DTX301-CL301: Gene Therapy for Late-onset Ornithine Transcarbamylase Deficiency (OTC deficiency)
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
Who can participate?
Gender: All genders
Age: Over 7 years old
Volunteers: Volunteers with special conditions
Location: In Person
- 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
- 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?