Gene therapy trial offers hope for babies with rare, deadly metabolic disease
NCT ID NCT06255782
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a one-time gene therapy called ECUR-506 in baby boys under 9 months old with a severe form of OTC deficiency, a genetic disorder that prevents the body from breaking down ammonia. The goal is to see if the treatment is safe and can reduce dangerous ammonia levels, potentially allowing babies to stop taking daily medications. The trial involves up to 20 participants and will monitor for side effects and effectiveness 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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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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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: •••••@•••••
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