Gene therapy patients tracked for a decade in groundbreaking safety study
NCT ID NCT06636383
First seen Nov 01, 2025 · Last updated Apr 15, 2026 · Updated 34 times
Summary
This study will follow 140 patients with Glycogen Storage Disease Type Ia for at least 10 years after they received DTX401 gene therapy. The main goal is to monitor long-term safety and effectiveness, including whether patients can reduce their dependence on cornstarch treatments and improve blood sugar control. Researchers will track health outcomes, side effects, and quality of life measures over the decade-long observation period.
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This is a summary of
the original study
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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: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Children's Hospital Colorado
RECRUITINGDenver, Colorado, 80045, United States
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Children's Hospital of Orange County
RECRUITINGOrange, California, 92868, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Duke University Medical Center
RECRUITINGDurham, North Carolina, 27710, United States
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Fujita Health University Hospital
RECRUITINGToyoake, 470-1192, Japan
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Groningen University
RECRUITINGGroningen, Provincie Groningen, 9700 RB, Netherlands
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Hospital Clínico Universitario de Santiago
RECRUITINGSantiago, 15706, Spain
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Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
NOT_YET_RECRUITINGPorto Alegre, Rio Grande do Sul, 90035-903, Brazil
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Istituto G Gaslini Ospedale Pediatrico IRCCS
RECRUITINGGenoa, 16147, Italy
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McGill University Health Center
RECRUITINGMontreal, Quebec, H4A3J1, Canada
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Osaka City General Hospital
RECRUITINGOsaka, 534-0021, Japan
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Primary Children's Hospital
NOT_YET_RECRUITINGSalt Lake City, Utah, 84108, United States
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Rigshospitalet
RECRUITINGCopenhagen, 2700, Denmark
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The Cleveland Clinic Foundation
NOT_YET_RECRUITINGCleveland, Ohio, 44195, United States
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University of Connecticut Health Center
RECRUITINGHartford, Connecticut, 06106, United States
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University of Michigan
RECRUITINGAnn Arbor, Michigan, 48109, United States
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University of Naples
RECRUITINGNaples, Campania, 80131, Italy
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University of Texas Health Science Center at Houston
RECRUITINGHouston, Texas, 77030, United States
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Universitätsklinikum Hamburg Eppendorf
RECRUITINGHamburg, 20246, Germany
Conditions
Explore the condition pages connected to this study.