Gene therapy could free GSD ia patients from constant cornstarch
NCT ID NCT05139316
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This Phase 3 trial tested a gene therapy called DTX401 in 49 people with glycogen storage disease type Ia (GSD Ia). The goal was to see if a single infusion could reduce or eliminate the need for frequent cornstarch doses to keep blood sugar stable. Participants were randomly assigned to receive either the gene therapy or a placebo, and their cornstarch intake was measured over 48 weeks.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
DTX401 (pariglasgene brecaparvovec) gene therapy
What this could lead to
If successful, this gene therapy could reduce or eliminate the need for frequent cornstarch doses to maintain normal blood sugar levels in people with GSD Ia.
What could go wrong
This is an early-stage Phase 3 trial with only 49 participants, so results may not apply to everyone. Gene therapy carries risks like immune reactions or liver issues, and long-term effects are unknown.
Disclaimer
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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.
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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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