*A Phase I/2 study in participants with Glycogen Storage Disease Type 1a (GSD1a)
Research type
Research Study
Full 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
IRAS ID
1007188
Contact name
Joseph Kupferman
Contact email
Sponsor organisation
Moderna Therapeutics (ModernaTX), Inc.
Clinicaltrials.gov Identifier
Research summary
ModernaTX, Inc. is sponsoring this clinical trial to study an experimental treatment for glycogen storage disease type 1a (GSD1a). People with GSD1a have difficulty converting glycogen (a type of sugar the body can store) into glucose (sugar the body uses for energy). This can lead to low blood sugar. It can also cause glycogen to build up in the liver, leading to liver problems.
The trial will take place in multiple countries, including the UK, and will include people who are at least 6 years old who have GSD1a. The experimental treatment is called mRNA-3745, which is given as an intravenous infusion.
In Part 1 of the trial, researchers will study the effects of a single dose. Different dose levels will be studied in small groups of participants. People who finish Part 1 can join Part 2. In Part 2, researchers will study the effects of repeated dosing (up to 8 doses given once every 2, 3, or 4 weeks). During both parts, participants will be asked to wear a glucose monitoring device, keep track of food they eat in a diary, and visit a trial site regularly. During site visits, doctors will give participants a full check-up and collect blood and urine samples.
Participants who finish Part 2 may be eligible to stay in the trial and continue receiving mRNA-3745. Participants who stop treatment will be asked to stay in contact with their trial site for about a year so doctors can monitor their health.
The time each participant spends in the trial will depend on which part they join and how long they stay in the trial.
The main goal is to learn about the safety/tolerability of mRNA-3745. Researchers will study new or worsening medical problems the participants have during and after treatment, including possible side effects.REC name
London - Riverside Research Ethics Committee
REC reference
23/LO/0905
Date of REC Opinion
15 Mar 2024
REC opinion
Further Information Favourable Opinion