Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
belcesiran · 1 trial · 1 indication
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...
Belcesiran is an investigational small molecule being developed for Alpha 1-Antitrypsin Deficiency, a rare genetic condition. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities.
Belcesiran is being developed by Novo Nordisk A/S, a pharmaceutical company listed on the stock exchange under the ticker symbol NVO. The drug is currently in Phase 1 clinical trials.
Belcesiran is in Phase 1 clinical development. It is an investigational drug, meaning it has not yet been approved for use in patients. The drug is being studied for the treatment of Alpha 1-Antitrypsin Deficiency.
Belcesiran has one completed clinical trial, identified as NCT04174118. This was a Phase 1 study in healthy adult volunteers, conducted in New Zealand and Sweden, with 30 participants. The trial was randomized, double-blind, and placebo-controlled.
Belcesiran is not FDA approved. It is an investigational drug currently in Phase 1 clinical development for Alpha 1-Antitrypsin Deficiency. The drug has not completed the clinical trials and regulatory review process required for approval.