Gene therapy for rare metabolic disorder shows promise in Long-Term safety check
NCT ID NCT03636438
First seen Nov 17, 2025 · Last updated May 10, 2026 · Updated 23 times
Summary
This study checks the long-term safety of a one-time gene therapy (DTX301) in 11 adults with late-onset OTC deficiency, a rare genetic disorder that causes dangerous ammonia buildup. Participants had already completed one year of the main study and will be followed for several more years. The goal is to see if the treatment remains safe and helps control ammonia levels without needing lifelong medication.
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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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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Hopital Femme Mere Enfant
Bron, Rhone, 69677, France
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Hospital Clinico Universitario de Santiago
Santiago de Compostela, Coruna, 15706, Spain
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Hospital Universitario de Cruces. Servicio de Pediatria
Barakaldo, Vizcaya, 48903, Spain
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Icahn School of Medicine
New York, New York, 10029, United States
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M.A.G.I.C. Clinic
Calgary, Alberta, T2E 7Z4, Canada
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Queen Elizabeth Hospital, Department of Endocrinology
Birmingham, B15 2TH, United Kingdom
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The Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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University Hospital Cleveland Medical Center/Case Western Reserve University
Cleveland, Ohio, 44106, United States
Conditions
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