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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?


For more information contact:

Carrie Bailey


IRB#: IRB_00150121

PI: Nicola Longo


Approval Date: 2023-03-22 06:00:00

Specialties: Pediatric Genetics

Last Updated: 6/8/23