Gene therapy patients monitored for a decade in groundbreaking safety study

NCT ID NCT06636383

Recruiting now Disease control Sponsor: Ultragenyx Pharmaceutical Inc Source: ClinicalTrials.gov ↗

Summary

This study will track 140 patients with Glycogen Storage Disease Type Ia for at least 10 years after they received an experimental gene therapy called DTX401. The main goal is to monitor long-term safety and see how well the treatment continues to work over time. Researchers will check for side effects, track disease symptoms, and measure how patients' daily lives are affected.

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

Contact

Phone: •••-•••-•••• Email: •••••@•••••
Contact

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Locations

Children's Hospital Colorado
RECRUITING

Denver, Colorado, 80045, United States
Children's Hospital of Orange County
RECRUITING

Orange, California, 92868, United States
Children's Hospital of Philadelphia
RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Duke University Medical Center
RECRUITING

Durham, North Carolina, 27710, United States
Fujita Health University Hospital
RECRUITING

Toyoake, 470-1192, Japan
Groningen University
RECRUITING

Groningen, Provincie Groningen, 9700 RB, Netherlands
Hospital Clínico Universitario de Santiago
RECRUITING

Santiago, 15706, Spain
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
NOT_YET_RECRUITING

Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
Istituto G Gaslini Ospedale Pediatrico IRCCS
RECRUITING

Genoa, 16147, Italy
McGill University Health Center
RECRUITING

Montreal, Quebec, H4A3J1, Canada
Osaka City General Hospital
RECRUITING

Osaka, 534-0021, Japan
Primary Children's Hospital
NOT_YET_RECRUITING

Salt Lake City, Utah, 84108, United States
Rigshospitalet
RECRUITING

Copenhagen, 2700, Denmark
The Cleveland Clinic Foundation
NOT_YET_RECRUITING

Cleveland, Ohio, 44195, United States
University of Connecticut Health Center
RECRUITING

Hartford, Connecticut, 06106, United States
University of Michigan
RECRUITING

Ann Arbor, Michigan, 48109, United States
University of Naples
RECRUITING

Naples, Campania, 80131, Italy
University of Texas Health Science Center at Houston
RECRUITING

Houston, Texas, 77030, United States
Universitätsklinikum Hamburg Eppendorf
RECRUITING

Hamburg, 20246, Germany

Conditions

Explore the condition pages connected to this study.

GLYCOGEN STORAGE DISEASE TYPE IA