drugset / Trial / NCT06996756
Gene Therapy for Alpha 1- Antitrypsin Deficiency
Phase 1
Recruiting
16 enrolled
Weill Medical College of Cornell University
National Heart, Lung, and Blood Institute (NHLBI) · collab
Non-randomizedSequentialOpen-labelTreatment
Summary
This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) 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 deficient individuals.
Timeline
- Start
- 2025-02-26
- Primary completion
- 2028-04-30
- Completion
- 2032-08-01
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | AAV8hAAT(AVL) | Unknown | 5e+11 vg/kg | Intravenous |
| Subject | AAV8hAAT(AVL) | Unknown | 2e+12 vg/kg | Intravenous |
| Subject | AAV8hAAT(AVL) | Unknown | 5e+12 vg/kg | Intravenous |
| Subject | AAV8hAAT(AVL) | Unknown | 2e+13 vg/kg | Intravenous |