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NCT06636383Recruiting

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Ultragenyx Pharmaceutical Inc

Start Date

11/4/2024

Completion Date

12/1/2036

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.

Detailed Description

The DTX401-CL401 Disease Monitoring Program (DMP) is a prospective, multicenter, long-term observational study to follow up participants with GSDIa for at least 10 years after the administration of DTX401.

Eligibility Criteria

Age Range: 2 years to No maximum

Inclusion Criteria: * Patient who had: * DTX401 (full or partial dose) administered in a parent clinical study (Group 1) or * Prescribed DTX401(full or partial dose) administered in a post-marketing setting (Group 2) * Patient is willing and able to provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. If a minor or an adult with cognitive limitations, the patient is willing and able (if possible) to provide assent and have a legally authorized representative provide informed consent after the nature of the study has been explained, and prior to any research-related assessments or procedures. Exclusion Criteria: * Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator

Interventions

OTHER

No Intervention

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Conditions

Glycogen Storage Disease Type Ia

Locations

Children's Hospital of Orange County

Orange, California 92868

United States

Children's Hospital Colorado

Denver, Colorado 80045

United States

University of Connecticut Health Center

Hartford, Connecticut 06106

United States

University of Michigan

Ann Arbor, Michigan 48109

United States

Duke University Medical Center

Durham, North Carolina 27710

United States

The Cleveland Clinic Foundation

Cleveland, Ohio 44195

United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania 19104

United States

University of Texas Health Science Center at Houston

Houston, Texas 77030

United States

Primary Children's Hospital

Salt Lake City, Utah 84108

United States

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS

Porto Alegre, Rio Grande do Sul 90035-903

Brazil

McGill University Health Center

Montreal, Quebec H4A3J1

Canada

Rigshospitalet

Copenhagen, 2700

Denmark

Universitätsklinikum Hamburg Eppendorf

Hamburg, 20246

Germany

University of Naples

Naples, Campania 80131

Italy

Istituto G Gaslini Ospedale Pediatrico IRCCS

Genoa, 16147

Italy

Osaka City General Hospital

Osaka, 534-0021

Japan

Fujita Health University Hospital

Toyoake, 470-1192

Japan

Groningen University

Groningen, Provincie Groningen 9700 RB

Netherlands

Hospital Clínico Universitario de Santiago

Santiago, 15706

Spain