One-Shot gene therapy aims to fix rare metabolic disorder
NCT ID NCT05345171
Summary
This study is testing whether a single dose of an experimental gene therapy called DTX301 can help the body control ammonia levels in people with late-onset OTC deficiency. About 32 participants, aged 12 and older, will be randomly assigned to receive either the gene therapy or a placebo and followed for up to 6 years. The main goal is to see if the treatment can safely improve the liver's ability to process ammonia, potentially reducing the need for strict diets and medications.
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Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Centro Hospitalar Universitario de Sao Joao
Porto, 4200-319, Portugal
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Clinica Universitaria Reina Fabiola
Córdoba, X5004, Argentina
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Erasmus Universitair Medisch Centrum Rotterrdam
Rotterdam, 3015, Netherlands
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Fujita Health University Hospital
Toyoake, 470-1192, Japan
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Fundacio Hospital Universitari Vall D'Hebron-Institute de Recerca
Barcelona, 08035, Spain
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Hopital Femme Mere Enfant
Bron, 69500, France
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Hospital Italiano de Buenos Aires
Buenos Aires, C1199, Argentina
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Hospital de Clinicas de Porto Alegre
Porto Alegre, 90035-903, Brazil
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Kumamoto University Hospital
Kumamoto, 860-8556, Japan
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Necker-Enfants Maladas Hospital
Paris, 75015, France
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The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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Universitatsklinikum Heidelberg
Heidelberg, 69120, Germany
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University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106, United States
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University of California
Los Angeles, California, 90095, United States
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University of Colorado
Aurora, Colorado, 80045, United States
Conditions
Explore the condition pages connected to this study.