ACTIVE_NOT_RECRUITING

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

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2022-06-01
Study Completion:2026-11-30
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05095727

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.

Ages Eligible for Study:6 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Documented GSD1a with confirmation of biallelic gene encoding glucose-6-phosphatase-α (G6PC) mutations by genetic testing.
  2. * Absence of hospitalization for hypoglycemia in the 4 weeks prior to Screening
  1. * Solid organ transplant
  2. * Received gene therapy for GSD1a
  3. * Presence of liver adenoma \>5 centimeters (cm) in size
  4. * Diagnosis of type 1 or type 2 diabetes mellitus
  5. * Presence of liver adenoma with growth of \>2 cm or \>5 newly diagnosed liver adenomas, in the previous 2 years

Contacts and Locations

Study Locations (Sites)

University of Connecticut Health Center
Farmington, Connecticut, 06030-0001
United States
Boston Children's Hospital
Boston, Massachusetts, 02115
United States
Duke University Medical Center
Durham, North Carolina, 27713
United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
United States
The University of Texas Health Science Center at Houston
Houston, Texas, 77030-1501
United States
Baylor College of Medicine
Houston, Texas, 77030
United States
University of Utah
Salt Lake City, Utah, 84132-0001
United States

Collaborators and Investigators

Sponsor: ModernaTX, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2022-06-01
Study Completion Date2026-11-30

Study Record Updates

Study Start Date2022-06-01
Study Completion Date2026-11-30

Terms related to this study

Keywords Provided by Researchers

  • Glycogen storage disease type 1a
  • GSD1a
  • Von Gierke disease
  • Glucose metabolism disorder
  • Genetic disorder
  • Autosomal recessive disorder
  • messenger RNA
  • mRNA
  • Pediatric

Additional Relevant MeSH Terms

  • Glycogen Storage Disease