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Long-Term Follow-up 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)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03970278
Recruitment Status : Enrolling by invitation
First Posted : May 31, 2019
Last Update Posted : September 4, 2019
Information provided by (Responsible Party):
Ultragenyx Pharmaceutical Inc

Brief Summary:
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.

Condition or disease Intervention/treatment
Glycogen Storage Disease Type IA Von Gierke's Disease (GSD Type Ia) Other: No intervention

Detailed Description:
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.

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Study Type : Observational
Estimated Enrollment : 12 participants
Observational Model: Cohort
Time Perspective: Prospective
Official 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)
Actual Study Start Date : July 15, 2019
Estimated Primary Completion Date : October 2024
Estimated Study Completion Date : October 2024

Resource links provided by the National Library of Medicine

Group/Cohort Intervention/treatment
All Participants
All participants enrolled in study 401GSDIA02 will have received a single IV dose of DTX401 during their participation in study 401GSDIA01 (NCT03517085).
Other: No intervention
No intervention

Primary Outcome Measures :
  1. Number of Participants With Adverse Events (AEs), Serious AEs and Discontinuations Due to AEs [ Time Frame: Up to 260 weeks following DTX401 administration ]

Secondary Outcome Measures :
  1. Change in Time to First Hypoglycemic Event During a Controlled Fasting Challenge from Day 0 (Study 401GSDIA01) to 260 weeks [ Time Frame: Up to 260 weeks following DTX401 administration ]

Biospecimen Retention:   Samples With DNA

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Subjects 18 years of age or older with GSDIa previously enrolled in 401GSDIA01.

Inclusion Criteria:

  1. Received DTX401 in study 401GSDIA01.
  2. 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.
  3. Willing and able to comply with all scheduled study visits, procedures, and requirements.

Exclusion Criteria:

  1. Planned or current participation in any other interventional clinical study that may confound the safety or efficacy evaluation of DTX401 during this study.
  2. 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03970278

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United States, Connecticut
UCONN Health
Farmington, Connecticut, United States, 06030-3213
United States, Michigan
Michigan Medicine University of Michigan
Ann Arbor, Michigan, United States, 48109
Sponsors and Collaborators
Ultragenyx Pharmaceutical Inc
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Study Director: Medical Director Ultragenyx Pharmaceutical Inc

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Responsible Party: Ultragenyx Pharmaceutical Inc Identifier: NCT03970278     History of Changes
Other Study ID Numbers: 401GSDIA02
2018-004473-27 ( EudraCT Number )
First Posted: May 31, 2019    Key Record Dates
Last Update Posted: September 4, 2019
Last Verified: September 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Due to the rarity of GSDIa and the small number of subjects in this trial, individual patient data will not be shared in order to safeguard patient privacy, consistent with the data sharing commitment statement listed on

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Studies a U.S. FDA-regulated Drug Product: Yes
Keywords provided by Ultragenyx Pharmaceutical Inc:
gene therapy
glucose metabolism disorder
carbohydrate metabolism, inborn errors
genetic diseases
Inborn Metabolic Diseases
Additional relevant MeSH terms:
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Glycogen Storage Disease
Glycogen Storage Disease Type I
Metabolic Diseases
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn