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mRNA-3745 for Glycogen Storage Disease

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
Baylor College of Medicine, Houston, TXGlycogen Storage DiseasemRNA-3745 - Drug
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

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

Eligible Conditions
  • Glycogen Storage Disease

Treatment Effectiveness

Effectiveness Progress

1 of 3

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Study Objectives

1 Primary · 15 Secondary · Reporting Duration: Pre-infusion, at mid-infusion, and at EOI on Day 1 up to Week 52

Baseline through Week 52
Change From Baseline in Maximum Effect (Emax) During Fasting Challenges
Change From Baseline in Metabolic Biomarkers
Change From Baseline in Time to Hypoglycemia During Fasting Challenges
Change From Baseline of Area Under the Effect Curve (AUEC) of Blood Glucose and Lactate During Fasting Challenges
Number of Participants With No Hypoglycemic Events for up to 12 Hours During Fasting Challenges
Number of Participants With No Hypoglycemic Events for up to 8 Hours During Fasting Challenges
Week 52
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Week 52
Area Under the Serum Concentration-Time Curve From Time 0 to the Time of the Last Measurable Serum Concentration (AUC0-t) of mRNA and LNP
Clearance (CL) of mRNA and LNP
RNA, Messenger
Time to Cmax of mRNA and LNP
Time to Reach Cmax (Tmax) of mRNA and LNP
Volume of Distribution at Steady State (Vss) of mRNA and LNP
Volume of Distribution at Steady State of mRNA and LNP
Week 52
RNA, Messenger
Maximum Observed Serum Concentration (Cmax) of mRNA and LNP

Trial Safety

Safety Progress

1 of 3

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Trial Design

1 Treatment Group

mRNA-3745
1 of 1

Experimental Treatment

18 Total Participants · 1 Treatment Group

Primary Treatment: mRNA-3745 · No Placebo Group · Phase 1

mRNA-3745
Drug
Experimental Group · 1 Intervention: mRNA-3745 · Intervention Types: Drug

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: pre-infusion, at mid-infusion, and at eoi on day 1 up to week 52

Who is running the clinical trial?

ModernaTX, Inc.Lead Sponsor
72 Previous Clinical Trials
50,291,002 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 2 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You have a history of blood sugar dropping dangerously low, causing symptoms of low blood sugar, without any current illness. This has happened at least once in the 4 weeks before you signed up for the study.
You have been diagnosed with GSD1a, which has been confirmed by a genetic test.

Who else is applying?

What state do they live in?
Minnesota100.0%
How old are they?
< 18100.0%
What site did they apply to?
Mayo Clinic100.0%
What portion of applicants met pre-screening criteria?
Met criteria100.0%
References

Frequently Asked Questions

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer

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." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

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