Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

An Open-label, Multinational, Multicenter, Intravenous Infusion Study of the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Avalglucosidase Alfa in Treatment-naïve Pediatric Participants with Infantile-Onset Pompe Disease (IOPD)

This important study, called a Phase III trial, is looking at a new medicine called Nexviadyme for very young children with a rare genetic condition called infantile-onset Pompe disease. This condition can severely affect muscles, including those needed for breathing and eating. The researchers want to find out if Nexviadyme is safe and effective in these children who haven't received other treatments. They will be checking if the children can breathe on their own without a breathing machine and if they're growing and developing well after about a year. The study will also carefully monitor any side effects and how the medicine works in the body. It's a key step in understanding if Nexviadyme could be a helpful new treatment option.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Sanofi-Aventis Recherche & Developpement

Enrolment target

Start

09 Jul 2024

Glycogen storage disease type II

What is this study about?

This study is looking into a new medicine called Nexviadyme for a very rare and serious genetic condition known as Pompe disease. Specifically, it's focusing on babies and young children who have the 'infantile-onset' type, which means the symptoms appear shortly after birth. This form of Pompe disease is particularly severe because the body can't properly break down a sugar called glycogen, which then builds up and damages muscles, including the heart and the muscles used for breathing. This can lead to serious problems with breathing and overall development.

The main goal of this particular study is to see if Nexviadyme helps these young patients, especially those who haven't had any other treatments for Pompe disease before. The researchers want to know if the medicine can help them stay alive and breathe on their own, without needing a breathing machine, after about a year of treatment. They will also be looking at other important things like how well the children are growing, how their hearts are doing, and their general muscle strength and development. Safety is also a major focus, so they will carefully watch for any side effects.

This study is a 'Phase III' trial, which means it’s one of the final steps before a new medicine might be approved for wider use. It's designed to confirm how well the medicine works and if it's safe enough. The hope is that Nexviadyme could offer a new and effective treatment option for very young children suffering from this challenging condition.

Key takeaways

This study is testing a new medicine (Nexviadyme) for babies with severe Pompe disease.
It's for children who haven't had other treatments for Pompe disease before.
The main aim is to see if the medicine helps children breathe on their own and grow well.
The medicine is given through a drip (infusion) at regular intervals.
Safety is a big focus; children will be closely monitored for any side effects.
The study will last about a year for each child.

Who may be eligible?

This study is looking for babies and very young children who have been diagnosed with infantile-onset Pompe disease. It's specifically for those who have not received any treatment for their Pompe disease before. This ensures the researchers can clearly see how well Nexviadyme works on its own.

While the exact age isn't strictly defined as 'any to any' in the raw information, because it focuses on 'infantile-onset' and 'pediatric participants,' it means the study is for young children, likely babies and toddlers.

Both boys and girls can take part in this study. The medical team involved in the study will review each child's specific health information to confirm if they meet all the requirements.

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.

Does your child have infantile-onset Pompe disease?
Has your child *not* received any previous treatment for Pompe disease?
Is your child a baby or young child?
Are you able to attend regular hospital visits for infusions and check-ups?

Answer every question to see your result.

What does participation involve?

If your child takes part in this study, they will receive the study medicine, Nexviadyme, through an intravenous infusion. This means the medicine will be given directly into their bloodstream using a drip. These infusions will happen regularly over a period of time.

Throughout the study, your child will have regular visits to the hospital or clinic. During these visits, the study team will carry out various checks and assessments. This includes measurements of their breathing, heart function, growth (such as length, weight, and head circumference), and muscle development. They will also take blood and urine samples to check how the medicine is working and to monitor for any changes in their health.

Before the study begins, your child will have a full check-up, and they’ll continue to be monitored for about a year (52 weeks) to see how they respond to the treatment. The research team will explain the full schedule of visits and assessments to you in detail.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. Your child might benefit if Nexviadyme helps improve their breathing, heart function, or overall development. However, there's no guarantee the medicine will work for every child, and your child might experience side effects, such as reactions during the infusion or other common health changes. The research team will closely monitor your child for any unexpected effects. You always have the right to withdraw your child from the study at any time, for any reason, without it affecting their ongoing medical care.

Locations (5)

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

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Unverified
Spain
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Unverified
Netherlands
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Unverified
Germany
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Unverified
Belgium
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Unverified
Italy

Common questions

What is infantile-onset Pompe disease?

It's a serious rare genetic condition affecting babies, where the body can't properly break down a sugar, causing muscle damage, especially to the heart and breathing muscles.

What is Nexviadyme?

Nexviadyme is the new medicine being tested in this study. It's designed to help replace the missing enzyme that causes Pompe disease.

How will the medicine be given?

The medicine will be given directly into your child's bloodstream through a drip, which is called an intravenous infusion.

What are the main things the doctors are looking for?

They mainly want to see if children can breathe on their own and stay alive after about a year, as well as checking their growth and overall development.

How long will the study last for my child?

Your child will be monitored for about a year (52 weeks) during the main part of the study, with regular check-ups and assessments.