Gene therapy for rare Sugar-Storage disease shows promise in Long-Term safety check
NCT ID NCT03970278
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study checked the long-term safety of a one-time gene therapy (DTX401) in 12 adults with glycogen storage disease type Ia, a rare condition that causes dangerously low blood sugar. Researchers monitored side effects and how well the therapy helped control blood sugar levels over time. The goal was to see if the treatment remains safe and effective in the long run.
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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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Locations
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Complejo Hospitalario Universitario de Santiago
Santiago de Compostela, A Coruna, 15706, Spain
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Michigan Medicine University of Michigan
Ann Arbor, Michigan, 48109, United States
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Montreal Children Hospital, McGill University Health Centre
Montreal, Quebec, H4A3J1, Canada
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UCONN Health
Farmington, Connecticut, 06030-3213, United States
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University Medical Center Groningen
Groningen, 9700RB, Netherlands
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University of Texas Health Science Center at Houston
Houston, Texas, 77030, United States