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Completed

NCT Number: NCT03970278

Study of Long-Term Safety and Efficacy on Gene Therapy in Glycogen Storage Disease Type Ia

The primary objective of this study is to determine the long-term safety of DTX401 following a single intravenous (IV) dose in adults with GSDIa.

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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 Von Gierke's Disease (GSD Type Ia)

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Montreal Children Hospital, McGill University Health Centre, Montreal, Quebec, Canada

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About this study

Only participants who received DTX401 in study 401GSDIA01 (NCT03517085) are eligible to participate in study 401GSDIA02. No investigational product will be administered during study 401GSDIA02. Participants will be followed in study 401GSDIA02 for at least 4 years, and up to 6 years after administration of DTX401.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Received DTX401 in study 401GSDIA01.
  • Willing and able to provide written informed consent after the nature of the study has been explained, and prior to any research-related procedures being performed.
  • Willing and able to comply with all scheduled study visits, procedures, and requirements.

Exclusion criteria

  • Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study.
  • Presence or history of any condition that, in the view of the Investigator, poses a risk to subject safety or places the subject at high risk of poor compliance or not completing the study or that would significantly affect the interpretation of study results.

Treatment and study plan

No intervention

Other

No intervention

Primary outcomes

  1. Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and Discontinuations Due to TEAEs

    Time frame: From Baseline (Week 52 of 401GSDIA01/Visit 1 of 401GSDIA02) Up to Week 329

    An adverse event (AE) is defined as any untoward medical occurrence, regardless of its causal relationship to study product. A TEAE is defined as any AE not present prior to the initiation of the drug treatment or any AE already present that worsens in either intensity or frequency following exposure to the drug treatment. A serious TEAE is an AE that meets any of the following criteria in the view of either the Investigator or Ultragenyx: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; disability/Incapacity; congenital anomaly/birth defect not present at screening; other important medical events. Severity of events were graded as mild (grade 1), moderate (grade 2), severe (grade 3), life-threatening (grade 4), or death (grade 5). As DTX401 was administered as part of Study 401GSDIA01, all AEs in Study 401GSDIA02 were considered TEAEs.

Secondary outcomes

  1. Change From Baseline in Time to First Hypoglycemic Event During a Controlled Fasting Challenge Over Time

    Time frame: Baseline (Week 0 of 401GSDIA01), Weeks 52 (Visit 1 of 401GSDIA02), 78, 104, 130, 156, 182, 208, 234, 260, Last Visit (Up to Week 329)

    The change from baseline in time (in hours) to first hypoglycemic event (defined as glucose < 54 mg/dL [< 3.0 mmol/L]) during a controlled fasting challenge at the participant's last visit. A positive change from baseline is favorable. Change from baseline is calculated from Baseline (Week 0) of the 401GSDIA01 study.

Sponsors and collaborators

Lead sponsor

Ultragenyx Pharmaceutical Inc

Industry

Registry information

Official study title

A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

Important dates

Study start
2019
Primary completion
2025
Study completion
2025
First posted
May 31, 2019
Registry last updated
Mar 18, 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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