Spinal gene injection aims to slow duchenne in toddlers
NCT ID NCT06817382
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-stage study tests a single injection of a gene therapy called INS1201, given into the spinal fluid of young boys (ages 2 to 5) with Duchenne muscular dystrophy who can still walk. The main goal is to check if the treatment is safe and to see how it spreads in the body. Researchers will also measure if the therapy helps produce a shortened version of the missing muscle protein.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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
Contact Email: •••••@•••••
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USA006
RECRUITINGColumbus, Ohio, 43205, United States
Contact Email: •••••@•••••
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USA008
RECRUITINGRochester, New York, 14642, United States
Contact Email: •••••@•••••
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USA009
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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USA010
RECRUITINGDavis, California, 95616, United States
Contact Email: •••••@•••••
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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: •••••@•••••