Ignite Creation Date:
2025-12-24 @ 11:32 PM
Ignite Modification Date:
2026-01-01 @ 6:05 AM
Study NCT ID:
NCT06996756
Status:
RECRUITING
Last Update Posted:
2025-09-23
First Post:
2025-05-21
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Gene Therapy for Alpha-1 Antitrypsin Deficiency
Sponsor:
Weill Medical College of Cornell University
Organization:
Study Overview
Official Title:
Gene Therapy for Alpha-1 Antitrypsin Deficiency
Status:
RECRUITING
Status Verified Date:
2025-09
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
This is a study of gene therapy to treat alpha-1 antitrypsin deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of the AAT protein, which if safe and if efficacious, will protect the lung on a persistent basis. We hope to learn the safety/toxicity and initial evidence of efficacy of intravenous delivery of this gene therapy to alpha 1-antitrypsin (AAT) deficient individuals.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
False
Is an FDA AA801 Violation?:
Secondary ID Infos
| Secondary ID | Type | Domain | Link | View |
|---|---|---|---|---|
| 1R61HL169190 | NIH | None | https://reporter.nih.gov/quic… | View |