One-Shot gene therapy aims to tame rare metabolic disorder
NCT ID NCT05345171
Summary
This study is testing whether a single injection of an experimental gene therapy, called DTX301, can help the body safely process ammonia 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 injection and followed for several years. The main goal is to see if the treatment can improve the body's natural ability to control ammonia levels without the need for constant dietary and medication management.
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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
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