One-Shot gene therapy aims to fix deadly baby disease
NCT ID NCT06255782
Summary
This study is testing a single-dose gene therapy called ECUR-506 for male infants under 9 months old with a severe, life-threatening form of OTC deficiency. The goal is to see if delivering a working copy of the faulty gene into the liver is safe and can help the body control toxic ammonia levels. If successful, this could reduce or eliminate the need for lifelong medications and strict diets.
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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
Contact Email: •••••@•••••
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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.