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NCT Number: NCT06636383

Glycogen Storage Disease Type Ia (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.

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Key information

Conditions

Glycogen Storage Disease Type IA Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glucose Metabolism Disorders Glycogen Storage Disease Glycogen Storage Disease Type I Hepatorenal form of glycogen storage disease Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

2 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Hospital de Clinicas de Porto Alegre (HCPA) - PPDS, Porto Alegre, Rio Grande do Sul, Brazil

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

No intervention

Other

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

Primary outcomes

  1. Incidence and Severity of Serious Adverse Events (SAEs) Assessed as Related to DTX401 by the Investigator

    Time frame: 10 Years

  2. Incidence, Relationship, Severity and Seriousness of Adverse Events of Special Interest (AESIs) for Adeno-Associated Virus (AAV) Therapies

    Time frame: 10 Years

  3. Incidence of Pregnancy in Patients Treated with DTX401 or Patient's Partner

    Time frame: 10 Years

  4. Outcomes of Pregnancy in Patients Treated with DTX401 or Patient's Partner

    Time frame: 10 Years

  5. Incidence and Severity of SAEs of Infusion-Related Reactions Including Hypersensitivity (Group 2 only)

    Time frame: 1 Year

  6. Incidence and Severity of SAEs Assessed by the Investigator as Related to Concomitant Immunomodulatory Therapies (Group 2 only)

    Time frame: 1 Year

Secondary outcomes

  1. Percent Change Over Time from Baseline in Total Cornstarch Intake

    Time frame: Baseline, Up to 10 Years

  2. Change from Baseline Over Time in Frequency of Cornstarch Intake

    Time frame: Baseline, Up to 10 Years

  3. Change from Baseline Over Time in Number of Nighttime Awakenings for Cornstarch

    Time frame: Baseline, Up to 10 Years

  4. Change from Baseline Over Time in Dependance on Continuous Overnight Tube Feeding for Exogenous Glucose Delivery

    Time frame: Baseline, Up to 10 Years

  5. 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

  6. 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

  7. 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

  8. 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

  9. Change from Baseline Over Time in Real-World Overnight Fasting Tolerance

    Time frame: Baseline, Up to 10 Years

  10. Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Height

    Time frame: 10 Years

  11. Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Weight

    Time frame: 10 Years

  12. Change from Baseline Over Time in Metabolic and GSDIa Related Parameters: Body Mass Index (BMI)

    Time frame: 10 Years

  13. Number of Participants with Clinically Significant Laboratory Values

    Time frame: 10 Years

  14. Frequency of Major Clinical Events (MCEs)

    Time frame: 10 Years

  15. Frequency of MCEs from Review of Medical Records

    Time frame: 10 Years

  16. Change from Baseline in Patient Experience Clinical Interview

    Time frame: Baseline, Up to 10 Years

  17. Change from Baseline in European Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) Questionnaire Score

    Time frame: Baseline, Up to 1 Year

  18. Change from Baseline in European Quality of Life 5 Dimensions Youth (EQ-5D-Y) Questionnaire Score

    Time frame: Baseline, Up to 1 Year

  19. 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

  20. Change from Baseline Over Time in Missed Work/School Days in the Past 3 Months

    Time frame: Baseline, Up to 10 Years

  21. Change from Baseline in Work/School Productivity Rating

    Time frame: Baseline, Up to 10 Years

  22. Change from Baseline in Employment

    Time frame: Baseline, Up to 10 Years

Study contacts

Contact information is provided by the study sponsor or research team.

HCPs Contact: Medical Information

CONTACT

[email protected]

1-888-756-8657

Patients Contact: Trial Recruitment

CONTACT

[email protected]

1-888-756-8657

Sponsors and collaborators

Lead sponsor

Ultragenyx Pharmaceutical Inc

Industry

Registry information

Official study title

GSDIa Disease Monitoring Program

Important dates

Study start
2024
Primary completion
2036
Study completion
2036
First posted
Oct 10, 2024
Registry last updated
Jul 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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