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โ† Trials
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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Recruitingn/aAges 2 Years - anyAllUltragenyx Pharmaceutical Inc
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In plain words

What the trial is, and whether it could suit you

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.

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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
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What it tests

  • No Intervention
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Where

19 sites
Children's Hospital of Orange County
Orange, United States
Children's Hospital Colorado
Denver, United States
University of Connecticut Health Center
Hartford, United States
University of Michigan
Ann Arbor, United States
Duke University Medical Center
Durham, United States
The Cleveland Clinic Foundation
Cleveland, United States
Children's Hospital of Philadelphia
Philadelphia, United States
University of Texas Health Science Center at Houston
Houston, United States

โ€ฆand 11 more on ClinicalTrials.gov.

๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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