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Study Details

VX-864: A Study Drug for People with Alpha-1 Antitrypsin Deficiency who have the PiZZ Genotype

(IRB#: IRB_00163260)

Alpha-1 Antitrypsin (ATT) Deficiency with the PiZZ Genotype is a genetic disorder that may result in severe health issues such as lung or liver disease. VX-864 is a study drug to improve the health of people with this condition. Research is needed to learn the safety and effectiveness of VX-864. The information we gain may aid future patients.


  • All genders
  • Over 18 years old
  • Volunteers with special conditions
  •   In Person
  • Paid

Who can participate?

 Gender: All genders

  Age: Over 18 years old

  Volunteers: Volunteers with special conditions

   Location: In Person

Inclusion Criteria

  • Ages 18 to 80 years old
  • PiZZ genotype diagnoses
  • Able to attend in person at the University of Utah

Exclusion Criteria

  • Pulmonary (lung) diseases such as lung cancer
  • Unable to stop augmentation therapy (use of healthy blood from others to improve the amount of ATT in the lungs)
  • Use of steroids for more than 3 months starting the study
  • Significant alcohol use 3 months before starting the study
  • Use of inhaled cannabis or cigarette smoking 6 months before starting the study or use of illegal drugs 1 year before starting the study

Will I be paid for my time?


For more information contact:

Cassie Larsen


IRB#: IRB_00163260

PI: Cheryl Pirozzi

Department: PULMONARY

Approval Date: 2023-05-03 06:00:00

Specialties: Pulmonary

Last Updated: 6/8/23