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Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

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: NCT04474197
Recruitment Status : Completed
First Posted : July 16, 2020
Results First Posted : May 26, 2022
Last Update Posted : May 26, 2022
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE July 13, 2020
First Posted Date  ICMJE July 16, 2020
Results First Submitted Date  ICMJE May 3, 2022
Results First Posted Date  ICMJE May 26, 2022
Last Update Posted Date May 26, 2022
Actual Study Start Date  ICMJE July 24, 2020
Actual Primary Completion Date May 4, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: May 3, 2022)
  • Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels [ Time Frame: From Baseline at Day 28 ]
  • Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Day 1 up to Week 8 ]
Original Primary Outcome Measures  ICMJE
 (submitted: July 13, 2020)
  • Change in plasma functional alpha-1 antitrypsin (AAT) levels [ Time Frame: From Baseline at Day 28 ]
  • Safety and tolerability as assessed by number of subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From Day 1 up to Week 8 ]
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: May 3, 2022)
  • Change in Plasma Antigenic AAT Levels [ Time Frame: From Baseline at Day 28 ]
  • Observed Pre-dose Plasma Concentration (Ctrough) of VX-864 [ Time Frame: Pre-dose at Day 7, Day 14, Day 21 and Day 28 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: July 13, 2020)
  • Change in plasma antigenic AAT levels [ Time Frame: From Baseline at Day 28 ]
  • Observed pre-dose concentration (Ctrough) of VX-864 [ Time Frame: From Day 1 up to Week 8 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
Official Title  ICMJE A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects
Brief Summary This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Alpha1-Antitrypsin Deficiency
Intervention  ICMJE
  • Drug: VX-864
    Tablets for oral administration.
  • Drug: Placebo
    Placebo matched to VX-864 for oral administration.
Study Arms  ICMJE
  • Placebo Comparator: Placebo
    Participants received placebo matched to VX-864 in the treatment period for 28 days.
    Intervention: Drug: Placebo
  • Experimental: VX-864 100 mg
    Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.
    Intervention: Drug: VX-864
  • Experimental: VX-864 300 mg
    Participants received VX-864 300 mg q12h in the treatment period for 28 days.
    Intervention: Drug: VX-864
  • Experimental: VX-864 500 mg
    Participants received VX-864 500 mg q12h in the treatment period for 28 days.
    Intervention: Drug: VX-864
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: June 15, 2021)
44
Original Estimated Enrollment  ICMJE
 (submitted: July 13, 2020)
40
Actual Study Completion Date  ICMJE May 4, 2021
Actual Primary Completion Date May 4, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Key Inclusion Criteria:

  • Subjects must have a PiZZ genotype confirmed at screening
  • Plasma AAT levels indicating severe deficiency at screening

Key Exclusion Criteria:

  • History of a medical condition that could negatively impact the ability to complete the study
  • Solid organ, or hematological transplantation or is currently on a transplant list
  • History of use of gene therapy or RNAi therapy at any time previously

Other protocol defined Inclusion/Exclusion criteria may apply

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 80 Years   (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 Canada,   Germany,   Ireland,   Sweden,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04474197
Other Study ID Numbers  ICMJE VX19-864-101
2019-004881-16 ( EudraCT Number )
Has Data Monitoring Committee Not Provided
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
Plan to Share IPD: No
Plan Description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing
Current Responsible Party Vertex Pharmaceuticals Incorporated
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date May 2022

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