Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

An Extension Trial Evaluating the Long-term Safety and Efficacy of Dasiglucagon for the Treatment of Children with Congenital Hyperinsulinism

This study is for children who have a rare condition called congenital hyperinsulinism. This condition means their body makes too much insulin, leading to very low blood sugar. Researchers are testing a medicine called dasiglucagon to see if it is safe and works well over a long period to help keep blood sugar levels stable. They will be carefully watching for any side effects. They will also look at how much sugar children need through tubes, how long they use these tubes, and if they need an operation. The study also uses continuous glucose monitors (CGM) to track blood sugar more closely and see how often low blood sugar happens.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Zealand Pharma A/S

Enrolment target

Start

09 Aug 2024

Children with congenital hyperinsulinism

What is this study about?

This study is an important follow-up to earlier research and is focusing on children who have a rare condition called congenital hyperinsulinism (CHI). Children with CHI produce too much insulin, which can cause their blood sugar to drop dangerously low. This can be very serious and lead to problems if not managed well.

The study is looking at a medicine called dasiglucagon. This medicine aims to help control blood sugar levels in these children. The main point of this study is to understand the long-term safety of dasiglucagon, meaning they want to know if it causes any harmful effects when used for a longer time. They will carefully record all side effects, no matter how small.

Beyond safety, the study also wants to see how well dasiglucagon works. Researchers will be looking at several things, such as how much sugar children need through feeding tubes to prevent low blood sugar, and if they can stop using these tubes. They will also check if the medicine might reduce the need for surgery on the pancreas. They'll also use special monitors (CGM) to track blood sugar levels continuously, helping them understand how stable blood sugar is and how often low blood sugar episodes happen.

Key takeaways

This study is for children with congenital hyperinsulinism.
It tests a medicine called dasiglucagon for long-term safety and effectiveness.
Researchers will monitor blood sugar levels and track any side effects.
It aims to see if dasiglucagon can reduce the need for tube feeding or surgery.
Participation involves taking the medicine and regular check-ups.

Who may be eligible?

This study is open to children who have been diagnosed with congenital hyperinsulinism, regardless of their age. Both boys and girls can take part.

Because this is an extension study, it likely means that the children taking part have already been in an earlier study for dasiglucagon. This helps researchers continue to gather information on the medicine over a longer period.

If you're interested, your child would need to meet other specific health criteria, which the study team would discuss with you in detail.

Quick self-check

Does your child have congenital hyperinsulinism?
Has your child previously taken part in a dasiglucagon study?
Is your child any age?
Is your child a boy or a girl?

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

What does participation involve?

If your child joins this study, they will take the medication called dasiglucagon 4 mg/ml. The study team will regularly check your child's health and look for any changes or side effects. They will also track their blood sugar levels, likely using a continuous glucose monitor (CGM). This involves wearing a small sensor that checks blood sugar throughout the day and night. You'll also need to keep track of how much sugar your child gets through feeding tubes, if they use them. The study will involve regular visits to the clinic for assessments and check-ups. The total duration of participation isn't specified here, but it's designed to look at long-term effects.

Potential risks and benefits

Participating in this study might offer your child the potential benefit of receiving a medicine that could help manage their congenital hyperinsulinism more effectively, potentially reducing severe low blood sugar episodes. However, like all medicines, dasiglucagon may have side effects, and the research team will carefully monitor for these. There's also the chance the medicine may not work for your child. You are free to withdraw your child from the study at any time, for any reason, without it affecting their usual medical care.

Locations (1)

—
Germany

Common questions

What is congenital hyperinsulinism?

It's a rare condition where a child's body makes too much insulin, which can cause very low and dangerous blood sugar levels.

What is dasiglucagon?

It's a medicine being tested in this study to help control blood sugar levels in children with congenital hyperinsulinism.

Why is this study important?

It helps researchers understand if dasiglucagon is safe and effective when used for a long time to manage this condition in children.

Will my child have to have surgery if they join?

The study aims to see if the medicine can reduce the need for pancreatic surgery, but it doesn't mean surgery is definitely involved in the study itself.

What does 'long-term safety' mean?

It means the study will carefully watch for any side effects or health problems that might happen when the medicine is used over many months or years.