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NCT Number: NCT05095727

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

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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This study is active but is not currently recruiting participants.

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Key information

Conditions

Glycogen Storage Disease Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Glucose Metabolism Disorders Glycogen Storage Disease Type I Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases

Age range

6 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

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

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About this study

The study includes a single ascending dose (SAD) stage and a multiple ascending dose (MAD) stage. Participants enrolled in the MAD stage have the option to continue treatment in an open-label extension (OLE) period that will assess long-term safety and clinical activity of mRNA-3745.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for 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.

Treatment and study plan

mRNA-3745

Drug

Sterile frozen liquid dispersion for injection

Primary outcomes

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and TEAEs Leading to Treatment Discontinuation

    Time frame: Day 1 up to approximately 3.5 years

Secondary outcomes

  1. Number of Participants Not Experiencing Hypoglycemia During Fasting Challenges

    Time frame: Baseline through up to Week 32

    Hypoglycemia is defined as blood glucose <60 milligrams (mg)/deciliter (dL) (3.3 millimoles [mmol]/liter [L]) and/or symptoms of hypoglycemia.

  2. Change From Baseline of Area Under the Effect Curve (AUEC) of Blood Glucose and Lactate During Fasting Challenges

    Time frame: Baseline through up to Week 32

  3. Change From Baseline in Time to Hypoglycemia During Fasting Challenges

    Time frame: Baseline through up to Week 32

  4. Change From Baseline in Maximum Effect (Emax) During Fasting Challenges

    Time frame: Baseline through up to Week 32

  5. SAD only: Maximum Observed Concentration (Cmax) of Messenger Ribonucleic Acid (mRNA) and Lipid Nanoparticle (LNP)

    Time frame: Pre-infusion, during infusion, at the end of infusion (EOI) and post-infusion on Day 1 up to Week 52

  6. SAD only: Area Under the Concentration-Time Curve From Time 0 to the Time of the Last Measurable Concentration (AUC0-t) of mRNA and LNP

    Time frame: Pre-infusion, during infusion, at EOI and post-infusion on Day 1 up to Week 52

  7. Change From Baseline in Metabolic Biomarkers of GSD1a

    Time frame: Baseline through up to approximately 6.5 years

  8. MAD only: Maximum Observed Concentration at Steady State (Cmax,ss) of mRNA and LNP

    Time frame: Pre-infusion, during infusion, at the EOI and post-infusion on Day 1 up to Week 52

Sponsors and collaborators

Lead sponsor

ModernaTX, Inc.

Industry

Registry information

Official study title

A Phase 1/2, Adaptive, Open-label, Single Ascending Dose to Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of mRNA-3745 in Participants With Glycogen Storage Disease Type 1a (GSD1a), Followed by an Open-label Extension

Important dates

Study start
2022
Primary completion
2026
Study completion
2026
First posted
Oct 27, 2021
Registry last updated
Dec 15, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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