Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
GSDIa Disease Monitoring Program
2 other identifiers
observational
140
9 countries
19
Brief 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for all trials
Started Nov 2024
Longer than P75 for all trials
19 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
September 20, 2024
CompletedFirst Posted
Study publicly available on registry
October 10, 2024
CompletedStudy Start
First participant enrolled
November 4, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
December 1, 2036
ExpectedStudy Completion
Last participant's last visit for all outcomes
December 1, 2036
April 23, 2026
April 1, 2026
12.1 years
September 20, 2024
April 21, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (6)
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
Secondary Outcomes (22)
Percent Change Over Time from Baseline in Total Cornstarch Intake
Baseline, Up to 10 Years
Change from Baseline Over Time in Frequency of Cornstarch Intake
Baseline, Up to 10 Years
Change from Baseline Over Time in Number of Nighttime Awakenings for Cornstarch
Baseline, Up to 10 Years
Change from Baseline Over Time in Dependance on Continuous Overnight Tube Feeding for Exogenous Glucose Delivery
Baseline, Up to 10 Years
Change from Baseline Over Time in Daily Dietary Intake of Foods, Beverages, and Medical Foods/Formulas (Non-Cornstarch)
Baseline, Up to 10 Years
- +17 more secondary outcomes
Study Arms (2)
Group 1: DTX401 in Prior Clinical Study
Patients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401
Group 2: DTX401 in Post-Marketing Setting
Patients administered prescribed DTX401(pariglasgene brecaparvovec), full or partial dose, in a post-marketing setting
Interventions
No investigational/study product will be administered in this DMP.
Eligibility Criteria
All participants who received DTX401 in a qualifying clinical study will be expected and asked to participate in the DMP upon completion or early termination (ET) from the parent study, as required by regulatory agencies for all gene therapy studies.
You may qualify if:
- 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.
You may not qualify if:
- Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (19)
Children's Hospital of Orange County
Orange, California, 92868, United States
Children's Hospital Colorado
Denver, Colorado, 80045, United States
University of Connecticut Health Center
Hartford, Connecticut, 06106, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
University of Texas Health Science Center at Houston
Houston, Texas, 77030, United States
Primary Children's Hospital
Salt Lake City, Utah, 84108, United States
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, 90035-903, Brazil
McGill University Health Center
Montreal, Quebec, H4A3J1, Canada
Rigshospitalet
Copenhagen, 2700, Denmark
Universitätsklinikum Hamburg Eppendorf
Hamburg, 20246, Germany
University of Naples
Naples, Campania, 80131, Italy
Istituto G Gaslini Ospedale Pediatrico IRCCS
Genoa, 16147, Italy
Osaka City General Hospital
Osaka, 534-0021, Japan
Fujita Health University Hospital
Toyoake, 470-1192, Japan
Groningen University
Groningen, 9700 RB, Netherlands
Hospital Clínico Universitario de Santiago
Santiago, 15706, Spain
Related Links
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director
Ultragenyx Pharmaceuticals Inc.
Central Study Contacts
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 20, 2024
First Posted
October 10, 2024
Study Start
November 4, 2024
Primary Completion (Estimated)
December 1, 2036
Study Completion (Estimated)
December 1, 2036
Last Updated
April 23, 2026
Record last verified: 2026-04