A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
Glycogen Storage DiseaseThe 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.
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Participation Requirements
-
Sex:
ALL -
Eligible Ages:
6 and up
Participation Criteria
Inclusion Criteria:
* Documented GSD1a with confirmation of biallelic gene encoding glucose-6-phosphatase-α (G6PC) mutations by genetic testing.
* 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
Note: Additional inclusion/exclusion criteria may apply, per protocol.
Study Location
Stollery Children's Hospital University of Alberta
Stollery Children's Hospital University of AlbertaEdmonton, Alberta
Canada
Contact Study Team
The Hospital for Sick Children
The Hospital for Sick ChildrenToronto, Ontario
Canada
Contact Study Team
- Study Sponsored By
- ModernaTX, Inc.
- Participants Required
- More Information
- Study ID:
NCT05095727