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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT02168686 | Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency | PHASE1 | COMPLETED | 6 | — | — | Nov 28, 2017 | Aug 29, 2019 | Oct 5, 2023 | 2 | United States |
Number and proportion of subjects experiencing treatment-related adverse events related to ADVM-043
Number of participants with ≥1 abnormal shift from Baseline in neutrophil count, hemoglobin, important serum chemistry parameters
| Arm | Type | Description |
|---|---|---|
| Part A: Dose 1 | EXPERIMENTAL | ADVM-043, at the lowest dose of three planned dose levels, of 8E13 total vg (equivalent to 1E12 vg/kg based on an 80-kg patient) administered IV |
| Part A: Dose 2 | EXPERIMENTAL | ADVM-043 at the intermediate dose of three planned dose levels, of 4E14 total vg (equivalent to 5E12 vg/kg based on an 80-kg patient) administered IV |
| Part A: Dose 3 | EXPERIMENTAL | ADVM-043 at the highest dose of three planned dose levels, of 1.2E15 total vg (equivalent to 1.5E13 vg/kg based on an 80-kg patient) administered IV |
| Part A: Dose 4 | EXPERIMENTAL | ADVM-043 administered at a dose that will be determined |
| Part B (optional): Intrapleural administration | EXPERIMENTAL | ADVM-043 administered intrapleurally at a dose that will be determined |
| Name | Type | Description |
|---|---|---|
| ADVM-043 | GENETIC | Gene transfer vector administration |
Key Inclusion Criteria: * Capable of providing informed consent * Alpha1AT genotype of ZZ or Z Null * Males and females 18 years and older * Ongoing treatment with A1AT augmentation is not required, however any subject receiving A1AT augmentation therapy must be willing to washout. Washout is defin...