Actively Recruiting
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Led by Ultragenyx Pharmaceutical Inc · Updated on 2026-04-23
140
Participants Needed
19
Research Sites
630 weeks
Total Duration
On this page
AI-Summary
What this Trial Is 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.
CONDITIONS
Official Title
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Who Can Participate
Eligibility Criteria
You may qualify if you...
- 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 study has been explained
- If a minor or adult with cognitive limitations, patient is willing and able to provide assent and have a legally authorized representative provide informed consent after the study has been explained
You will not qualify if you...
- Presence of any condition that would interfere with study participation, interpretation of results, or affect patient's safety in the opinion of the Investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 19 locations
1
Children's Hospital of Orange County
Orange, California, United States, 92868
Actively Recruiting
2
Children's Hospital Colorado
Denver, Colorado, United States, 80045
Actively Recruiting
3
University of Connecticut Health Center
Hartford, Connecticut, United States, 06106
Actively Recruiting
4
University of Michigan
Ann Arbor, Michigan, United States, 48109
Actively Recruiting
5
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
6
The Cleveland Clinic Foundation
Cleveland, Ohio, United States, 44195
Not Yet Recruiting
7
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
8
University of Texas Health Science Center at Houston
Houston, Texas, United States, 77030
Actively Recruiting
9
Primary Children's Hospital
Salt Lake City, Utah, United States, 84108
Not Yet Recruiting
10
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
Actively Recruiting
11
McGill University Health Center
Montreal, Quebec, Canada, H4A3J1
Actively Recruiting
12
Rigshospitalet
Copenhagen, Denmark, 2700
Actively Recruiting
13
Universitätsklinikum Hamburg Eppendorf
Hamburg, Germany, 20246
Actively Recruiting
14
University of Naples
Naples, Campania, Italy, 80131
Actively Recruiting
15
Istituto G Gaslini Ospedale Pediatrico IRCCS
Genoa, Italy, 16147
Actively Recruiting
16
Osaka City General Hospital
Osaka, Japan, 534-0021
Actively Recruiting
17
Fujita Health University Hospital
Toyoake, Japan, 470-1192
Actively Recruiting
18
Groningen University
Groningen, Netherlands, 9700 RB
Actively Recruiting
19
Hospital Clínico Universitario de Santiago
Santiago, Spain, 15706
Actively Recruiting
Research Team
P
Patients Contact: Trial Recruitment
CONTACT
H
HCPs Contact: Medical Information
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
2
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