301 OTC 02 Gene Therapy
A Phase 1/2, A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency
- Sex: Any
- Age: Child (Birth - 17), Adult (18 - 64), Older Adult (65+)
- Accepting Healthy People: No
- Type: Others
- Trial Phase: Phase I/II
- Conditions Being Studied:
Study Purpose
OTC Deficiency is an inherited disorder that causes ammonia to accumulate in the blood (hyperammonemia). The purpose of this clinical research study is to learn more about the long-term safety and efficacy of the experimental gene transfer product, DTX301 as a treatment for OTC Deficiency. You may participate in this study only if you have previously received a single dose of DTX301 in the 301OTC01 study.
Who Can Participate
Previously received a single dose of DTX301 in the 301OTC01 study.
Locations
UH Cleveland Medical Center
11100 Euclid Ave
Cleveland OH, 44106
- UH IRB: STUDY20210213
- StudyID: 2021-0566
- ClinicalTrials.gov: NCT02991144
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