Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Purpose

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.

Condition

  • Glycogen Storage Disease Type Ia

Eligibility

Eligible Ages
Over 2 Years
Eligible Genders
All
Accepts 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

Study Design

Phase
Study Type
Observational
Observational Model
Cohort
Time Perspective
Prospective

Arm Groups

ArmDescriptionAssigned Intervention
Group 1: DTX401 in Prior Clinical Study Patients administered DTX401 (full or partial dose) in prior clinical study involving DTX401
  • Other: No Intervention
    No investigational/study product will be administered in this DMP.
Group 2: DTX401 in Post-Marketing Setting Patients administered prescribed DTX401(full or partial dose) in a post-marketing setting
  • Other: No Intervention
    No investigational/study product will be administered in this DMP.

Recruiting Locations

University of Texas Health Science Center at Houston
Houston, Texas 77030

More Details

Status
Recruiting
Sponsor
Ultragenyx Pharmaceutical Inc

Study Contact

Patients Contact: Trial Recruitment
1-888-756-8657
trialrecruitment@ultragenyx.com

Detailed 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.