Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

August 25, 2026 updated by: Ultragenyx Pharmaceutical Inc

GSDIa Disease Monitoring Program

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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

The DTX401-CL401 Disease Monitoring Program (DMP) is a prospective, multicenter, long-term observational study to follow up participants with GSDIa for at least 10 years after the administration of DTX401.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
140

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Contact Backup

Study Locations

  • Brazil
    • Rio Grande do Sul
      • Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
        • Recruiting
        • Hospital de Clinicas de Porto Alegre (HCPA) - PPDS
  • Canada
    • Quebec
      • Montreal, Quebec, Canada, H4A3J1
        • Recruiting
        • McGill University Health Center
  • Denmark
      • Copenhagen, Denmark, 2700
        • Recruiting
        • Rigshospitalet
  • Germany
      • Hamburg, Germany, 20246
        • Recruiting
        • Universitatsklinikum Hamburg Eppendorf
  • Italy
      • Genoa, Italy, 16147
        • Recruiting
        • Istituto G Gaslini Ospedale Pediatrico IRCCS
    • Campania
      • Naples, Campania, Italy, 80131
        • Recruiting
        • University of Naples
  • Japan
      • Osaka, Japan, 534-0021
        • Recruiting
        • Osaka City General Hospital
      • Toyoake, Japan, 470-1192
        • Recruiting
        • Fujita Health University Hospital
  • Netherlands
    • Provincie Groningen
      • Groningen, Provincie Groningen, Netherlands, 9700 RB
        • Recruiting
        • Groningen University
  • Spain
      • Santiago, Spain, 15706
        • Recruiting
        • Hospital Clínico Universitario de Santiago
  • United States
    • California
      • Orange, California, United States, 92868
        • Recruiting
        • Children's Hospital of Orange County
    • Colorado
      • Denver, Colorado, United States, 80045
        • Recruiting
        • Children's Hospital Colorado
    • Connecticut
      • Hartford, Connecticut, United States, 06106
        • Recruiting
        • University of Connecticut Health Center
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Medical Center
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Not yet recruiting
        • The Cleveland Clinic Foundation
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • University of Texas Health Science Center at Houston
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • Not yet recruiting
        • Primary Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

All participants who received DTX401 in a qualifying clinical study will be expected and asked to participate in the DMP upon completion or early termination (ET) from the parent study, as required by regulatory agencies for all gene therapy studies.

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Group 1: DTX401 in Prior Clinical Study
Patients administered DTX401(pariglasgene brecaparvovec), full or partial dose, in prior clinical study involving DTX401
Other: 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
Other: No Intervention
No investigational/study product will be administered in this DMP.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator
Time Frame: 10 Years
10 Years
Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies
Time Frame: 10 Years
10 Years
Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner
Time Frame: 10 Years
10 Years
Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner
Time Frame: 10 Years
10 Years
Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only)
Time Frame: 1 Year
1 Year
Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only)
Time Frame: 1 Year
1 Year

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percent Change Over Time from Baseline in Total Cornstarch Intake
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years
Change from Baseline Over Time in Frequency of Cornstarch Intake
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years
Change from Baseline Over Time in Number of Nighttime Awakenings for Cornstarch
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years
Change from Baseline Over Time in Dependance on Continuous Overnight Tube Feeding for Exogenous Glucose Delivery
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years
Change from Baseline Over Time in Daily Dietary Intake of Foods, Beverages, and Medical Foods/Formulas (Non-Cornstarch)
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years
Change from Baseline Over Time in Daytime and Nighttime Percentage of Glucose Values During the 2 Weeks Leading Up to the Assessment Visits
Time Frame: Baseline, Up to 10 Years
The following ranges will be assessed: < 54 mg/dL, < 60 mg/dL, < 70 mg/dL, 70-120 mg/dL, > 120 mg/dL
Baseline, Up to 10 Years
Change from Baseline Over Time in Total Number of Hypoglycemic Events on Continuous Glucose Monitoring (CGM) During the 2 Weeks Leading Up to the Assessment Visits
Time Frame: Baseline, Up to 10 Years
The following ranges will be assessed: < 54 mg/dL, < 70 mg/dL
Baseline, Up to 10 Years
Change from Baseline Over Time in Total Duration of Hypoglycemic Events on CGM During the 2 Weeks Leading Up to the Assessment Visits
Time Frame: Baseline, Up to 10 Years
The following ranges will be assessed: < 54 mg/dL, < 70 mg/dL
Baseline, Up to 10 Years
Change from Baseline Over Time in Real-World Overnight Fasting Tolerance
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years
Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Height
Time Frame: 10 Years
10 Years
Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Weight
Time Frame: 10 Years
10 Years
Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Body Mass Index (BMI)
Time Frame: 10 Years
10 Years
Number of Participants with Clinically Significant Laboratory Values
Time Frame: 10 Years
10 Years
Frequency of Major Clinical Events (MCEs)
Time Frame: 10 Years
10 Years
Frequency of MCEs from Review of Medical Records
Time Frame: 10 Years
10 Years
Change from Baseline in Patient Experience Clinical Interview
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years
Change from Baseline in European Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) Questionnaire Score
Time Frame: Baseline, Up to 1 Year
Baseline, Up to 1 Year
Change from Baseline in European Quality of Life 5 Dimensions Youth (EQ-5D-Y) Questionnaire Score
Time Frame: Baseline, Up to 1 Year
Baseline, Up to 1 Year
Change from Baseline Over Time in Annual Number of GSDIa Related Hospitalizations, Emergency Room Visits, Outpatient Visits, and Hospital Days
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years
Change from Baseline Over Time in Missed Work/School Days in the Past 3 Months
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years
Change from Baseline in Work/School Productivity Rating
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years
Change from Baseline in Employment
Time Frame: Baseline, Up to 10 Years
Baseline, Up to 10 Years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Ultragenyx Pharmaceutical Inc

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Study Director: Medical Director, Ultragenyx Pharmaceuticals Inc.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 4, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 1, 2036

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2036

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 20, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 9, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 10, 2024

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 26, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 25, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • DTX401-CL401
  • 2023 (U.S. NIH Grant/Contract: GRAMMY Museum Foundation)
  • 2023-510219-20-00 (Ctis)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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