Study Details
A Study Using AMT-191 Gene Therapy in Adult Males with Classic Fabry Disease
(IRB#: IRB_00180825)
Fabry disease is a rare genetic condition that keeps the body from breaking down certain fatty materials that build up in cells. This can result in health issues such as pain in the hands and feet and damage to the kidneys, brain, and heart. This study will test a drug called AMT-191, a gene therapy drug. A gene therapy drug is designed to provide the genetic material not functioning correctly in the body. The study wants to see if the drug will help people and is safe. People in the study will be given a single drug dose through a vein. Participation requires attending in-person visits at the study clinic, which can last about two years. Medical tests will be done during the study to track participants' health.
- Male Only
- Over 18 years old
- Volunteers with special conditions
- In Person
- Paid
Who can participate?
Gender: Male Only
Age: Over 18 years old
Volunteers: Volunteers with special conditions
Location: In Person
Inclusion Criteria
- Ages 18 to 50 years old
- Assigned male gender at birth
- Diagnosis of Fabry disease with specific details
- Use birth control during the study period
- Able to attend in person at the University of Utah
Exclusion Criteria
- Previous study drug use 3 months before participation
- Previous gene therapy treatment
- Planning to receive another treatment for Fabry disease
- Specific active or ongoing infection
- Liver disease or moderate to severe heart disease
Will I be paid for my time?
Yes
IRB#: IRB_00180825
PI: Brian Shayota
Department: PEDIATRICS
Approval Date: 2025-01-08 07:00:00
Specialties: Pediatric Genetics
I am Interested