Ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humansInterventional

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

This research study is investigating a new treatment called mRNA-3745 for people with Glycogen Storage Disease Type 1a (GSD1a). This condition affects how the body stores and releases sugar, leading to low blood sugar. The trial is divided into two main parts. First, it will carefully check the safety of mRNA-3745 and how well people tolerate it, starting with small amounts and slowly increasing the dose. Researchers will also look at how the medicine moves through the body and what effects it has. The study will then continue to explore these aspects in more detail. The main goal is to understand if this new medicine is safe and to see its potential to help manage blood sugar levels and other related health markers in GSD1a.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- First administration to humans

Sponsor

Moderna Therapeutics Inc.

Enrolment target

Start

27 May 2024

Glycogen storage disease type 1a (GSD1a)

What is this study about?

This study is looking into a new experimental medicine, mRNA-3745, for people who have a condition called Glycogen Storage Disease Type 1a (GSD1a). GSD1a is a rare inherited condition that makes it hard for the body to properly store and release glucose, which is a type of sugar the body uses for energy. This can lead to dangerously low blood sugar levels, especially when someone hasn't eaten for a while. Currently, managing GSD1a often involves frequent feeds and special diets to keep blood sugar stable.

The main purpose of this study is to find out if mRNA-3745 is safe and how well people can tolerate it. This is one of the very first times this medicine will be given to people, so researchers will start with very small doses and gradually increase them while keeping a close eye on participants. They will also be learning how the medicine works inside the body and what effects it has on various body systems.

Ultimately, researchers hope this new medicine could offer a different way to help manage GSD1a by improving the body's ability to keep blood sugar levels steady. They will be looking at important indicators like whether the medicine can help people go for longer periods without their blood sugar dropping too low, and how it affects other health markers related to the condition.

Key takeaways

This study is testing a new medicine for Glycogen Storage Disease Type 1a (GSD1a).
It's a first-in-human study, focusing on safety and how the medicine works.
Participants will receive the new medicine and other common medications.
Close medical monitoring, including blood tests and fasting challenges, will be involved.
The study aims to improve understanding of GSD1a treatment.
Participation is voluntary, and you can withdraw at any time.

Who may be eligible?

To be considered for this study, you need to be an adult, 18 years of age or older. Both men and women are welcome to take part.

There might be other specific health requirements or conditions that would determine if this study is suitable for you. These are put in place to ensure your safety and that the study can gather accurate information about the new medicine.

It's important to discuss your full medical history with the study team to see if you meet all the criteria.

Quick self-check

Are you 18 years old or older?
Do you have a diagnosis of Glycogen Storage Disease Type 1a (GSD1a)?
Are you able to commit to regular clinic visits and monitoring?
Are you willing to undergo fasting challenges as part of the study requirements?
Are you willing to follow specific study procedures and take study medications?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to join this study, you will be given the experimental medicine, mRNA-3745, as well as some common medicines like paracetamol or antihistamines (such as fexofenadine, cetirizine, diphenhydramine, hydroxyzine). Other treatments like dexamethasone (an anti-inflammatory), famotidine (for stomach acid), ibuprofen (for pain/inflammation), and ondansetron (for nausea) may also be used. You will also receive careful supervision with carbohydrates and intravenous fluids.

Your participation will involve regular visits to the study clinic. During these visits, the study team will closely monitor your health. This will include checking your vital signs (like blood pressure and heart rate), taking electrocardiograms (ECGs) to check your heart, and carrying out blood and urine tests. These tests will help them understand how your body is reacting to the medicine, both in terms of safety and how it might be affecting your GSD1a. You will also undergo 'fasting challenges' where researchers will observe your blood sugar levels for up to 12 hours without food to see how the medicine impacts your body's ability to manage sugar. The total duration of your participation will be explained by the study team.

Potential risks and benefits

Taking part in a study like this might offer potential benefits, such as contributing valuable knowledge to finding new treatments for GSD1a. There's also a chance you could directly benefit from the experimental medicine, though this is not guaranteed as it's still being tested. On the other hand, there are potential risks, including side effects from the experimental medicine or other medications given, or discomfort from procedures like blood tests. The study team will carefully explain all known and potential risks. It's important to remember that you have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (4)

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Spain
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Poland
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Netherlands
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France

Common questions

What is Glycogen Storage Disease Type 1a (GSD1a)?

GSD1a is a rare genetic condition where the body has trouble breaking down glycogen (stored sugar) to maintain normal blood sugar levels. This can lead to low blood sugar.

What is mRNA-3745?

mRNA-3745 is a new, investigational medicine specifically designed to help the body better manage sugar levels in people with GSD1a. It's still in the early stages of testing.

Why are different doses being used?

Researchers start with very low doses to ensure safety and then gradually increase them. This helps them find the safest and most effective amount of the medicine.

Will I have to fast during the study?

Yes, the study involves 'fasting challenges' where your blood sugar and other health markers will be monitored for a period without food to see how the medicine works.

What is the purpose of Phase 1 / Phase 2 studies?

Phase 1 studies primarily focus on safety and how the medicine acts in the body, while Phase 2 studies begin to look at how well the medicine works for the condition, always keeping safety as a top priority. This study combines both.