Study Details
DTX301-CL301: Gene Therapy for Late-onset Ornithine Transcarbamylase Deficiency (OTC deficiency)
(IRB#: IRB_00150121)
Ornithine Tanscarbamylase Deficiency (OTC deficiency) is an inherited disorder that causes toxic levels in the blood. The disease increases the risk of mental health problems and other health issues. DTX301-CL301 is a study drug used for gene therapy. Research is needed to learn the safety and effectiveness of the treatment. The information we gain may aid future patients.
- All genders
- Over 7 years old
- Volunteers with special conditions
- In Person
- Paid
Who can participate?
Gender: All genders
Age: Over 7 years old
Volunteers: Volunteers with special conditions
Location: In Person
Inclusion Criteria
- Ages 12 and older
- Diagnosis of late-onset OTC deficiency
- History of hyperammonemia episode
- Use of specific birth control during study participation
- In-person at the University of Utah
Exclusion Criteria
- Liver transplant
- History of liver disease
- Evidence of hepatitis B or hepatitis C
- History of autoimmune disease
- Pregnant or breastfeeding
Will I be paid for my time?
Yes
IRB#: IRB_00150121
PI: Nicola Longo
Department: PEDIATRIC GENETICS
Approval Date: 2023-03-22 06:00:00
Specialties: Pediatric Genetics