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 |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04174118 | Study of DCR-A1AT in Healthy Adult Volunteers | PHASE1 | COMPLETED | 30 | — | — | Oct 24, 2019 | Mar 6, 2023 | Nov 6, 2024 | 2 | New Zealand, Sweden |
The incidence of adverse events (AE), serious adverse events (SAE), DLT, and AE leading to study drug discontinuation
The incidence of clinically significant physical examination (PE) findings
Absolute QTc \> 500 msec and/or QTc change of \> 60 msec from baseline will be evaluated
| Arm | Type | Description |
|---|---|---|
| belcesiran | EXPERIMENTAL | Healthy volunteers will be administered a single dose of belcesiran. |
| Placebo | PLACEBO_COMPARATOR | Healthy volunteers will be administered a single dose of matching placebo. |
| Name | Type | Description |
|---|---|---|
| belcesiran | DRUG | belcesiran will be administered subcutaneously (SC) at dose levels planned. |
| Placebo | DRUG | Sterile normal saline (0.9% NaCL) matching volume of belcesiran doses will be administered subcutaneously (SC). |
Inclusion Criteria: * Male or Female aged 18 to 55 years, inclusive. Female participants must be either surgically sterile or postmenopausal. No women of childbearing potential are eligible for enrollment. * Overtly Healthy, as determined by the investigator. * Serum A1AT protein concentration \>10...