Gene therapy breakthrough aims to control rare metabolic disease
NCT ID NCT06735755
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new gene therapy called BEAM-301 in 36 adults with glycogen storage disease type Ia (GSDIa) who have a specific gene change. The goal is to see if a single dose can safely improve blood sugar control and reduce the need for cornstarch supplements. Participants will be monitored for side effects and changes in their condition over time.
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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
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
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can home tests replace lab draws for rare disease patients?
- Can a simple Finger-Stick replace lab tests for rare metabolic disease?
- Gene therapy for rare Sugar-Storage disease shows promise in Long-Term safety check
- Gene therapy could free GSD ia patients from constant cornstarch
- GSDIa patients monitored for 10 years after gene therapy