Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
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|ClinicalTrials.gov Identifier: NCT04167345|
Recruitment Status : Recruiting
First Posted : November 18, 2019
Last Update Posted : October 9, 2020
|Condition or disease||Intervention/treatment||Phase|
|Alpha 1-Antitrypsin Deficiency||Drug: VX-814 Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||50 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|
|Estimated Primary Completion Date :||November 2020|
|Estimated Study Completion Date :||November 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
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