Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Open-label, Phase 3b study to evaluate effectiveness, safety and pharmacokinetic parameters of metreleptin in patients under 6 years of age with generalised lipodystrophy and associated diabetes mellitus and/or hypertriglyceridaemia

This study is testing a medicine called Myalepta in children under 6 years old who have a rare condition called generalised lipodystrophy. This condition can lead to problems like high blood sugar (diabetes) and high levels of fats in the blood (hypertriglyceridaemia). We want to find out how well Myalepta helps manage these issues and if it is safe for very young children. Doctors will monitor important health markers like blood sugar and fat levels over 12 months. This is a Phase 3 study, meaning we are confirming the medicine's effectiveness and safety in a larger group before it can be widely used.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Amryt Pharmaceuticals Designated Activity Company

Enrolment target

Start

03 May 2023

Generalised lipodystrophy

What is this study about?

This study is looking at a medicine called Myalepta. It's for children under 6 years old who have a very rare condition called generalised lipodystrophy. This condition affects how the body uses and stores fat, and it can cause serious health problems like diabetes (when blood sugar is too high) and hypertriglyceridaemia (when there are too many fats in the blood).

We want to see how well Myalepta works in these young children. Specifically, we'll be checking if it helps bring down high blood sugar and fat levels over a year. We'll also carefully watch for any side effects to make sure the medicine is safe to use. This kind of study helps doctors understand more about how new treatments work and if they are suitable for very young patients.

The main things we'll be measuring are changes in a blood test called HbA1c, which shows average blood sugar levels over time, and fasting triglyceride levels, which measure fats in the blood. We'll also look at other important health markers, like liver health, as these can also be affected by generalised lipodystrophy.

Key takeaways

This study is for children under 6 with generalised lipodystrophy.
It aims to see if Myalepta helps control blood sugar and fat levels.
The study will also check the safety of Myalepta in young children.
Participation involves regular clinic visits and injections of the medicine.
The study lasts for 12 months, with possible follow-up.

Who may be eligible?

This study is specifically designed for children under 6 years old who have been diagnosed with generalised lipodystrophy. Both boys and girls can take part.

To be considered, your child would also need to have certain levels of blood sugar or fats in their blood. For example, if their diabetes isn't well controlled (shown by a high HbA1c test) or if they have high levels of fats called triglycerides.

The study team will review all your child's medical information carefully to see if they are a good fit for this research. They will explain everything clearly to you and answer any questions you have.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is my child under 6 years old?
Does my child have a diagnosis of generalised lipodystrophy?
Does my child have high blood sugar (diabetes) or high fat levels (triglycerides)?
Am I able to bring my child to regular clinic visits?

Answer every question to see your result.

What does participation involve?

If your child takes part, they will receive the study medicine, Myalepta, as an injection. The medicine comes in two different strengths. You'll have regular visits to the clinic over 12 months, where doctors and nurses will check your child's health. These visits will involve blood tests to measure sugar and fat levels, and may include other checks like ultrasounds to look at the liver. The team will also monitor your child closely for any side effects. After the 12-month treatment period, there may be follow-up appointments to see how your child is doing.

Potential risks and benefits

Taking part in a study like this might offer some potential benefits, as your child would receive regular medical care and a new medicine that could help manage their condition. However, there's no guarantee the medicine will work, and it might have side effects, which the study team will explain in detail. You are free to withdraw your child from the study at any time, for any reason, without it affecting their usual medical care.

Locations (4)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Italy
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Unverified
Germany
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Unverified
Belgium
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Unverified
France

Common questions

What is generalised lipodystrophy?

It's a rare condition where the body has trouble storing fat, which can lead to other health issues like high blood sugar and fats in the blood.

What is Myalepta?

Myalepta is a medicine being tested in this study to help manage the problems caused by generalised lipodystrophy, like high blood sugar and fat levels.

Will my child need injections?

Yes, if your child participates, they will receive the study medicine, Myalepta, as an injection.

How long does the study last?

The main part of the study involves receiving the medicine and having regular check-ups for 12 months.

What are HbA1c and triglycerides?

HbA1c is a blood test showing average blood sugar levels over a few months. Triglycerides are a type of fat found in the blood.