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

Description

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.

Conditions

Glycogen Storage Disease

Talk to us

Study Overview

On this page

Study Details

Study overview

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.

A Phase 1/2, Adaptive, Open-label, Single Ascending Dose to Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of mRNA-3745 in Participants With Glycogen Storage Disease Type 1a (GSD1a), Followed by an Open-label Extension

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

Condition
Glycogen Storage Disease
Intervention / Treatment

-

Contacts and Locations

Farmington

University of Connecticut Health Center, Farmington, Connecticut, United States, 06030-0001

Boston

Boston Children's Hospital, Boston, Massachusetts, United States, 02115

Durham

Duke University Medical Center, Durham, North Carolina, United States, 27713

Cincinnati

Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States, 45229

Philadelphia

Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States, 19104

Houston

The University of Texas Health Science Center at Houston, Houston, Texas, United States, 77030-1501

Houston

Baylor College of Medicine, Houston, Texas, United States, 77030

Salt Lake City

University of Utah, Salt Lake City, Utah, United States, 84132-0001

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

6 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

ModernaTX, Inc.,

Study Record Dates

2028-12-26