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Moderna mRNA Clinical Trial for Patients with GSD 1a
This study is now recruiting.
Our team has partnered with Moderna on a clinical trial for patients with glycogen storage disease type 1a (GSD1a). We would like to invite you to take part in a clinical trial for an mRNA investigational drug that could potentially correct the cause of GSD1a, by teaching your body to break down glycogen, correct low glucose, and avoid starch intake. The purpose of this trial is to see if a new investigational mRNA study drug, called mRNA-3745, is safe and if it works in the way researchers expect. mRNA technology uses messenger ribonucleic acid (mRNA), an instructional molecule that naturally occurs in the body and carries information to cells. In this case, we are studying if mRNA-3745 given through an IV infusion can instruct the body to make the protein that is missing in people with GSD1a.
mRNA-3725 is not approved by the FDA; the FDA has allowed us to provide this investigational medicine to patients enrolled in this phase 1/2 trial. Phase 1/2 clinical trials like this one aim to test the safety, side effects, and best dose of an investigational medication. In the phase 2 part, participants usually receive the highest dose of treatment discovered in the phase 1 part. This is the first study in which humans are administered this study drug.
If successful, mRNA-3745 would enable certain organs in the body to effectively break down glycogen and avoid low glucose levels and starch intake. If mRNA-3745 could correct the cause of GSD1a, it would help keep your glucose levels where they need to be all the time.
Participation in this clinical trial could last up to approximately two years, depending on which stage you enroll in and whether or not you decide to participate in the long-term follow-up period of the study. Some of the visits in this trial will require you to stay overnight at the hospital, and some of the visits will require you to visit the hospital or doctor's office. Most of the visits in this clinical trial can be done at your home, where a home health nurse will perform the assessments needed.
All study-related procedures, travel, and compensation are provided.
Who Do I Contact?
If you are interested in participating in the study or want to learn more please contact our study team at HIResearch [at] chop.edu or 267-426-9915.
ClinicalTrials.gov Identifier
Eligibility & Criteria
IRB #:
23-021129
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
Study Phase:
Phase I
Phase II
Eligible Age Range:
12 - 80 Years
Gender:
All
Visit Criteria
The study team will inform you which study tasks you will be asked to complete and how frequently they will be done before you consent to participating in the study. Depending on which part of the study you participate in, you may be asked to complete some or all of the following tasks:
- Receive the study drug (once or multiple times)
- Have research inpatient admissions
- Complete fasting tests
- Have a nurse come to your home for remote health visits
- Have a research magnetic resonance imaging (MRI) scan of your liver (which can include contrast)
- Have research urine tests and blood work done (which may include genetic testing solely to confirm your diagnosis of GSD 1a)
- Receive electrocardiograms (ECGs)
- Complete interviews and questionnaires
- Wear a continuous glucose monitor (CGM)
