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.
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.
Study Type
OBSERVATIONAL
Enrollment
12
No intervention
UCONN Health
Farmington, Connecticut, United States
Michigan Medicine University of Michigan
Ann Arbor, Michigan, United States
University of Texas Health Science Center at Houston
Houston, Texas, United States
Montreal Children Hospital, McGill University Health Centre
Montreal, Quebec, Canada
Number of Participants With Adverse Events (AEs), Serious AEs and Discontinuations Due to AEs
Time frame: Up to 312 weeks following DTX401 administration
Change in Time to First Hypoglycemic Event During a Controlled Fasting Challenge from Day 0 (Study 401GSDIA01) to 312 weeks
Time frame: Up to 312 weeks following DTX401 administration
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
University Medical Center Groningen
Groningen, Netherlands
Complejo Hospitalario Universitario de Santiago
Santiago de Compostela, A Coruna, Spain