New mRNA therapy aims to control blood sugar in rare genetic disease

NCT ID NCT05095727

Ongoing Disease control Sponsor: ModernaTX, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage trial tests an mRNA drug called mRNA-3745 in 15 adults and children with glycogen storage disease type 1a (GSD1a), a rare genetic condition that causes dangerously low blood sugar. The drug is given by IV infusion and aims to help the body produce a missing enzyme. The main goal is to check safety and tolerability, with a secondary focus on whether it prevents hypoglycemia during fasting challenges.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: mRNA-3745 (a messenger RNA drug given by IV infusion)
What this could lead to: If successful, this could lead to a new treatment that helps control blood sugar levels and reduces the risk of dangerously low blood sugar in people with GSD1a.
What could go wrong: This is an early-phase trial with only 15 participants, so results may not apply to everyone. The treatment is new and may cause side effects or not work as hoped.

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Conditions

The condition(s) this trial relates to.

disorder of glycogen metabolism glucose metabolism disease glycogen storage disease due to glucose-6-phosphatase deficiency type IA glycogen storage disease I hereditary disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

GLYCOGEN STORAGE DISEASE

Contacts and locations

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Locations

AP-HP - Hôpital Antoine Béclère

Clamart, 92140, France
Baylor College of Medicine

Houston, Texas, 77030, United States
Boston Children's Hospital

Boston, Massachusetts, 02115, United States
CHRU Tours - Hopital Clocheville

Tours, 37000, France
Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States
Duke University Medical Center

Durham, North Carolina, 27713, United States
Hospital Regional Universitario de Malaga

Málaga, 29011, Spain
Hospital Universitario 12 de Octubre

Madrid, 28026, Spain
Instytut Pomnik Centrum Zdrowia Dziecka

Warsaw, 04-730, Poland
Stollery Children's Hospital University of Alberta

Edmonton, Alberta, T6G 2R7, Canada
The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada
The University of Texas Health Science Center at Houston

Houston, Texas, 77030-1501, United States
Universitair Medisch Centrum Groningen

Groningen, 9713 GZ, Netherlands
University of Connecticut Health Center

Farmington, Connecticut, 06030-0001, United States
University of Utah

Salt Lake City, Utah, 84132-0001, United States

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