Gene Therapy for Alpha 1- Antitrypsin Deficiency

Recruiting Phase 1 Interventional Study
Alpha 1-Antitrypsin Deficiency
No Placebo Group Every participant receives an active treatment — no one gets a placebo.
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At a Glance
Age
18 – 70
Sex
Any
Trial phase
Phase 1
Study type
Interventional
Purpose
Treatment
Participants needed
16 (estimated)
Sponsor
Weill Medical College of Cornell University · Other
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About This Trial
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 intr…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * AAT genotype ZZ, or Z null heterozygotes, and if on augmentation therapy, pre-therapy AAT serum levels \<11 μM * Emphysema as assessed by chest high resolution computational tomography (HRCT) * Lung function parameters consistent with mild to moderate loss of lung function and…
Contacts

Niamh Savage

646-962-5527

nis2049@med.cornell.edu

CONTACT

Sandra Hyde

646-962-2672

sah2003@med.cornell.edu

CONTACT