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.
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