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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

Study Description
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Brief Summary:
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.

Condition or disease Intervention/treatment Phase
Alpha1-Antitrypsin Deficiency Drug: VX-864 Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 44 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-864 in PiZZ Subjects
Actual Study Start Date : July 24, 2020
Actual Primary Completion Date : May 4, 2021
Actual Study Completion Date : May 4, 2021

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Placebo Comparator: Placebo
Participants received placebo matched to VX-864 in the treatment period for 28 days.
 Drug: Placebo
Placebo matched to VX-864 for oral administration.
Experimental: VX-864 100 mg
Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days.
 Drug: VX-864
Tablets for oral administration.
Experimental: VX-864 300 mg
Participants received VX-864 300 mg q12h in the treatment period for 28 days.
 Drug: VX-864
Tablets for oral administration.
Experimental: VX-864 500 mg
Participants received VX-864 500 mg q12h in the treatment period for 28 days.
 Drug: VX-864
Tablets for oral administration.


Outcome Measures
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Primary Outcome Measures :
  1. Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels [ Time Frame: From Baseline at Day 28 ]
  2. 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 ]

Secondary Outcome Measures :
  1. Change in Plasma Antigenic AAT Levels [ Time Frame: From Baseline at Day 28 ]
  2. Observed Pre-dose Plasma Concentration (Ctrough) of VX-864 [ Time Frame: Pre-dose at Day 7, Day 14, Day 21 and Day 28 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04474197


Locations
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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
  Study Documents (Full-Text)

Documents provided by Vertex Pharmaceuticals Incorporated:
Study Protocol  [PDF] January 12, 2021
Statistical Analysis Plan  [PDF] May 11, 2021

More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT04474197    
Other Study ID Numbers: VX19-864-101
2019-004881-16 ( EudraCT Number )
First Posted: July 16, 2020    Key Record Dates
Results First Posted: May 26, 2022
Last Update Posted: May 26, 2022
Last Verified: May 2022
Individual Participant Data (IPD) Sharing Statement:
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Alpha 1-Antitrypsin Deficiency
Liver Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
 Genetic Diseases, Inborn
Subcutaneous Emphysema
Emphysema
Pathologic Processes