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Evaluation of the Efficacy and Safety of VX-814 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. Identifier: NCT04167345
Recruitment Status : Terminated (at Sponsor's discretion)
First Posted : November 18, 2019
Last Update Posted : November 30, 2020
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Brief Summary:
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.

Condition or disease Intervention/treatment Phase
Alpha 1-Antitrypsin Deficiency Drug: VX-814 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 48 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-814 in PiZZ Subjects
Actual Study Start Date : January 13, 2020
Actual Primary Completion Date : November 14, 2020
Actual Study Completion Date : November 14, 2020

Arm Intervention/treatment
Experimental: VX-814
Subjects will be randomized to receive different dose levels of VX-814.
Drug: VX-814
Tablet for oral administration.

Subjects will receive placebo matched to VX-814.
Drug: Placebo
Placebo matched to VX-814 for oral administration.

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 subjects with adverse events (AEs) and serious adverse events (SAEs) [ Time Frame: From 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 concentration (Ctrough) of VX-814 [ Time Frame: From Day 1 up to Week 8 ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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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

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

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 identifier (NCT number): NCT04167345

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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
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Responsible Party: Vertex Pharmaceuticals Incorporated Identifier: NCT04167345    
Other Study ID Numbers: VX19-814-101
First Posted: November 18, 2019    Key Record Dates
Last Update Posted: November 30, 2020
Last Verified: November 2020
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:

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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
Pathologic Processes