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

2026-08-26

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

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)

Facility	City	Region	Status
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

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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