A Phase 1/2, adaptive, open-label, single ascending dose to multiple ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of mRNA-3745 in participants with glycogen storage disease type 1a (GSD1a), followed by an open-label extension

Glycogen storage disease type 1a (GSD1a)

• Incidence and severity of TEAEs, SAEs, and TEAEs leading to treatment discontinuation. • Changes in vital signs, ECG results, and laboratory findings (including hematology, serum chemistry, urinalysis, and coagulation parameters).

• Absence of hypoglycemiaa for up to 12 hours during fasting challenges. • Change from baseline of area under the AUEC of blood glucose and lactate during fasting challenges. • Change from baseline in time to hypoglycemia during fasting challenges. Change in other PD parameters including Emax and TEmax, during fasting challenges., • PK parameters determined during the SAD portion of the study including, but not limited to Cmax, Tmax, AUC0-t, AUC0-inf, t1/2, CL, and Vz. • Additional PK parameters determined during the MAD portion of the study including, but not limited to Cmax,ss, AUC0-t,ss, Vss, and Rac determined by the ratio of AUC0-t,ss and AUC0-t. • Change from baseline in metabolic biomarkers such as blood levels of uric acid, triglycerides, LDL, and HDL.

No related papers found yet.

France, Netherlands, Poland, Spain