NCT03517085View on ClinicalTrials.gov

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

PHASE1/PHASE2CompletedINTERVENTIONAL

Enrollment

Participants

Timeline

Start Date

May 18, 2018

Primary Completion Date

November 2, 2021

Study Completion Date

November 2, 2021

Conditions

GSD1

Interventions

GENETIC

DTX401

DTX401 administered as a single peripheral intravenous (IV) infusion

DRUG

steroid regimen

prednisone or prednisolone to manage alanine aminotransferase (ALT) elevation

Trial Locations (6)

15706

Complejo Hospitalario Universitario de Santiago, Santiago de Compostela

48109

Michigan Medicine University of Michigan, Ann Arbor

77030

UT Health - McGovern Medical School, Houston

06030-3213

UCONN Health, Farmington

H4A3J1

Montreal Children Hospital, McGill University Health Centre, Montreal

9700RB

University Medical Center Groningen, Groningen

All Listed Sponsors

lead

Ultragenyx Pharmaceutical Inc

INDUSTRY

NCT03517085 - Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa) | Biotech Hunter | Biotech Hunter