An Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of mRNA-3745 in Participants with Glycogen Storage Disease Type 1a (GSD1a)

Clinicaltrials.gov ID# 05095727

This is an open label study for adults with GSD1a that evaluates pharmacokinetics and pharmacodynamics following a single dose of mRNA-3745.  Patients undergo an inpatient controlled fasting challenge for up to 8 hours, on 3 separate occasions to assess effect of mRNA-3745. Patients must be >18 years old, have documented GSD1a by genetic testing and a history of hypoglycemic event or symptoms of hypoglycemia without acute illness within 4 weeks prior to enrollment.  Patients are excluded if they have a history of liver transplant, hepatocyte cell therapy/transplant, prior gene therapy for GSD1a, presence of liver adenoma growth of >2cm or adenoma >5cm in the past 2 years.

Recruitment status: not yet active at BCH. 2 international sites and 4 sites in USA are actively recruiting PI:  Dr. Walla Al-Hertani

Contact information: 

Dr. Walla Al-Hertani  -- Walla.Al-Hertani@childrens.harvard.edu

Lora Pixley, study coordinator – Lora.Pixley@childrens.harvard.edu