Actively Recruiting
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program for Long-Term Safety and Effectiveness of DTX401
Led by Ultragenyx Pharmaceutical Inc · Updated on 2026-05-29
140
Participants Needed
19
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are conducting a long-term observational study to track the safety and effects of DTX401 in people with Glycogen Storage Disease Type Ia (GSDIa). This study aims to follow participants for at least 10 years after they have received DTX401, a gene therapy treatment, to monitor its lasting impacts and any potential adverse events. The research is being led by Ultragenyx Pharmaceutical Inc and focuses on understanding the outcomes after treatment in both clinical trial and post-marketing settings. Participants in this study include those who received either a full or partial dose of DTX401 in previous clinical trials or as prescribed in real-world medical practice. There is no new administration of the study drug during this monitoring program. Instead, patients are observed over time to assess various health parameters and any related medical events. The study groups are divided based on whether the DTX401 dose was given in a clinical trial or post-marketing context. During the study, participants will undergo regular assessments including the tracking of serious adverse events, pregnancy outcomes, and potential infusion-related reactions. Researchers will also monitor changes in dietary intake, glucose levels, hypoglycemic events through continuous glucose monitoring, and other metabolic parameters. Quality of life, hospital visits, and work or school productivity are also evaluated. This follow-up lasts for a minimum of 10 years to gather comprehensive data on long-term safety and effectiveness.
CONDITIONS
Brief Title
Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patient who received DTX401 (full or partial dose) in a previous clinical study or as prescribed in a post-marketing setting
- Patient is willing and able to provide informed consent or assent, with legal representative consent if applicable
You will not qualify if you...
- Any condition that, in the investigator's opinion, would interfere with study participation, affect the interpretation of results, or compromise patient safety
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 10 years
Participants are followed for at least 10 years to monitor safety and effectiveness after receiving DTX401.
Regular visits over 10 years for assessments and monitoring
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
H
HCPs Contact: Medical Information
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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