Late-Onset OTC Deficiency - Healing Genes

Late-Onset OTC Deficiency

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Safety and Dose-Finding Study of DTX301 (scAAV8OTC) in Adults With Late-Onset OTC Deficiency (CAPtivate)

A Phase 1/2, Open-Label Safety and Dose-Finding Study of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults With Late-Onset OTC Deficiency

Phase 1/2


Doctors at university medical centers in CA, CO, MA, NY, OH, and OR are recruiting adult patients with late-onset Ornithine transcarbamylase (OTC) deficiency to trial an investigative gene therapy, DTX301. The therapy involves a single infusion of a genetically engineered virus designed to transfer a functional version of the OTC gene. This replacement gene may produce OTC and achieve a meaningful increase the urea cycle in the liver.

A single infusion of the drug at one of 3 dosages is required, with follow up for 1 year. Subjects will be followed for 52 weeks and then asked to enroll in a 4-year extension study to evaluate the long term (a total of 5 years) safety and efficacy of DTX301.


  • Be 18 years of age or older
  • Diagnosis of late-onset OTC with confirmation by molecular testing
  • Be on stable dose of ammonia scavenger therapy for ≥4 weeks


  1. Prescreening tests to confirm eligibility of the patient to participate.
  2. A single intravenous administration of the trial drug.
  3. Follow up over the year after treatment.

The following study sites are coordinating:
Ronald Reagan UCLA Medical Center in Los Angeles, CA, Map.
University of California San Francisco in San Francisco, CA, Map.
The Children’s Hospital Colorado in Aurora, CO, Map.
Boston Children’s Hospital in Boston, MA, Map.
Icahn School of Medicine at Mount Sinai in New York, NY, Map.
University Hospital Cleveland Medical Center/Case Western Reserve University in Cleveland, OH, Map.
Oregon Health and Science University in Portland, OR, Map.

Patient Advocacy  |  1-415-483-8800  |  [email protected]
HCPs Contact  |  1-888-756-8657  |  [email protected]

Ultragenyx Pharmaceutical Inc

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