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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

NCT06636383

RecruitingN/A

Sponsor: Ultragenyx Pharmaceutical Inc

Conditions: Glycogen Storage Disease Type Ia

Interventions: No Intervention

Countries: United States, Brazil, Canada, Denmark, Germany, Italy, Japan, Netherlands

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 overview

Sex: ALL

Age: 2 Years to —

Healthy volunteers: No

Study type: OBSERVATIONAL

Eligibility criteria
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
Locations (19)
  • Orange, California, United States
  • Denver, Colorado, United States
  • Hartford, Connecticut, United States
  • Ann Arbor, Michigan, United States
  • Durham, North Carolina, United States
  • Cleveland, Ohio, United States
  • Philadelphia, Pennsylvania, United States
  • Houston, Texas, United States
  • Salt Lake City, Utah, United States
  • Porto Alegre, Rio Grande do Sul, Brazil
  • Montreal, Quebec, Canada
  • Copenhagen, Denmark
  • Hamburg, Germany
  • Naples, Campania, Italy
  • Genoa, Italy
  • Osaka, Japan
  • Toyoake, Japan
  • Groningen, Provincie Groningen, Netherlands
  • Santiago, Spain