A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)

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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.
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Who can take part

You may be eligible to participate in the study if you meet the following criteria:

INCLUSION CRITERIA

Inclusion Criteria:

  1. Documented GSD1a with confirmation by genetic testing
  2. Documented history of ≥1 hypoglycemic event with blood glucose <60 mg/dL (<3.3 mmol/L) and symptoms of hypoglycemia in the absence of acute illness, with at least one such event in the 4 weeks before starting run in (Day -14)

EXCLUSION CRITERIA

Exclusion Criteria:

  1. Liver transplant, including hepatocyte cell therapy/transplant
  2. Received gene therapy for GSD1a
  3. Presence of liver adenoma >5 centimeters (cm) (±10%) in size
  4. Participant has a diagnosis of type 1 or type 2 diabetes mellitus
  5. Presence of liver adenoma with growth of >2 cm or >5 newly diagnosed liver adenomas, in the previous 2 years
  6. Participants with known allergy to magnetic resonance imaging (MRI) contrast can be enrolled and receive MRI without contrast. Participants for whom MRI is contraindicated could be enrolled if an alternative imaging technique can be performed.

Note: Additional inclusion/exclusion criteria may apply, per protocol.

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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’s details


Contition
Glycogen Storage Disease
Age (in years)
12+
Phase
Phase 1
Participants needed
33
Est. Completion Date
Oct 21, 2025
Treatment type
Interventional

Sponsor
ModernaTX, Inc.
ClinicalTrials.gov identifier
NCT05095727
Study number
mRNA-3745-P102

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