First spinal gene therapy tested in toddlers with muscular dystrophy
NCT ID NCT06817382
Summary
This is an early safety study testing a single spinal injection of an experimental gene therapy called INS1201. It involves up to 12 young boys, aged 2 to under 5 years old, who have Duchenne muscular dystrophy and can still walk. The main goal is to see if the treatment is safe and to learn how it moves through the body, while also checking early signs of whether it helps produce a needed muscle protein.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Rare Disease Research (USA004)
RECRUITINGAtlanta, Georgia, 30329, United States
Contact Email: •••••@•••••
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USA001
RECRUITINGMemphis, Tennessee, 38105, United States
Contact Email: •••••@•••••
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USA002
RECRUITINGPalo Alto, California, 94070, United States
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USA005
RECRUITINGSan Diego, California, 93123, United States
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USA006
RECRUITINGColumbus, Ohio, 43205, United States
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USA008
RECRUITINGRochester, New York, 14642, United States
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USA010
RECRUITINGDavis, California, 95616, United States
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USA012
RECRUITINGLittle Rock, Arkansas, 72202, United States
Contact Email: •••••@•••••
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USA015
RECRUITINGNorfolk, Virginia, 23507, United States
Contact Email: •••••@•••••
Conditions
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