VX-659, TEZ, M1-TEZ, and IVA [ Time Frame: from Day 1 through Week 4 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of VX-659 Combination Therapy in CF Subjects Homozygous for F508del (F/F)
Official Title  ICMJE A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)
Brief Summary This study will evaluate the efficacy of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation (F/F).
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-659/TEZ/IVA
    Participants received VX-659/TEZ/IVA orally once daily in the morning.
    Other Names:
    • VX-659/VX-661/VX-770
    • VX-659/tezacaftor/ivacaftor
  • Drug: TEZ/IVA
    Participants received TEZ/IVA orally once daily in the morning.
    Other Names:
    • VX-661/VX-770
    • tezacaftor/ivacaftor
  • Drug: IVA
    Participants received IVA orally once daily in the evening.
    Other Names:
    • VX-770
    • ivacaftor
  • Drug: Placebo
    Participants received placebo matched TEZ/IVA orally once daily in the morning.
  • Drug: Placebo
    Participants received placebo matched to VX-659/TEZ/IVA orally once daily in the morning.
Study Arms  ICMJE
  • Active Comparator: TEZ/IVA
    Following a run-in period of 4 weeks with Tezacaftor (TEZ)/Ivacaftor (IVA), participants received TEZ 100 milligram (mg)/IVA 150 mg as fixed-dose combination (FDC) tablet in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the triple combination (TC) treatment period.
    Interventions:
    • Drug: TEZ/IVA
    • Drug: IVA
    • Drug: Placebo
  • Experimental: VX-659/TEZ/IVA TC
    Following a run-in period of 4 weeks with TEZ/IVA, participants received VX-659 240 mg/TEZ 100 mg/IVA 150 mg as FDC tablets in the morning and IVA 150 mg as mono tablet in the evening for 4 weeks in the TC treatment period.
    Interventions:
    • Drug: VX-659/TEZ/IVA
    • Drug: IVA
    • Drug: Placebo
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)
116
Original Estimated Enrollment  ICMJE
 (submitted: March 7, 2018)
100
Actual Study Completion Date  ICMJE October 8, 2018
Actual Primary Completion Date September 26, 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Homozygous for the F508del mutation (F/F)
  • 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,   Germany,   Ireland,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03460990
Other Study ID Numbers  ICMJE VX17-659-103
2017-004133-82 ( 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 September 2019

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