Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Ultragenyx

Status

Enrolling

Conditions

Glycogen Storage Disease Type Ia

Treatments

Other: No Intervention

Study type

Observational

Funder types

Industry

Identifiers

NCT06636383

2023-510219-20-00 (EU Trial (CTIS) Number)

DTX401-CL401

Details and patient eligibility

About

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.

Full description

The DTX401-CL401 Disease Monitoring Program (DMP) is a prospective, multicenter, long-term observational study to follow up participants with GSDIa for at least 10 years after the administration of DTX401.

Enrollment

140 estimated patients

Sex

All

Ages

2+ years old

Volunteers

No 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