Active not recruitingPHASE3INTERVENTIONAL

Clinical Study for Treatment-naïve IOPD Babies to Evaluate Efficacy and Safety of ERT With Avalglucosidase Alfa

This research study is looking at a new medicine called avalglucosidase alfa for babies under one year old who have recently been diagnosed with Infantile-Onset Pompe Disease (IOPD) and haven't had any previous treatment. It's a Phase 3 study, meaning it's in the later stages of testing before a medicine can be approved for wider use. The researchers want to understand how well the medicine works (its efficacy) and if it's safe. They will also look at how the medicine moves through the body and what effects it has. Babies taking part will receive the medicine and be followed for up to four years, with regular check-ups to monitor their health.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Sanofi

Enrolment target

Start

01 Sep 2021

Estimated completion

10 Aug 2027

Glycogen Storage Disease Type II

What is this study about?

This study is particularly important for babies born with a rare genetic condition called Infantile-Onset Pompe Disease (IOPD). This condition means the body can't properly break down a sugar called glycogen, which then builds up in cells and causes damage, especially to muscles and the heart. Currently, treatments exist, but researchers are always looking for ways to improve them. This study aims to evaluate a new treatment, avalglucosidase alfa, which works by replacing the missing enzyme that helps break down glycogen.

By taking part, babies will receive this new medicine, and doctors will carefully monitor their health over several years. This will help researchers understand if avalglucosidase alfa can effectively reduce the build-up of glycogen and improve the symptoms of IOPD, while also ensuring it's safe for these young patients. The information gathered from this study could lead to a new and improved treatment option for babies with this challenging condition.

It's called a 'Phase 3' study, which is an important stage in developing new medicines. It means the treatment has already shown some promise in earlier tests, and now researchers need to gather more detailed information about its effectiveness and safety in a larger group of patients before it can potentially be made available to everyone who needs it.

Key takeaways

This study is for babies (under 1 year old) newly diagnosed with Infantile-Onset Pompe Disease (IOPD).
It tests a new medicine, avalglucosidase alfa, to see if it's safe and effective.
Babies taking part will receive the new medicine and be closely monitored.
The study lasts up to four years with frequent clinic visits.
Eligibility is strict, including confirmation of IOPD and specific heart conditions.
You can withdraw your baby from the study at any time.

Who may be eligible?

This study is specifically for babies diagnosed with Infantile-Onset Pompe Disease (IOPD) who are under one year old and have not received any treatment for Pompe disease before. To be included, your baby must have a confirmed diagnosis of IOPD, which means specific genetic tests and enzyme tests will confirm the condition. They also need to have heart problems related to IOPD at the time of diagnosis, as confirmed by a heart scan. Additionally, doctors will need to know your baby's 'CRIM status', which is a type of genetic information.

There are also some reasons why a baby might not be able to join the study. For instance, if your baby is already experiencing breathing difficulties and needs help with breathing (like using a ventilator), or has other major health problems not related to Pompe disease, they wouldn't be eligible. Also, if your baby has already received any treatment for Pompe disease, or has participated in a previous study testing this specific medicine, they wouldn't be able to join this particular trial. The study team will review all of your baby's health information to ensure they meet all the criteria.

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 your baby less than 12 months old?
Has your baby just been diagnosed with Infantile-Onset Pompe Disease (IOPD)?
Has your baby *not* previously received any treatment for Pompe disease?
Does your baby's diagnosis include heart problems related to IOPD?
Does your baby *not* currently have breathing problems requiring help (like a ventilator)?
Does your baby *not* have any other major health conditions not linked to Pompe disease?

Answer every question to see your result.

What does participation involve?

If your baby takes part in this study, they will receive the study medicine, avalglucosidase alfa. The study will last for up to four years in total. During this time, you can expect frequent visits to the clinic, initially every other week and potentially every week, so that doctors can closely monitor your baby's health and how they are responding to the treatment. These visits will involve various assessments, including checking your baby’s overall health, taking blood samples, and performing other tests to see how the medicine is working and to monitor for any side effects. There is an initial screening period of up to 4 weeks to determine eligibility. After the first year of treatment, there will be two further periods of extended treatment, each lasting about a year, followed by a final follow-up period.

Potential risks and benefits

Taking part in any clinical study carries potential benefits and potential risks. The potential benefit for your baby is access to a new treatment, avalglucosidase alfa, which may help improve their Pompe disease symptoms. However, as with all medicines, there is a risk of side effects, which the study team will carefully monitor. You will receive detailed information about all known potential risks before deciding whether to participate. It's very important to remember that participation is completely voluntary, and you have the right to withdraw your baby from the study at any time, for any reason, without it affecting their future medical care.

Locations (16)

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

Stanford Hospital- Site Number : 8400006
Verified postcode
Stanford, United States
Children's Hospitals and Clinics of Minnesota- Site Number : 8400008
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Minneapolis, United States
Advanced Medical Genetics- Site Number : 8400002
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Hawthorne, United States
Duke University Medical Center- Site Number : 8400004
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Durham, United States
Cincinnati Children's Hospital Medical Center- Site Number : 8400001
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Cincinnati, United States
Seattle Children's Hospital- Site Number : 8400003
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Seattle, United States
Investigational Site Number : 0560001
Verified postcode
Leuven, Belgium
Investigational Site Number : 1560002
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Qingdao, China
Investigational Site Number : 1560001
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Shanghai, China
Investigational Site Number : 2760001
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Bad Oeynhausen, Germany
Investigational Site Number : 3800002
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Monza, Italy
Investigational Site Number : 5280001
Verified postcode
Rotterdam, Netherlands

Common questions

What is Infantile-Onset Pompe Disease (IOPD)?

IOPD is a rare genetic condition where the body can't break down a sugar called glycogen, leading to its build-up and damage, mainly to muscles and the heart.

What is avalglucosidase alfa?

It's a new medicine being tested that aims to replace a missing enzyme in the body to help break down glycogen.

How long will the study last?

The study will last up to four years, with regular check-ups throughout this period.

Can my baby stop participating at any time?

Yes, you have the right to withdraw your baby from the study at any time, for any reason, without affecting their future medical care.

Will my baby receive other treatments for Pompe disease during the study?

No, this study is for babies who have not received any previous treatment for Pompe disease.