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

Glycogen Storage Disease

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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Conditions de participation

  • Sexe:

    ALL

  • Âges admissibles:

    6 and up

Critères de participation

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.

Lieu de l'étude

Stollery Children's Hospital University of Alberta
Stollery Children's Hospital University of Alberta
Edmonton, Alberta
Canada

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The Hospital for Sick Children
The Hospital for Sick Children
Toronto, Ontario
Canada

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Étude parrainée par
ModernaTX, Inc.
Participants recherchés
Plus d'informations
ID de l'étude: NCT05095727