A Study of mRNA-3745 in Adult and Pediatric Participants With Glycogen Storage Disease Type 1a (GSD1a)
NCT ID: NCT05095727
Last Updated: 2025-12-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
15 participants
INTERVENTIONAL
2022-06-01
2026-11-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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SAD: mRNA-3745
Participants will receive a single intravenous (IV) dose of mRNA-3745 on Day 1 in an inpatient setting. Participants that are/have been enrolled in the study and receive an administration of mRNA-3745 may also enroll in one of the MAD cohorts. The first MAD dose must occur at least 21 days after the SAD dose.
mRNA-3745
Sterile frozen liquid dispersion for injection
MAD: mRNA-3745
Participants will receive multiple IV doses of mRNA-3745 in an inpatient setting. Participants will have the option to continue treatment in the OLE.
mRNA-3745
Sterile frozen liquid dispersion for injection
Interventions
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mRNA-3745
Sterile frozen liquid dispersion for injection
Eligibility Criteria
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Inclusion Criteria
* Absence of hospitalization for hypoglycemia in the 4 weeks prior to Screening
Exclusion Criteria
* 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
6 Years
ALL
No
Sponsors
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ModernaTX, Inc.
INDUSTRY
Responsible Party
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Locations
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University of Connecticut Health Center
Farmington, Connecticut, United States
Boston Children's Hospital
Boston, Massachusetts, United States
Duke University Medical Center
Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
The University of Texas Health Science Center at Houston
Houston, Texas, United States
Baylor College of Medicine
Houston, Texas, United States
University of Utah
Salt Lake City, Utah, United States
Stollery Children's Hospital University of Alberta
Edmonton, Alberta, Canada
The Hospital for Sick Children
Toronto, Ontario, Canada
AP-HP - Hôpital Antoine Béclère
Clamart, , France
CHRU Tours - Hopital Clocheville
Tours, , France
Universitair Medisch Centrum Groningen
Groningen, , Netherlands
Instytut Pomnik Centrum Zdrowia Dziecka
Warsaw, , Poland
Hospital Universitario 12 de Octubre
Madrid, , Spain
Hospital Regional Universitario de Malaga
Málaga, , Spain
Countries
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References
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Cao J, Choi M, Guadagnin E, Soty M, Silva M, Verzieux V, Weisser E, Markel A, Zhuo J, Liang S, Yin L, Frassetto A, Graham AR, Burke K, Ketova T, Mihai C, Zalinger Z, Levy B, Besin G, Wolfrom M, Tran B, Tunkey C, Owen E, Sarkis J, Dousis A, Presnyak V, Pepin C, Zheng W, Ci L, Hard M, Miracco E, Rice L, Nguyen V, Zimmer M, Rajarajacholan U, Finn PF, Mithieux G, Rajas F, Martini PGV, Giangrande PH. mRNA therapy restores euglycemia and prevents liver tumors in murine model of glycogen storage disease. Nat Commun. 2021 May 25;12(1):3090. doi: 10.1038/s41467-021-23318-2.
Other Identifiers
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2022-502963-39-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
mRNA-3745-P102
Identifier Type: -
Identifier Source: org_study_id
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