Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
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|ClinicalTrials.gov Identifier: NCT04474197|
Recruitment Status : Recruiting
First Posted : July 16, 2020
Last Update Posted : January 22, 2021
|Condition or disease||Intervention/treatment||Phase|
|Alpha1-Antitrypsin Deficiency||Drug: VX-864 Drug: Placebo||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||40 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-864 in PiZZ Subjects|
|Actual Study Start Date :||July 24, 2020|
|Estimated Primary Completion Date :||May 2021|
|Estimated Study Completion Date :||May 2021|
Subjects will be randomized to receive different dose levels of VX-864.
Tablets for oral administration.
Placebo Comparator: Placebo
Subjects will receive placebo matched to VX-864.
Placebo matched to VX-864 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-864 [ 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): NCT04474197
|Contact: Medical Informationfirstname.lastname@example.org|