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

mRNA-3745-P102

The main goal of this trial is to evaluate the safety and tolerability of mRNA-3745 via intravenous (IV) administration in adult and pediatric participants with GSD1a.

2022-06-01

2026-01-07

2028-12-26

Recruiting

Early phase I

45

Inclusion Criteria: * Documented GSD1a with confirmation by genetic testing * Documented history of a symptomatic hypoglycemic event with blood glucose \<60 mg/dL (\<3.3 mmol/L) * Absence of hospitalization for hypoglycemia in the 4 weeks prior to Screening Exclusion Criteria: * Solid organ transplant * Received gene therapy for GSD1a * Presence of liver adenoma \>5 centimeters (cm) in size * Diagnosis of type 1 or type 2 diabetes mellitus * Presence of liver adenoma with growth of \>2 cm or \>5 newly diagnosed liver adenomas, in the previous 2 years * Requirement for continuous feeds via gastrostomy or nasogastric tubes Note: Additional inclusion/exclusion criteria may apply, per protocol.

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 45, 'type': 'ESTIMATED'}}

2024-05-06