Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)
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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. |
ClinicalTrials.gov Identifier: NCT03517085 |
Recruitment Status :
Completed
First Posted : May 7, 2018
Last Update Posted : November 23, 2021
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
GSD1 | Genetic: DTX401 | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 12 participants |
Allocation: | Non-Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase 1/2, Open-Label Safety and Dose-Finding Study 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 24, 2018 |
Actual Primary Completion Date : | November 2, 2021 |
Actual Study Completion Date : | November 2, 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: DTX401 Dose 1
DTX401 solution for intravenous (IV) infusion
|
Genetic: DTX401
DTX401 administered as a single peripheral IV infusion
Other Name: AAV8G6PC |
Experimental: DTX401 Dose 2
DTX401 solution for intravenous (IV) infusion
|
Genetic: DTX401
DTX401 administered as a single peripheral IV infusion
Other Name: AAV8G6PC |
Experimental: DTX401 Dose 3
DTX401 solution for intravenous (IV) infusion
|
Genetic: DTX401
DTX401 administered as a single peripheral IV infusion
Other Name: AAV8G6PC |
Experimental: DTX401 Dose 4
DTX401 solution for intravenous (IV) infusion
|
Genetic: DTX401
DTX401 administered as a single peripheral IV infusion
Other Name: AAV8G6PC |
- Adverse Events (AEs), Treatment-emergent Adverse Events (TEAEs), and Serious Adverse Events (SAEs) [ Time Frame: Up to 52 Weeks ]
- Change from Baseline in Time (Minutes) to First Hypoglycemic Event During a Controlled Fasting Challenge at Weeks 12, 24, and 52 [ Time Frame: Baseline and Weeks 12, 24, and 52 ]The change from baseline in time (in minutes) to first hypoglycemic event (defined as glucose <54 mg/dL [<3.0 mmol/L]) during a controlled fasting challenge after IV administration of DTX401).

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Key Inclusion Criteria:
- Males and females ≥18 years of age
- Documented GSDIa with confirmation by molecular testing
- Documented history of ≥1 hypoglycemic event with blood glucose <60 mg/dL (<3.33 mmol/L)
- Patient's GSDIa disease is stable as evidenced by no hospitalization for severe hypoglycemia during the 4-week period preceding the screening visit
Key Exclusion Criteria:
- Anti-AAV8 neutralizing antibody titer ≥1:5
- Screening or Baseline (Day 0) blood glucose level <60 mg/dL (<3.33 mmol/L)
- Liver transplant, including hepatocyte cell therapy/transplant
- Presence of liver adenoma >5 cm in size
- Presence of liver adenoma >3 cm and ≤5 cm in size that has a documented annual growth rate of ≥0.5 cm per year
- Significant hepatic inflammation or cirrhosis as evidenced by imaging or any of the following laboratory abnormalities: alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > upper limit of normal (ULN), total bilirubin >1.5 × ULN, alkaline phosphatase >2.5 × ULN
Note additional inclusion/exclusion criteria may apply, per protocol.

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 ClinicalTrials.gov identifier (NCT number): NCT03517085
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 | |
United States, Texas | |
UT Health - McGovern Medical School | |
Houston, Texas, United States, 77030 | |
Canada, Quebec | |
Montreal Children Hospital, McGill University Health Centre | |
Montréal, Quebec, Canada, H4A3J1 | |
Netherlands | |
University Medical Center Groningen | |
Groningen, Netherlands, 9700RB | |
Spain | |
Complejo Hospitalario Universitario de Santiago | |
Santiago De Compostela, A Coruna, Spain, 15706 |
Study Director: | Medical Director | Ultragenyx Pharmaceutical Inc |
Responsible Party: | Ultragenyx Pharmaceutical Inc |
ClinicalTrials.gov Identifier: | NCT03517085 |
Other Study ID Numbers: |
401GSDIA01 1706-1617 ( Other Identifier: NIH Protocol Registration Number ) |
First Posted: | May 7, 2018 Key Record Dates |
Last Update Posted: | November 23, 2021 |
Last Verified: | November 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
glycogen storage disorder Ia AAV gene therapy von Gierke disease glucose metabolism disorder |
Glycogen Storage Disease Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Metabolic Diseases |