Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ADVM-043 · 1 trial · 1 indication
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...
ADVM-043 is an investigational gene therapy being developed for the treatment of Alpha 1-Antitrypsin Deficiency, a rare genetic condition. It is designed to address the underlying cause of the disease by delivering a functional gene to the body. The therapy is currently in clinical development and has not been approved by regulatory authorities.
ADVM-043 is being developed by Adverum Biotechnologies, Inc., a biopharmaceutical company. The company is conducting clinical research to evaluate the safety and efficacy of this gene therapy for Alpha 1-Antitrypsin Deficiency. Adverum Biotechnologies is publicly traded under the ticker symbol ADVM.
ADVM-043 is in Phase 1 clinical development. It has completed a Phase 1 trial that evaluated safety and dose finding in patients with Alpha 1-Antitrypsin Deficiency. The therapy remains investigational and is not yet approved for commercial use.
ADVM-043 has been studied in one completed Phase 1 clinical trial, identified as NCT02168686. This trial was a safety and dose-finding study conducted in the United States, enrolling 6 participants with Alpha 1-Antitrypsin Deficiency. The study was controlled but not randomized or double-blinded.
ADVM-043 is a gene therapy designed to treat Alpha 1-Antitrypsin Deficiency by delivering a functional copy of the gene responsible for producing alpha-1 antitrypsin protein. This approach aims to restore normal protein levels in the body, potentially addressing the underlying genetic cause of the disease.