Treatment for Glycogen Storage Disease Type 1a using mRNA-3745

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

This study is testing a new treatment called mRNA-3745 to see if it is safe and effective for adults and kids with Glycogen Storage Disease Type 1a.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 45 (estimated)
• Ages: 6 Years and up
• Sex: All
• Sponsor: ModernaTX, Inc. (industry)
• Locations: 16 sites (Farmington, Connecticut and 15 other locations)
• Trial ID: NCT05095727 on ClinicalTrials.gov

What this trial studies

This trial evaluates the safety and tolerability of mRNA-3745 administered intravenously to both adult and pediatric participants diagnosed with Glycogen Storage Disease Type 1a (GSD1a). The study consists of a single ascending dose (SAD) stage followed by a multiple ascending dose (MAD) stage, with an option for participants to continue into an open-label extension period to assess long-term safety and clinical activity. The goal is to determine the effectiveness of this innovative treatment in managing GSD1a.

Who should consider this trial

Good fit: Ideal candidates for this study are individuals with documented GSD1a confirmed by genetic testing and who have not been hospitalized for hypoglycemia in the month prior to screening.

Not a fit: Patients who have undergone solid organ transplants or received prior gene therapy for GSD1a may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of Glycogen Storage Disease Type 1a, potentially reducing complications and improving quality of life for patients.

How similar studies have performed: While this approach using mRNA technology is relatively novel, similar studies in other genetic disorders have shown promising results.

Eligibility criteria

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.

Where this trial is running

Farmington, Connecticut and 15 other locations

• University of Connecticut Health Center — Farmington, Connecticut, United States (RECRUITING)
• Boston Children's Hospital — Boston, Massachusetts, United States (TERMINATED)
• Duke University Medical Center — Durham, North Carolina, United States (RECRUITING)
• Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio, United States (TERMINATED)
• Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (RECRUITING)
• The University of Texas Health Science Center at Houston — Houston, Texas, United States (RECRUITING)
• Baylor College of Medicine — Houston, Texas, United States (RECRUITING)
• University of Utah — Salt Lake City, Utah, United States (RECRUITING)
• Stollery Children's Hospital University of Alberta — Edmonton, Alberta, Canada (RECRUITING)
• The Hospital for Sick Children — Toronto, Ontario, Canada (RECRUITING)
• AP-HP - Hôpital Antoine Béclère — Clamart, France (RECRUITING)
• CHRU Tours - Hopital Clocheville — Tours, France (RECRUITING)
• Universitair Medisch Centrum Groningen — Groningen, Netherlands (RECRUITING)
• Instytut Pomnik Centrum Zdrowia Dziecka — Warsaw, Poland (RECRUITING)
• Hospital Universitario 12 de Octubre — Madrid, Spain (RECRUITING)
• Hospital Regional Universitario de Malaga — Malaga, Spain (RECRUITING)

Study contacts

• Study coordinator: Moderna WeCare Team

Conditions: Glycogen Storage Disease, Glycogen storage disease type 1a, GSD1a, Von Gierke disease, Glucose metabolism disorder, Genetic disorder, Autosomal recessive disorder, messenger RNA