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Clinical Study

Vertex VX-864 in PiZZ Subjects

We are looking for adults between the ages of 18 and 80 with Alpha-1 Antitrypsin Deficiency to participate in this clinical research study. The purpose of this study is to evaluate the safety, tolerability, and effectiveness of an investigational medication in individuals with Alpha-1 Antitrypsin Deficiency. The word investigational means this medication is not yet approved for use by the Food and Drug Administration (FDA) in the United States or other regulatory agencies in Canada, Europe, or elsewhere. Interested individuals will be evaluated during a screening period to determine their eligibility to participate. Your participation could last approximately 1318 weeks depending on whether or not you have ever been on augmentation therapy. Those who qualify will receive all study-related medications and procedures at no cost. You may also be compensated financially for your study-related time and travel.


For more information contact:

Amy Denardo

IRB#: IRB_00134801 | PI: Cheryl Pirozzi | Department: PULMONARY | Approval Date: 2020-08-19 06:00:00
Specialties: Pulmonary

Who can participate?

 Gender: All

 Age: Over 18 years old

 Volunteers: Volunteers with special conditions

 Location: In Person

Inclusion Criteria:

  • Male or female between the ages of 18 and 80 (inclusive)
  • Diagnosed with Alpha-1 Antitrypsin Deficiency
  • Have the genetic pattern most commonly associated with Alpha-1 (the PiZZ genotype)  A genetic blood test will be used to confirm genotype
  • Have never been treated with gene therapy
  • Do not smoke cigarettes and/or use electronic nicotine delivery systems (i.e., electronic cigarettes, vape pens) or chewing tobacco products

Will I be paid for my time?


Last Updated: 4/5/21