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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
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.
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.
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Who is running the clinical trial?
ModernaTX, Inc.Lead Sponsor
120 Previous Clinical Trials
61,588,164 Total Patients Enrolled
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I have had a low blood sugar event with symptoms.I have had a solid organ transplant.I need constant feeding through a tube in my stomach or nose.My liver tumor grew more than 2 cm or I found more than 5 new tumors in the last 2 years.I have a liver tumor larger than 5 cm.I have been diagnosed with diabetes.I have received gene therapy for GSD1a.My GSD1a diagnosis is confirmed by genetic testing.I haven't been hospitalized for low blood sugar in the last month.
Research Study Groups:
This trial has the following groups:- Group 1: SAD: mRNA-3745
- Group 2: MAD: mRNA-3745
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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