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

Last updated: March 22, 2025
Sponsor: ModernaTX, Inc.
Overall Status: Active - Recruiting

Phase

1/2

Condition

Liver Disorders

Metabolic Disorders

Treatment

mRNA-3745

Clinical Study ID

NCT05095727
mRNA-3745-P102
2022-502963-39-00
  • Ages > 6
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Documented GSD1a with confirmation of biallelic gene encodingglucose-6-phosphatase-α (G6PC) mutations by genetic testing.

  • Absence of hospitalization for hypoglycemia in the 4 weeks prior to Screening

Exclusion

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.

Study Design

Total Participants: 45
Treatment Group(s): 1
Primary Treatment: mRNA-3745
Phase: 1/2
Study Start date:
June 01, 2022
Estimated Completion Date:
December 26, 2028

Study Description

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.

Connect with a study center

  • Stollery Children's Hospital University of Alberta

    Edmonton, Alberta T6G 2R7
    Canada

    Active - Recruiting

  • The Hospital for Sick Children

    Toronto, Ontario M5G 1X8
    Canada

    Active - Recruiting

  • AP-HP - Hôpital Antoine Béclère

    Clamart, 92140
    France

    Active - Recruiting

  • CHRU Tours - Hopital Clocheville

    Tours, 37000
    France

    Active - Recruiting

  • Universitair Medisch Centrum Groningen

    Groningen, 9713 GZ
    Netherlands

    Active - Recruiting

  • Instytut Pomnik Centrum Zdrowia Dziecka

    Warsaw, 04-730
    Poland

    Active - Recruiting

  • Hospital Universitario 12 de Octubre

    Madrid, 28026
    Spain

    Active - Recruiting

  • Hospital Regional Universitario de Malaga

    Malaga, 29011
    Spain

    Active - Recruiting

  • University of Connecticut Health Center

    Farmington, Connecticut 06030-0001
    United States

    Active - Recruiting

  • Boston Children's Hospital

    Boston, Massachusetts 02115
    United States

    Terminated

  • Mayo Clinic

    Rochester, Minnesota 55905
    United States

    Site Not Available

  • Columbia University Medical Center

    New York, New York 10032-3822
    United States

    Site Not Available

  • Duke University Medical Center

    Durham, North Carolina 27713
    United States

    Active - Recruiting

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio 45229
    United States

    Terminated

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

  • Baylor College of Medicine

    Houston, Texas 77030
    United States

    Active - Recruiting

  • The University of Texas Health Science Center at Houston

    Houston, Texas 77030-1501
    United States

    Active - Recruiting

  • University of Utah

    Salt Lake City, Utah 84132-0001
    United States

    Active - Recruiting

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