One-Shot gene therapy aims to cure devastating baby disease
NCT ID NCT06255782
Summary
This study is testing a single-dose gene therapy called ECUR-506 in baby boys with a severe, life-threatening genetic disorder called OTC deficiency. The goal is to see if delivering a working copy of the faulty gene directly into the body is safe and can help the body properly process ammonia, preventing brain damage and death. The trial will test up to three different dose levels in a small group of infants to find the best and safest amount.
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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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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
Contact Email: •••••@•••••
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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
Contact Email: •••••@•••••
Conditions
Explore the condition pages connected to this study.