Gene therapy aims to tame rare metabolic disorder
NCT ID NCT05345171
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This Phase 3 trial tests a gene therapy called DTX301 for people with late-onset OTC deficiency, a rare genetic disorder that causes dangerous ammonia buildup. The study involves 37 participants and compares the gene therapy to a placebo. The goal is to see if the treatment can help control ammonia levels and reduce the number of metabolic crises.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
DTX301 (avalotcagene ontaparvovec) gene therapy
What this could lead to
If successful, this gene therapy could help people with OTC deficiency maintain safe ammonia levels and reduce the risk of dangerous metabolic crises.
What could go wrong
This is an early-stage Phase 3 trial with only 37 participants. The therapy may not work for everyone, and side effects from the gene vector or required steroids are possible.
Disclaimer
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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