Gene therapy aims to tame deadly ammonia spikes in rare disease
NCT ID NCT05345171
First seen Nov 01, 2025 · Last updated May 14, 2026 · Updated 21 times
Summary
This study tests a one-time gene therapy (DTX301) for adults with late-onset OTC deficiency, a genetic disorder that causes dangerous ammonia buildup in the blood. The goal is to safely keep ammonia levels normal without long-term medication. About 32 participants will receive either the gene therapy or a placebo, and researchers will monitor ammonia levels and crisis events over time.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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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