Active not recruitingPHASE3INTERVENTIONAL

RZ358 Treatment for Congenital Hyperinsulinism

This research study is looking at a new medicine called RZ358 for people with congenital hyperinsulinism (CHI), a condition where the body makes too much insulin, leading to dangerously low blood sugar. Currently, available treatments don't always work well or can have unwanted side effects. The study aims to find out if RZ358, when added to current treatments, can better control blood sugar levels and if it is safe. Participants, aged 3 months to 45 years, will be randomly given either RZ358 or a placebo (a dummy medicine) alongside their usual care for 24 weeks. After this, they might continue into a longer-term study to look at the medicine's effects over time. This is a 'Phase 3' study, meaning it's a key step in testing this potential new treatment.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Rezolute

Enrolment target

Start

11 Jan 2024

Estimated completion

14 Nov 2027

Congenital Hyperinsulinism

What is this study about?

Congenital hyperinsulinism (often called CHI) is a rare condition that mainly affects babies and young children, though it can continue into adulthood. It happens when the body produces too much insulin, which causes blood sugar levels to drop too low. This can be very serious and lead to various health problems if not managed well. While there are some treatments available, they don't always work perfectly for everyone or can come with their own side effects, meaning there's a real need for better options.

This study is designed to test a new medicine named RZ358. The main goal is to see if RZ358 can help control blood sugar better and be a safer treatment option for people with CHI. It's a 'Phase 3' study, which means it's one of the final steps before a new medicine might be considered for wider use. Researchers want to compare RZ358 to the usual treatments that patients already receive to see if combining them gives a better outcome.

Participants will be placed into different groups by chance (like flipping a coin) to receive either RZ358 or a dummy medicine (called a placebo) in addition to their current treatments. This helps the researchers understand if any improvements are truly due to RZ358. The study will last for at least 24 weeks, followed by an optional longer period where everyone receives RZ358, to gather more information on its long-term effects.

Key takeaways

This study is for people with congenital overproduction of insulin (CHI).
It's testing a new medicine, RZ358, to help manage low blood sugar.
RZ358 will be compared to a dummy medicine alongside usual care.
Participation lasts at least 24 weeks, with optional longer follow-up.
The goal is to find a better, safer treatment for CHI.

Who may be eligible?

To be able to join this study, people generally need to be between 3 months and 45 years old and have a confirmed diagnosis of congenital hyperinsulinism.

Importantly, the study is for those whose current treatments haven't fully brought their blood sugar under control, meaning they still have at least three episodes of low blood sugar per week despite trying standard medical care. Your doctor will need to agree that you've given your current treatments a good try.

There are also some reasons why someone might not be able to join. For example, if you have certain liver problems, are very overweight (for adults) or have diabetes. Also, if you're allergic to RZ358 or any of its ingredients, you wouldn't be able to take part.

Quick self-check

Are you between 3 months and 45 years old?
Do you have a clear diagnosis of congenital hyperinsulinism?
Do you still experience low blood sugar (at least 3 times a week) even with your current treatments?
Are your liver tests generally within a normal range?
Do you NOT have diabetes or pre-diabetes?
Are you NOT allergic to RZ358?

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

What does participation involve?

If you decide to take part in this study, you would first go through a screening process to make sure you're suitable. Once accepted, you would be randomly assigned to one of several groups. Some groups will receive RZ358 in different doses, while others will receive a placebo (a dummy medicine), all in addition to your usual care.

Throughout the main part of the study, which lasts 24 weeks, you'll have regular visits to the clinic. At these visits, doctors and nurses will check your health, take blood and other samples, and monitor your blood sugar levels. You'll also likely need to keep track of your blood sugar at home. The study medication or placebo will be given to you to take as instructed. After the 24 weeks, you might have the option to join a longer-term part of the study, where everyone receives RZ358, to further monitor its safety and effects. The total duration of your involvement would depend on whether you participate in this extension phase.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit of joining this study is that RZ358 might improve your blood sugar control compared to your current treatment alone, which could improve your quality of life. However, like all medicines, RZ358 might cause side effects, some of which are not yet fully known. The research team will carefully monitor you for any side effects throughout the study. Remember, participating is entirely your choice, and you are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (18)

Children's Hospital of Philadelphia
Philadelphia, United States
Cook Children's Medical Center
Fort Worth, United States
Rezolute Investigative Site, Varna, Bulgaria
Varna, Bulgaria
Rezolute Investigative Site, Odense, Denmark
Odense, Denmark
Rezolute Investigative Site, Bron, France
Bron, France
Rezolute Investigative Site, Paris, France
Paris, France
Rezolute Investigative Site, Tbilisi, Georgia
Tbilisi, Georgia
Rezolute Investigative Site, Berlin, Germany
Berlin, Germany
Rezolute Investigative Site, Dusseldorf, Germany
Düsseldorf, Germany
Rezolute Investigative Site, Athens, Greece
Athens, Greece
Rezolute Investigative Site, Seeb, Oman
Seeb, Oman
Rezolute Investigative Site, Al Rayyan, Qatar
Al Rayyan, Qatar

+6 more sites — see the official record for the full list.

Common questions

What is congenital hyperinsulinism (CHI)?

It's a condition where the body makes too much insulin, which causes blood sugar levels to drop dangerously low. It's often found in babies and young children.

What is RZ358?

RZ358 is a new medicine being tested in this study to see if it can help control low blood sugar in people with CHI.

What does 'Phase 3 study' mean?

Phase 3 is a stage in medicine testing where the drug is given to many people to see how well it works and if it's safe, before it can be approved for general use.

Will I definitely get the new medicine if I join?

No, you might receive RZ358 or a placebo (a dummy medicine) along with your usual care during the main part of the study. This is decided by chance.

How long will the study last for me?

The main treatment period is 24 weeks. After that, you might have the option to continue in a longer-term part of the study, where everyone receives RZ358.