Ultragenyx DTX301-CL301
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated Virus Serotype 8 (AAV8) Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-Onset OTC Deficiency
- Sex: Any
- Age: Child (Birth - 17), Adult (18 - 64)
- Accepting Healthy People: No
- Type: Interventional / Therapeutic
- Trial Phase: Phase III
- Conditions Being Studied:
Study Purpose
OTC deficiency is an inherited disorder that causes ammonia to accumulate in the blood (hyperammonemia). The purpose of this research study is to assess if an investigational gene transfer product, called DTX301 (avalotcagene ontaparvovec) is an effective and safe treatment for adults and children aged 12 years and older with late-onset ornithine transcarbamylase (OTC) deficiency.
Who Can Participate
Age: 12 years and older
Locations
UH Cleveland Medical Center
11100 Euclid Ave
Cleveland OH, 44106
- UH IRB: STUDY20211194
- StudyID: 2021-0739
- ClinicalTrials.gov: NCT00338425
Questions or concerns? We're here to help.
Non-cancer Trials: 1-833-78TRIAL or Contact Us Online
Cancer Trials: 1-800-641-2422