Recruiting

DTX401Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

Sponsor:

Ultragenyx Pharmaceutical Inc

Code:

NCT06636383

Conditions

Glycogen Storage Disease Type Ia

Eligibility Criteria

Sex: All

Age: 2+

Healthy Volunteers: Not accepted

Interventions

No Intervention

Study Details

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.

Conditions

Glycogen Storage Disease Type Ia

Study ID

NCT06636383

Start date

Nov 4, 2024

Status verified date

Aug, 2026

Completion date

Dec, 2036

Anticipated

Primary completion date

Dec, 2036

Anticipated

Eligibility Criteria

Sex: All

Age: 2+

Healthy Volunteers: Not accepted

Inclusion Criteria:

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.

Exclusion Criteria:

Presence of any condition that would interfere with study participation, interpretation of results or affect patient's safety in the opinion of the Investigator

Study Design

Enrollment

140 participants

Anticipated

Interventions and Outcome Measures

Arms

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 Intervention

No investigational/study product will be administered in this DMP.

Primary outcome measure

Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator [ Time Frame: 10 Years ]
Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies [ Time Frame: 10 Years ]
Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner [ Time Frame: 10 Years ]
Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner [ Time Frame: 10 Years ]
Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only) [ Time Frame: 1 Year ]
Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only) [ Time Frame: 1 Year ]

Central Contacts and Locations

Central contacts

Patients Contact: Trial Recruitment

1-888-756-8657 trialrecruitment@ultragenyx.com

HCPs Contact: Medical Information

1-888-756-8657 medinfo@ultragenyx.com

Locations

Children's Hospital of Orange County

Recruiting

Orange, California, United States, 92868

Children's Hospital Colorado

Recruiting

Denver, Colorado, United States, 80045

University of Connecticut Health Center

Recruiting

Hartford, Connecticut, United States, 06106

University of Michigan

Recruiting

Ann Arbor, Michigan, United States, 48109

Duke University Medical Center

Recruiting

Durham, North Carolina, United States, 27710

Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

University of Texas Health Science Center at Houston

Recruiting

Houston, Texas, United States, 77030

McGill University Health Center

Recruiting

Montreal, Quebec, Canada, H4A3J1

More Information

Sponsor

Ultragenyx Pharmaceutical Inc

Last update posted

Aug 26, 2026

Last verified

Aug, 2026

Keywords

Glycogen storage disorder Ia
AAV
Gene therapy
Von Gierke disease
Glucose metabolism disorder
GSD1
GSDIa

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-09. This information was provided to ClinicalTrials.gov by Ultragenyx Pharmaceutical Inc on 2026-08-26.