Gene therapy may free GSDIa patients from constant cornstarch
NCT ID NCT05139316
First seen Nov 19, 2025 · Last updated May 15, 2026 · Updated 19 times
Summary
This phase 3 study tested a gene therapy (DTX401) in 49 people with glycogen storage disease type Ia, a rare condition that requires frequent cornstarch doses to prevent dangerously low blood sugar. The goal was to see if the treatment could reduce or eliminate the need for cornstarch while keeping blood sugar stable. Participants received either the gene therapy or a placebo, and researchers measured changes in daily cornstarch use over 48 weeks.
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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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Locations
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Orange County
Orange, California, 92868, United States
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Cleveland Clinic
Cleveland, Ohio, 44195, United States
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Duke University
Durham, North Carolina, 27710, United States
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Fujita Health University Hospital
Toyoake, Japan
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Groningen University
Groningen, 9700 RB, Netherlands
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Hospital Clinico Universitario de Santiago
Santiago de Compostela, A Coruna, 15706, Spain
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Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
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Istituto Giannina Gaslini
Genova, Linguria, 16147, Italy
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Kumamoto University Hospital
Kumamoto, Japan
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McGill University
Montreal, Quebec, H3H 1P3, Canada
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Mount Sinai
The Bronx, New York, 10467, United States
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Osaka City General Hospital
Osaka, Japan
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Primary Children's Hospital
Salt Lake City, Utah, 84132, United States
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Righospitalet
Copenhagen, Capital, 2100, Denmark
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University Medical Center Eppendorf
Hamburg, 20251, Germany
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University of Connecticut Health Center
Farmington, Connecticut, 06030, United States
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University of Naples
Naples, 80131, Italy
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University of Texas
Houston, Texas, 77030, United States
Conditions
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