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

Sponsored by ModernaTX, Inc.

About this trial

Last updated 2 years ago

Study ID

mRNA-3745-P102

Status

Recruiting

Type

Interventional

Phase

Phase 1

Placebo

No

Accepting

18-75 Years
12+ Years
All
All

Not accepting

Not accepting
Healthy Volunteers

Trial Timing

Started 3 years ago

What is this trial about?

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.

What are the participation requirements?

Yes

Inclusion Criteria

- Documented GSD1a with confirmation by genetic testing

- 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)

No

Exclusion Criteria

- Liver transplant, including hepatocyte cell therapy/transplant

- Received gene therapy for GSD1a

- Presence of liver adenoma >5 centimeters (cm) (±10%) in size

- Participant has a 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

- 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.

Locations

Location

Status

Recruiting
Recruiting
Recruiting
Recruiting
Recruiting
Recruiting
Recruiting
Recruiting