Baylor College of Medicine

A Study to Evaluate mRNA-3745 in Participants with Glycogen Storage Disease Type 1a (GSD1a) (H-51770)



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)

Objectives: Primary Objectives

  • Evaluate the safety and tolerability of messenger RNA encoding human G6Pase-α S298C encapsulated in SM-716, OL-56, Cholesterol, and DSPC (mRNA-3745) via intravenous (IV) administration in participants with glycogen storage disease 1a (GSD1a) Secondary Objectives
  • Characterize the pharmacodynamic (PD) response following IV administration of mRNA-3745 assessed by evaluation of blood glucose and lactate levels during fasting challenges
  • Characterize the pharmacokinetics (PK) of mRNA encoding human glucose-6-phosphatase-α (hG6PC-α mRNA) and of lipid SM-716, following IV administration of mRNA-3745
  • Characterize the PD response to mRNA-3745 as assessed by other GSD1a metabolic biomarkers such as uric acid, triglycerides, low-density lipoprotein (LDL), and high-density lipoprotein (HDL) Exploratory Objectives
  • Characterize the change in interstitial glucose trends obtained through a continuous glucose monitoring (CGM) device, following IV administration of mRNA-3745
  • Assess for the presence and development of anti-polyethylene glycol (PEG) antibodies and antibodies to the therapeutic protein ie, hG6Pase-α)


Dalia Attia

Phone 1: 713–798–7862

IRB: H-51770




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