One-Shot gene therapy trial offers hope for rare metabolic disease
NCT ID NCT06735755
Summary
This early-stage study is testing a single dose of an experimental gene therapy called BEAM-301 in adults with a specific genetic form of glycogen storage disease type Ia (GSDIa). The main goals are to see if the treatment is safe and to find the best dose. Researchers will measure if it helps patients better control their blood sugar and reduces their need for frequent starch supplements.
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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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Clinical Study Site
RECRUITINGOrange, California, 92868, United States
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Clinical Study Site
RECRUITINGFarmington, Connecticut, 06030, United States
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Clinical Study Site
RECRUITINGHouston, Texas, 77030, United States
Conditions
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