One-Shot gene therapy aims to cure devastating baby disease
NCT ID NCT06255782
Summary
This study is testing a first-of-its-kind, one-time gene editing treatment for male babies with a severe, life-threatening metabolic disorder called OTC deficiency. The treatment, called ECUR-506, is given through an IV and aims to deliver a working copy of the faulty gene to the liver. The main goals are to see if the treatment is safe and if it can help the body control toxic ammonia levels, potentially eliminating the need for lifelong medications.
Contacts and locations
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Study contacts
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Contact
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Contact
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611, United States
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Children's Hospital of Colorado, Anshutz Medical Campus
RECRUITINGAurora, Colorado, 80045, United States
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Great Ormond Street Hospital
RECRUITINGLondon, United Kingdom
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Hopsital Sant Joan de Deu
RECRUITINGBarcelona, 08950, Spain
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Hospital Universitario 12 de Octubre
RECRUITINGMadrid, 28041, Spain
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Icahn School of Medicine at Mount Sinai
RECRUITINGNew York, New York, 10029, United States
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The Children's Hospital at Westmead
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
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The Royal Children's Hospital
RECRUITINGMelbourne, Victoria, 3052, Australia
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UCLA Mattel Children's Hospital
RECRUITINGLos Angeles, California, 90095, United States
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