Gene Therapy for Alpha 1- Antitrypsin Deficiency

Part of paid clinical trials in New York, New York.

Sponsor
Weill Medical College of Cornell University
Study ID
NCT06996756
Phase
PHASE1
Status
Recruiting
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Conditions

  • Alpha 1-Antitrypsin Deficiency

Eligibility Criteria

Sex
ALL
Age
18 Years - 70 Years
Healthy Volunteers
Not accepted

Interventions

  • AAV8hAAT(AVL) — BIOLOGICAL
    AAV8hAAT(AVL) gene transfer vector

Study Details

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.

Key Dates

Start date
Feb 26, 2025
Status verified
Mar 2026
Primary completion
Apr 30, 2028
Completion
Aug 1, 2032

Study Design

Enrollment
16 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: AAV8hAAT(AVL) - 5x10¹¹ gc/kg
    Lowest dose of vector genome copies per kilogram
  • Experimental: AAV8hAAT(AVL) - 2x10¹² gc/kg
  • Experimental: AAV8hAAT(AVL) - 5x10¹² gc/kg
  • Experimental: AAV8hAAT(AVL) - 2x10¹³ gc/kg
    Highest dose of vector genome copies per kilogram

Primary Outcome Measure

Safety of AAV8hAAT(AVL), as measured by number of subjects with at least 1 serious adverse event. [ Time Frame: Approximately 1 year ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
WCMC Department of Genetic MedicineNew YorkNew York10021
Sandra Hyde
[email protected]
646-962-2672
Niamh Savage
[email protected]
646-962-5527
Ronald Crystal, MD (PRINCIPAL_INVESTIGATOR)

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By research site
WCMC Department of Genetic Medicine· New York, NY

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