RecruitingNCT06636383

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

GSDIa Disease Monitoring Program


Sponsor

Ultragenyx Pharmaceutical Inc

Enrollment

140 participants

Start Date

Nov 4, 2024

Study Type

OBSERVATIONAL

Conditions

Summary

The main objective of this observational study is to evaluate the long-term safety and effectiveness of DTX401 for at least 10 years after DTX401 administration.


Eligibility

Min Age: 2 Years

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying No Intervention for people with glycogen storage disease type ia. The study is currently recruiting participants at 19 locations. People eligible for this study include aged 2 Years and older.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

OTHERNo Intervention

No investigational/study product will be administered in this DMP.


Locations(19)

Children's Hospital of Orange County

Orange, California, United States

Children's Hospital Colorado

Denver, Colorado, United States

University of Connecticut Health Center

Hartford, Connecticut, United States

University of Michigan

Ann Arbor, Michigan, United States

Duke University Medical Center

Durham, North Carolina, United States

The Cleveland Clinic Foundation

Cleveland, Ohio, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

University of Texas Health Science Center at Houston

Houston, Texas, United States

Primary Children's Hospital

Salt Lake City, Utah, United States

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

Porto Alegre, Rio Grande do Sul, Brazil

McGill University Health Center

Montreal, Quebec, Canada

Rigshospitalet

Copenhagen, Denmark

Universitätsklinikum Hamburg Eppendorf

Hamburg, Germany

University of Naples

Naples, Campania, Italy

Istituto G Gaslini Ospedale Pediatrico IRCCS

Genoa, Italy

Osaka City General Hospital

Osaka, Japan

Fujita Health University Hospital

Toyoake, Japan

Groningen University

Groningen, Provincie Groningen, Netherlands

Hospital Clínico Universitario de Santiago

Santiago, Spain

View Full Details on ClinicalTrials.gov

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NCT06636383


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