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
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.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
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
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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