Last updated on December 2018

Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency


Are you eligible to participate in this study?

You may be eligible for this study if you meet the following criteria:

  • Conditions: alpha-1-Antitrypsin deficiency
  • Age: Between 18 - 100 Years
  • Gender: Male or Female

Key Inclusion Criteria:

  • Capable of providing informed consent
  • Alpha1AT genotype of ZZ or Z Null
  • Males and females 18 years and older
  • Ongoing treatment with A1AT augmentation is not required, however any subject receiving A1AT augmentation therapy must be willing to washout. Washout is defined as at least 8 weeks between last augmentation therapy and pre-treatment plasma A1AT level
  • Willing to remain off PAT for at least 3 months following treatment
  • Body mass index 18 to 35 kg/m2
  • Fertile men and women of childbearing potential must agree to use barrier contraception for 3 months after treatment

Key Exclusion Criteria:

  • FEV1 <35 percent of predicted value at the Screening visit
  • Receiving systemic corticosteroids or other immunosuppressive medications
  • Immunodeficiency disease or evidence of active infection of any type, including human immunodeficiency virus
  • Abnormal liver function tests
  • Organ transplant recipient or awaiting transplantation
  • Participation in another current or previous gene transfer study
  • AAVrh.10 neutralizing antibody titer 1:5
  • Female who is pregnant or lactating
  • History of alcohol or drug abuse within the past 5 years
  • Any history of allergies that may prohibit study-specific investigations
  • Receiving an investigational medicinal product or participating in another investigational study within 3 months prior to consent
  • Cigarette smoking, or any other tobacco use, e-cigarettes or other recreational inhalant within 1 year of the Screening Visit

Recruitment Status: Closed


Brief Description Eligibility Contact Research Team


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