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Clinical Trials in the USA / NCT06636383
Recruiting Observational

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

NCT06636383 · tracked via the Priya Life Science USA tracker
Sponsor
Ultragenyx Pharmaceutical Inc
Phase
Observational
Started
2024-11-04
Last updated
2026-10-14

Condition(s) studied

Glycogen Storage Disease Type Ia

Investigational drug(s) / intervention(s)

No Intervention

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

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.

Eligibility

Sex
ALL
Min age
2 Years
Max age
—
Healthy volunteers
No
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

Primary outcome measure(s)

  • Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator — 10 Years
  • Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies — 10 Years
  • Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner — 10 Years
  • Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner — 10 Years
  • Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only) — 1 Year
  • Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only) — 1 Year

Trial sites (19)

FacilityCityRegionStatus
Children's Hospital of Orange County Orange California Recruiting
Children's Hospital Colorado Denver Colorado Recruiting
University of Connecticut Health Center Hartford Connecticut Recruiting
University of Michigan Ann Arbor Michigan Recruiting
Duke University Medical Center Durham North Carolina Recruiting
The Cleveland Clinic Foundation Cleveland Ohio Not Yet Recruiting
Children's Hospital of Philadelphia Philadelphia Pennsylvania Recruiting
University of Texas Health Science Center at Houston Houston Texas Recruiting
Primary Children's Hospital Salt Lake City Utah Not Yet Recruiting
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS Porto Alegre Rio Grande do Sul Recruiting
McGill University Health Center Montreal Quebec Recruiting
Rigshospitalet Copenhagen Denmark Recruiting
Universitätsklinikum Hamburg Eppendorf Hamburg Germany Recruiting
University of Naples Naples Campania Recruiting
Istituto G Gaslini Ospedale Pediatrico IRCCS Genoa Italy Recruiting
Osaka City General Hospital Osaka Japan Recruiting
Fujita Health University Hospital Toyoake Japan Recruiting
Groningen University Groningen Provincie Groningen Recruiting
Hospital Clínico Universitario de Santiago Santiago Spain Recruiting

More Ultragenyx Pharmaceutical Inc trials in the USA

Mucopolysaccharidosis VII Disease Monitoring Program Recruiting
X-linked Hypophosphatemia Disease Monitoring Program Active, not recruiting
Long-Chain Fatty Acid Oxidation Disorders In-Clinic Disease Monitoring Program Active, not recruiting
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta Active, not recruiting
Long Term Follow Up to Evaluate DTX301 in Adults With Late-Onset OTC Deficiency Active, not recruiting

Other trials for the same condition

A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06636383 on ClinicalTrials.gov ↗ ← All trials in the USA