Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04474197 | Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype | PHASE2 | COMPLETED | 44 | — | — | Jul 24, 2020 | May 4, 2021 | May 26, 2022 | 30 | United States, Canada +4 |
| Arm | Type | Description |
|---|---|---|
| Placebo | PLACEBO_COMPARATOR | Participants received placebo matched to VX-864 in the treatment period for 28 days. |
| VX-864 100 mg | EXPERIMENTAL | Participants received VX-864 100 milligrams (mg) every 12 hours (q12h) in the treatment period for 28 days. |
| VX-864 300 mg | EXPERIMENTAL | Participants received VX-864 300 mg q12h in the treatment period for 28 days. |
| VX-864 500 mg | EXPERIMENTAL | Participants received VX-864 500 mg q12h in the treatment period for 28 days. |
| Name | Type | Description |
|---|---|---|
| VX-864 | DRUG | Tablets for oral administration. |
| Placebo | DRUG | Placebo matched to VX-864 for oral administration. |
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...