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Study Details

INZ-701: A Study Drug for Infants with Generalized Arterial Calcification of Infancy (GACI) due to ENPP1 Deficiency

(IRB#: IRB_00158538)

Generalized Arterial Calcification of Infancy (GACI) is a rare genetic disease that causes calcium buildup in the blood vessels throughout the body. This calcium buildup narrow these blood vessels, and especially the arteries, which often leads to high blood pressure, heart disease, strokes, and kidney disease. One of the main genetic causes of GACI is the lack of an enzyme called ENPP1. INZ-701, is a study drug to help infants with this ENPP1 Deficiency. Research is needed to learn the safety and effectiveness of INZ-701. The information we gain may aid future patients. INZ-701 is a medication given under the skin. In this study, participants would receive study medication at least once a week. Study visits are in person and weekly for the first month and then every 4 weeks.


  • All genders
  • Under 7 years old
  • Volunteers with special conditions
  •   In Person
  • Paid

Who can participate?

 Gender: All genders

  Age: Under 7 years old

  Volunteers: Volunteers with special conditions

   Location: In Person

Inclusion Criteria

  • Ages 0 to 1 year old
  • Caregiver and their infant
  • Diagnosis of ENPPI Deficiency
  • Able and willing to stop taking other specific drugs
  • In-person at the University of Utah

Exclusion Criteria

  • Current participation in a similar study

Will I be paid for my time?


For more information contact:

Carrie Bailey


IRB#: IRB_00158538

PI: Lorenzo Botto

Department: PEDIATRICS

Approval Date: 2023-04-19 06:00:00

Specialties: Pediatric Genetics

Last Updated: 6/8/23