Glycogen Storage Disease Type IA
Glycogen Storage Disease Type IA (GSDIa) is a rare genetic disorder that affects the body's ability to break down glycogen into glucose, leading to low blood sugar levels and various metabolic issues.
GSDIa: DTX401 Treatment Study
We are examining the long-term safety and pregnancy outcomes of DTX401 for individuals with GSDIa. This study will help us understand how effective this treatment is over time.
>2 yearsConfirmation phase (III)Monitoring phase (IV)No PlaceboPartially RemoteInvestigational MedicinesInjectionEndocrinologyInternal Medicine
What diseases are being studied?
Health conditions and diseases that the clinical trial is designed to study and treat.
Who can participate in the clinical trial?
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
- Have you received DTX401 (either full or partial dose) in a previous clinical study or post-marketing setting?
- Are you able to understand the study and provide consent for participation?
- Do you have any medical conditions that might affect your safety or ability to participate in this study?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Pariglasgene Brecaparvovec
Where is the clinical trial being conducted?
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
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Rigshospitalet
Department of Paediatrics and Clinical Genetics
Copenhagen, Denmark
University Medical Center Hamburg-Eppendorf
pediatric and adolescent medicine
Hamburg, Germany
IRCCS Istituto Giannina Gaslini
Pediatric gastroenterology and digestive endoscopy Unit
Genoa, Italy
Sponsor: Ultragenyx Pharmaceutical Inc.
Last updated: Nov 27, 2025
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.