Brief Title
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
Official Title
A Phase 2, Randomized, Double-blind, Placebo-controlled Study of the Efficacy and Safety of VX-814 in PiZZ Subjects
Brief Summary
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 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
Alpha 1-Antitrypsin Deficiency
Intervention
VX-814
Study Arms / Comparison Groups
Parts A1, A2 and B Combined: Placebo
Description: Participants received placebo matched to VX-814 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
48
Start Date
January 13, 2020
Completion Date
November 14, 2020
Primary Completion Date
November 14, 2020
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
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Location Countries
Canada
Location Countries
Canada
Administrative Informations
NCT ID
NCT04167345
Organization ID
VX19-814-101
Responsible Party
Sponsor
Study Sponsor
Vertex Pharmaceuticals Incorporated
Study Sponsor
, ,
Verification Date
January 2022