Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program
NCT ID: NCT06636383
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
140 participants
OBSERVATIONAL
2024-11-04
2036-12-31
Brief Summary
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Detailed Description
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Conditions
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Keywords
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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Group 1: DTX401 in Prior Clinical Study
Patients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401
No Intervention
No investigational/study product will be administered in this DMP.
Group 2: DTX401 in Post-Marketing Setting
Patients administered prescribed DTX401(pariglasgene brecaparvovec), full or partial dose, in a post-marketing setting
No Intervention
No investigational/study product will be administered in this DMP.
Interventions
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No Intervention
No investigational/study product will be administered in this DMP.
Eligibility Criteria
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Inclusion Criteria
* 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
2 Years
ALL
No
Sponsors
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Ultragenyx Pharmaceutical Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Ultragenyx Pharmaceuticals Inc.
Locations
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Children's Hospital of Orange County
Orange, California, United States
Children's Hospital Colorado
Denver, Colorado, United States
University of Connecticut Health Center
Hartford, Connecticut, United States
University of Michigan
Ann Arbor, Michigan, United States
Duke University Medical Center
Durham, North Carolina, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
University of Texas Health Science Center at Houston
Houston, Texas, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
Porto Alegre, Rio Grande do Sul, Brazil
McGill University Health Center
Montreal, Quebec, Canada
Rigshospitalet
Copenhagen, , Denmark
Universitätsklinikum Hamburg Eppendorf
Hamburg, , Germany
University of Naples
Napoli, Campania, Italy
Istituto G Gaslini Ospedale Pediatrico IRCCS
Genoa, , Italy
Osaka City General Hospital
Osaka, , Japan
Fujita Health University Hospital
Toyoake, , Japan
Groningen University
Groningen, , Netherlands
Hospital Clínico Universitario de Santiago
Santiago, , Spain
Countries
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Central Contacts
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Related Links
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Our Clinical Trial Transparency Commitment-Ultragenyx
Ultragenyx Patient Advocacy Website
Other Identifiers
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2023-510219-20-00
Identifier Type: CTIS
Identifier Source: secondary_id
DTX401-CL401
Identifier Type: -
Identifier Source: org_study_id