Gene therapy breakthrough for rare Sugar-Storage disease
NCT ID NCT06735755
First seen Jan 05, 2026 · Last updated May 22, 2026 · Updated 18 times
Summary
This study tests a new gene therapy called BEAM-301 in 36 adults with a specific form of glycogen storage disease type Ia. The goal is to see if it is safe and can help control blood sugar levels, reducing the need for frequent cornstarch doses. Participants must have a confirmed genetic variant and a history of low blood sugar episodes.
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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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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
Explore the condition pages connected to this study.