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)
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
THE STUDY INVOLVES:
- Screening before the treatment.
- A single infusion of DTX401 will be administered.
- Monitoring of the patient for a few hours after dosing, then minimal follow up for the next 2 years.
LOCATIONS AND CONTACTS:
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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