Scientists test One-Shot gene therapy to silence root cause of Muscle-Wasting disease
NCT ID NCT06907875
Summary
This early-stage study is testing a new, one-time gene therapy called EPI-321 in adults with FSHD, a genetic muscle-wasting disease. The main goal is to find out if the treatment is safe and what dose is best, while also looking for early signs that it might be working. About 12 participants will receive the therapy through an IV and be closely monitored for up to five years.
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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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David Geffen School of Medicine at University of California, Los Angeles
RECRUITINGLos Angeles, California, 90095, United States
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Pacific Clinical Research Network
RECRUITINGAuckland, 0622, New Zealand
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 303329, United States
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Royal Alfred Hospital
NOT_YET_RECRUITINGSydney, New South Wales, 2050, Australia
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of Massachusetts Chan Medical School
RECRUITINGWorcester, Massachusetts, 01605, United States
Contact Email: •••••@•••••
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Utah Program for Inherited Neuromuscular Disorders - University of Utah
RECRUITINGSalt Lake City, Utah, 84112, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
Conditions
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