GSDIa patients monitored for 10 years after gene therapy
NCT ID NCT06636383
First seen Jun 24, 2026 · Last updated Jul 07, 2026 · Updated 3 times
Summary
This observational study follows people with Glycogen Storage Disease Type Ia who have already received the gene therapy DTX401. The goal is to track their health and safety for at least 10 years after treatment. No new drugs or treatments are given in this study. It will help researchers understand the long-term effects of DTX401.
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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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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
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
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Other studies related to the condition(s) this trial covers.
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