Glycogen Storage Disease Type Ia (GSDIa) - Healing Genes

Glycogen Storage Disease Type Ia (GSDIa)

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Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa)

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)

Phase 1


Doctors at research locations in CT, MI, and TX seek adult patients with Glycogen Storage Disease Type Ia (GSDIa) to trial a genetic therapy called DTX401. This therapy uses a disabled virus to transfer a functional copy of the gene for enzyme glucose-6-phosphatase-α, to improve the patients’ ability to regulate blood sugar (glucose).

The study will require a single IV infusion of DTX401, then follow up for a year that includes a fasting challenge with several blood draws.


  • Be male and 18 years or older
  • Documented GSDIa with confirmation by molecular testing
  • Patient’s GSDIa disease is stable as evidenced by no hospitalization for severe hypoglycemia during the 4-week period preceding the screening visit
  • Not have received a liver transplant, including hepatocyte cell therapy/transplant


  1. Screening before the treatment.
  2. A single infusion of DTX401 will be administered.
  3. Monitoring of the patient for a few hours after dosing, then minimal follow up for the next 2 years.

The three study site locations are in CT, MI, and TX:
UCONN Health in Farmington, CT, Map.
Michigan Medicine University of Michigan in Ann Arbor, MI, Map.
UT Health – McGovern Medical School in Houston, TX, Map.
Patient Advocacy  |  1-415-483-8800  |  [email protected]
HCPs Contact  |  1-888-756-8657  |  [email protected]
Ultragenyx Pharmaceutical Inc
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