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

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Brief Title

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

Official Title

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.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Change in Plasma Functional Alpha-1 Antitrypsin (AAT) Levels

Secondary Outcome

 Change in Plasma Antigenic AAT Levels

Condition

Alpha1-Antitrypsin Deficiency

Intervention

VX-864

Study Arms / Comparison Groups

 Placebo
Description:  Participants received placebo matched to VX-864 in the treatment period for 28 days.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

44

Start Date

July 24, 2020

Completion Date

May 4, 2021

Primary Completion Date

May 4, 2021

Eligibility 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
      

Gender

All

Ages

18 Years - 80 Years

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Canada

Location Countries

Canada

Administrative Informations


NCT ID

NCT04474197

Organization ID

VX19-864-101

Secondary IDs

2019-004881-16

Responsible Party

Sponsor

Study Sponsor

Vertex Pharmaceuticals Incorporated


Study Sponsor

, , 


Verification Date

May 2022