Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Recruiting now

Conditions studied: Glycogen Storage Disease Type Ia

In brief

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.

Key facts

Study ID

NCT06636383

Run by

Ultragenyx Pharmaceutical Inc

People needed

140

Starts

2024-11-04

Expected to finish

2036-12-01

Last updated by the study team

2026-07-06

Who can join

Age: 2 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator

Where it is running

• Hospital Clínico Universitario de Santiago — Santiago, Spain (enrolling)
• Children's Hospital Colorado — Denver, Colorado, United States (enrolling)
• University of Connecticut Health Center — Hartford, Connecticut, United States (enrolling)
• University of Michigan — Ann Arbor, Michigan, United States (enrolling)
• Duke University Medical Center — Durham, North Carolina, United States (enrolling)
• Groningen University — Groningen, Provincie Groningen, Netherlands (enrolling)
• Children's Hospital of Orange County — Orange, California, United States (enrolling)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (enrolling)
• University of Texas Health Science Center at Houston — Houston, Texas, United States (enrolling)
• Hospital de Clinicas de Porto Alegre (HCPA) - PPDS — Porto Alegre, Rio Grande do Sul, Brazil (enrolling)
• McGill University Health Center — Montreal, Quebec, Canada (enrolling)
• Rigshospitalet — Copenhagen, Denmark (enrolling)
• Universitätsklinikum Hamburg Eppendorf — Hamburg, Germany (enrolling)
• University of Naples — Naples, Campania, Italy (enrolling)
• Istituto G Gaslini Ospedale Pediatrico IRCCS — Genoa, Italy (enrolling)
• Osaka City General Hospital — Osaka, Japan (enrolling)
• Fujita Health University Hospital — Toyoake, Japan (enrolling)
• The Cleveland Clinic Foundation — Cleveland, Ohio, United States
• Primary Children's Hospital — Salt Lake City, Utah, United States

Full record on ClinicalTrials.gov

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