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Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

NCT06636383 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 140

Last updated 2026-04-23

No results posted yet for this study

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.

Conditions

  • Glycogen Storage Disease Type Ia

Interventions

OTHER

No Intervention

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

Sponsors & Collaborators

Principal Investigators

  • Medical Director · Ultragenyx Pharmaceuticals Inc.

Eligibility

Min Age
2 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-11-04
Primary Completion
2036-12-31
Completion
2036-12-31
FDA Drug
Yes

Countries

  • United States
  • Brazil
  • Canada
  • Denmark
  • Germany
  • Italy
  • Japan
  • Netherlands
  • Spain

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06636383 on ClinicalTrials.gov