One-Time gene therapy could free babies from lifelong medication for rare liver disease
NCT ID NCT06255782
First seen Nov 01, 2025 · Last updated May 06, 2026 · Updated 25 times
Summary
This study tests a single-dose gene therapy called ECUR-506 in baby boys under 9 months old with a severe form of OTC deficiency, a genetic liver disorder that causes dangerous ammonia buildup. The goal is to see if the treatment is safe and can reduce or eliminate the need for daily medications and a strict low-protein diet. About 20 babies will receive the therapy intravenously and be monitored for side effects and ammonia control.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
Contact Email: •••••@•••••
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Children's Hospital of Colorado, Anshutz Medical Campus
RECRUITINGAurora, Colorado, 80045, United States
Contact Email: •••••@•••••
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Emory University School of Medicine
RECRUITINGAtlanta, Georgia, 30322, United States
Contact Email: •••••@•••••
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Great Ormond Street Hospital
RECRUITINGLondon, United Kingdom
Contact Email: •••••@•••••
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Hopsital Sant Joan de Deu
RECRUITINGBarcelona, 08950, Spain
Contact Email: •••••@•••••
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Hospital Universitario 12 de Octubre
RECRUITINGMadrid, 28041, Spain
Contact Email: •••••@•••••
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Icahn School of Medicine at Mount Sinai
RECRUITINGNew York, New York, 10029, United States
Contact Email: •••••@•••••
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Oregon Health and Science University
RECRUITINGPortland, Oregon, 97239, United States
Contact Email: •••••@•••••
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The Children's Hospital at Westmead
ACTIVE_NOT_RECRUITINGSydney, New South Wales, Australia
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The Newcastle upon Tyne Hospitals NHS Foundation Trust- Great North Children's Hospital
RECRUITINGNewcastle upon Tyne, United Kingdom
Contact Email: •••••@•••••
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The Royal Children's Hospital
ACTIVE_NOT_RECRUITINGMelbourne, Victoria, 3052, Australia
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UCLA Mattel Children's Hospital
RECRUITINGLos Angeles, California, 90095, United States
Contact Email: •••••@•••••
Conditions
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