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.|
|ClinicalTrials.gov Identifier: NCT04167345|
Recruitment Status : Terminated (at Sponsor's discretion)
First Posted : November 18, 2019
Last Update Posted : November 30, 2020
|Condition or disease||Intervention/treatment||Phase|
|Alpha 1-Antitrypsin Deficiency||Drug: VX-814 Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||48 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)|
|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|
Subjects will be randomized to receive different dose levels of VX-814.
Tablet for oral administration.
Subjects will receive placebo matched to VX-814.
Placebo matched to VX-814 for oral administration.
- 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 in plasma antigenic AAT levels [ Time Frame: From Baseline at Day 28 ]
- Observed pre-dose concentration (Ctrough) of VX-814 [ Time Frame: From Day 1 up to Week 8 ]
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): NCT04167345