A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
About the study
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.
Who can take part
You may be eligible to participate in the study if you meet the following criteria:
INCLUSION CRITERIA
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
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.
Study Locations
Enter your ZIP code/Postal code/PIN code to locate study sites near you:
How to Apply
Contact the study center to learn if this study is a good match for you.
Study Details
Contition
Glycogen Storage Disease
Age
6+
Phase
PHASE1/PHASE2
Participants Needed
45
Est. Completion Date
Dec 26, 2028
Treatment Type
INTERVENTIONAL
Sponsor
ModernaTX, Inc.
ClinicalTrials.gov NCT Identifier
NCT05095727
Study Number
mRNA-3745-P102
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