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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 approximately 6.5 years

Awards & highlights

Study Summary

This trial is testing a new medication to see if it is safe and tolerated by people with a certain medical condition.

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.Check my eligibility

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.See study design

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 approximately 6.5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline through up to approximately 6.5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline through up to approximately 6.5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Number of Participants With Treatment-emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs) and TEAEs Leading to Treatment Discontinuation

Secondary outcome measures

Change From Baseline in Maximum Effect (Emax) During Fasting Challenges

Change From Baseline in Metabolic Biomarkers of GSD1a

Change From Baseline in Time to Hypoglycemia During Fasting Challenges

+5 more

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.

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

ModernaTX, Inc.Lead Sponsor

108 Previous Clinical Trials

61,379,272 Total Patients Enrolled

Media Library

mRNA-3745 (RNA-based Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05095727 — Phase 1 & 2

Glycogen Storage Disease Research Study Groups: SAD: mRNA-3745, MAD: mRNA-3745

Glycogen Storage Disease Clinical Trial 2023: mRNA-3745 Highlights & Side Effects. Trial Name: NCT05095727 — Phase 1 & 2

mRNA-3745 (RNA-based Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05095727 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are there still vacancies within this research endeavor for participants?

"According to the records posted on clinicaltrials.gov, patient recruitment for this medical trial is ongoing and open. This study was made public on June 1st 2022 and underwent its last modification November 21st of the same year."

Answered by AI

How many healthcare facilities are actively conducting this research?

"Currently, 6 clinical sites are recruiting individuals for this trial. These locations include New york, Cincinnati and Houston plus 3 additional places. To reduce the necessary travel, it is beneficial to pick a site which is closest to your residence."

Answered by AI

What risks do patients incur when taking mRNA-3745?

"The safety of mRNA-3745, which is currently the subject of a Phase 1 trial with limited data for both efficacy and safety, has been rated at level one."

Answered by AI

How many participants are actively involved in this research study?

"For the successful completion of this clinical trial, 18 patients must meet its eligibility criteria. The sponsor is ModernaTX Inc., and they are running the study out of two renowned healthcare institutions: Columbia University Medical Center in New york City, and Cincinnati Children's Hospital Medical Center in Ohio."

Answered by AI