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

Purpose

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.

Condition

  • Glycogen Storage Disease

Eligibility

Eligible Ages
Over 6 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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.

Study Design

Phase
Phase 1/Phase 2
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
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.
  • Drug: mRNA-3745
    Sterile frozen liquid dispersion for injection
Experimental
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.
  • Drug: mRNA-3745
    Sterile frozen liquid dispersion for injection

Recruiting Locations

The University of Texas Health Science Center at Houston
Houston, Texas 77030-1501
Contact:
713-500-7098
heather.saavedra@uth.tmc.edu

More Details

Status
Recruiting
Sponsor
ModernaTX, Inc.

Study Contact

Moderna Clinical Trials Support Center
1-877-777-7187
clinicaltrials@modernatx.com

Detailed 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.