Drugs / ADVM-043
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT02168686 | Nov 2017 → Aug 2019 | alpha 1-antitrypsin deficiency | Adverum Biotechnologies, Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 1
| Date | Issuer | Release |
|---|---|---|
| 2018-11-01 | Adverum Biotechnologies, Inc. | Results Adverum Biotechnologies Provides Program Updates adverum.com ↗
Preliminary data from the ADVANCE Phase 1/2 Study in A1AT deficiency showed ADVM-043 in doses of up to 1.5 x 10 13 vg/kg were safely administered and well tolerated; Protein expression did not meet a clinically meaningful level |
All press releases naming this drug 3 releases
| Date | Issuer | Release |
|---|---|---|
| 2018-11-01 | Adverum Biotechnologies, Inc. | Adverum Biotechnologies Provides Program Updates adverum.com ↗ |
| 2018-02-26 | Adverum Biotechnologies, Inc. | Adverum Biotechnologies Announces Completion of Dosing of First Cohort of Patients in the ADVANCE Phase 1/2 Clinical Trial of ADVM-043 Gene Therapy for A1AT Deficiency adverum.com ↗ |
| 2017-12-28 | Adverum Biotechnologies, Inc. | Adverum Biotechnologies Doses First Patient in the ADVANCE Phase 1/2 Clinical Trial of ADVM-043 for A1AT Deficiency adverum.com ↗ |
Evidence & citations 4 cited values
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | ADVM-043 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT02168686 ↗ |
| Action | Restore | “intended to deliver a functional gene to the liver of patients with A1AT deficiency” NCT02168686 ↗ |
| Modality | Gene therapy (AAV / viral vector) | “ADVM-043 is an investigational gene therapy product (serotype AAVrh.10 vector)” NCT02168686 ↗ |
| Route | Intravenous | “administered IV” NCT02168686 ↗ |