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?
Yes
IRB#: IRB_00158538
PI: Lorenzo Botto
Department: PEDIATRICS
Approval Date: 2023-04-19 06:00:00
Specialties: Pediatric Genetics