ClinConnect ClinConnect Logo
Search / Trial NCT05095727

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

Launched by MODERNATX, INC. · Oct 14, 2021

Trial Information

Current as of July 21, 2025

Recruiting

Keywords

Glycogen Storage Disease Type 1a Gsd1a Von Gierke Disease Glucose Metabolism Disorder Genetic Disorder Autosomal Recessive Disorder Messenger Rna M Rna Pediatric

ClinConnect Summary

This clinical trial is studying a new treatment called mRNA-3745 for people with Glycogen Storage Disease Type 1a (GSD1a), a condition that affects how the body processes sugar. The main goal of the study is to find out if mRNA-3745 is safe and well-tolerated when given through an IV (a small tube inserted into a vein) in both adults and children with this disease. The trial is currently looking for participants, and anyone aged 2 years or older who has a confirmed diagnosis of GSD1a may be eligible to join.

To participate, individuals must have specific genetic changes related to their GSD1a diagnosis and should not have been hospitalized for low blood sugar in the month before the trial. However, people with certain medical conditions, such as recent solid organ transplants, liver tumors, or those who have received previous gene therapy for GSD1a, cannot participate. If you decide to join, you will be monitored closely to ensure your safety throughout the study. This trial could help researchers learn more about potential new treatments for GSD1a, which could benefit many patients in the future.

Gender

ALL

Eligibility criteria

  • 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.

About Modernatx, Inc.

ModernaTX, Inc. is a pioneering biotechnology company focused on developing transformative medicines based on messenger RNA (mRNA) technology. Established with a commitment to addressing unmet medical needs, Moderna leverages its proprietary platform to create innovative therapies and vaccines for a variety of diseases, including infectious diseases and cancer. The company gained global recognition for its rapid development of an mRNA-based COVID-19 vaccine, showcasing its agility and expertise in the biopharmaceutical landscape. Through robust clinical trials and a commitment to scientific excellence, ModernaTX aims to redefine the future of medicine and enhance patient outcomes worldwide.

Locations

Rochester, Minnesota, United States

Houston, Texas, United States

Durham, North Carolina, United States

Philadelphia, Pennsylvania, United States

Madrid, , Spain

Cincinnati, Ohio, United States

Houston, Texas, United States

Rochester, Minnesota, United States

Boston, Massachusetts, United States

Warsaw, , Poland

Groningen, , Netherlands

Malaga, , Spain

Edmonton, Alberta, Canada

Toronto, Ontario, Canada

Farmington, Connecticut, United States

New York, New York, United States

Salt Lake City, Utah, United States

Malaga, Avenida Carolos Haya S/N, Spain

Clamart, , France

Tours, , France

Patients applied

0 patients applied

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

Similar Trials