MedTrace Logo

Gene Therapy for Alpha 1- Antitrypsin Deficiency

NCT06996756 · Status: RECRUITING · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2026-03-13

No results posted yet for this study

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.

Conditions

Interventions

BIOLOGICAL

AAV8hAAT(AVL)

AAV8hAAT(AVL) gene transfer vector

Sponsors & Collaborators

  • National Heart, Lung, and Blood Institute (NHLBI)

    collaborator NIH

  • Weill Medical College of Cornell University

    lead OTHER

Principal Investigators

  • Ronald G Crystal, MD · Weill Medical College of Cornell University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
18 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2025-02-26
Primary Completion
2028-04-30
Completion
2032-08-01
FDA Drug
Yes

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06996756 on ClinicalTrials.gov