What side effects are associated with this type of intervention?

Common side effects of mRNA therapies, such as mRNA-3745 for Glycogen Storage Disease Type 1a (GSD1a), include injection site reactions, flu-like symptoms (fever, chills, fatigue), and potential immune responses. Traditional treatments for GSD1a, like dietary management and cornstarch therapy, can cause gastrointestinal discomfort and hypoglycemia if not properly managed.

What prior FDA approvals exist?

As of now, there are no FDA-approved mRNA therapies specifically for the treatment of Glycogen Storage Disease Type 1a (GSD1a). Traditional treatments for GSD1a have focused on dietary management to maintain normal blood glucose levels. The trial of mRNA-3745 represents a novel approach, aiming to correct the underlying genetic defect. Other genetic therapies for metabolic disorders are still largely in experimental stages, with ongoing research and clinical trials exploring their potential.

What other types of research exist?

As of 2024, promising alternatives to mRNA-3745 for GSD1a include gene therapy approaches that aim to correct the genetic defect at its source. These may involve adeno-associated virus (AAV) vectors to deliver functional copies of the G6PC gene. Additionally, enzyme replacement therapies that provide the deficient enzyme glucose-6-phosphatase or small molecule therapies that enhance residual enzyme activity or reduce glycogen accumulation are also being explored. Patients should discuss these options with their healthcare provider to determine the most appropriate treatment based on the latest clinical trial data and individual health status.

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RNA-based Therapy

mRNA-3745 for Glycogen Storage Disease

Phase 1 & 2

Recruiting

Research Sponsored by ModernaTX, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline through up to week 32

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new treatment called mRNA-3745, which uses messenger RNA to help the body produce a missing protein. It is aimed at adults and children with Glycogen Storage Disease Type Ia (GSD1a). The study will check if the treatment is safe and how well it works.

Who is the study for?

This trial is for adults and kids with Glycogen Storage Disease Type 1a (GSD1a). They must have had a low blood sugar event recently and confirmed GSD1a by genetic testing. It's not for those who've had liver transplants, gene therapy for GSD1a, large liver tumors, diabetes, or severe allergies to MRI contrast unless an alternative imaging method is available.

What is being tested?

The study tests mRNA-3745 given through the veins to see if it's safe and tolerable for people with GSD1a. The focus is on how participants react to this new treatment over time.

What are the potential side effects?

Since the trial aims to assess safety, potential side effects are being studied but may include reactions at the infusion site, general discomforts like headaches or nausea, allergic reactions, or other unforeseen issues related to mRNA-3745.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline through up to week 32

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline through up to week 32

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through up to week 32 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Number of Participants Not Experiencing Hypoglycemia During Fasting Challenges

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups

Experimental Treatment

Group I: SAD: mRNA-3745Experimental Treatment1 Intervention

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.

Group II: MAD: mRNA-3745Experimental Treatment1 Intervention

Participants will receive multiple IV doses of mRNA-3745 in an inpatient setting. Participants will have the option to continue treatment in the OLE.

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

The most common treatments for Glycogen Storage Disease (GSD) aim to manage symptoms and prevent complications by maintaining normal blood glucose levels. For GSD1a, mRNA-3745 therapy works by delivering synthetic mRNA into the body, which instructs cells to produce the functional enzyme glucose-6-phosphatase that is deficient in these patients. This approach directly addresses the underlying genetic defect, potentially offering a more effective and long-term solution compared to traditional treatments like dietary management and glucose supplementation. By correcting the enzyme deficiency, mRNA therapy can help prevent the accumulation of glycogen in the liver and kidneys, reducing the risk of severe hypoglycemia and other metabolic complications, thereby improving the quality of life for GSD patients.