One-Shot gene therapy trial offers hope for boys with devastating muscle disease
NCT ID NCT06138639
Summary
This study is testing a single intravenous dose of an experimental gene therapy called SGT-003 in boys with Duchenne muscular dystrophy (DMD). The main goals are to see if the treatment is safe and if it helps produce a needed muscle protein (microdystrophin) and improves muscle function. The trial will enroll 60 boys, from infants up to age 18, and follow them for five years.
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Contacts and locations
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Study contacts
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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, 60611-2605, United States
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's Hospital of the King's Daughters
RECRUITINGNorfolk, Virginia, 23510, United States
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
RECRUITINGRome, 00168, Italy
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Great Ormond Street Hospital
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43215, United States
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Oregon Health and Sciences University
RECRUITINGPortland, Oregon, 97239, United States
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 30329, United States
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105, United States
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The Hospital for Sick Children
RECRUITINGToronto, Ontario, M5G 0A4, Canada
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University of California
RECRUITINGSan Diego, California, 92037, United States
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University of California, Davis
RECRUITINGSacramento, California, 95817, United States
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University of California, Los Angeles Medical Center
RECRUITINGLos Angeles, California, 90095, United States
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Washington University in St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
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Conditions
Explore the condition pages connected to this study.