Ongoing, recruitingTherapeutic confirmatory (Phase III)Interventional

Open Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Pegunigalsidase Alpha (PRX-102) 2 mg/kg Administered by Intravenous Infusion Every 4 Weeks in Patients with Fabry Disease

This research is an 'open label extension' study for adults with Fabry disease, a genetic condition that causes various health problems. It's looking at the long-term safety and effectiveness of a drug called pegunigalsidase alpha (also known as PRX-102), given as an IV drip every four weeks. Because it's 'open label,' everyone in the study will receive the active drug, and both patients and doctors will know what treatment is being given. Researchers will closely monitor participants' health, including blood tests, physical exams, and heart checks, to understand any side effects. They'll also measure how well the drug helps with symptoms and kidney/heart function. This is a Phase 3 study, meaning it's a final step before potentially becoming widely available.

At a glance

Status

Ongoing, recruiting

Phase

Therapeutic confirmatory (Phase III)

Sponsor

Chiesi Farmaceutici S.p.A.

Enrolment target

Start

13 Sep 2024

Fabry disease (α-galactosidase A deficiency)

What is this study about?

Fabry disease is a rare condition that's passed down through families. It happens because the body is missing an important enzyme, which is a type of protein that helps break down a fatty substance. Without this enzyme, the fatty substance builds up in different parts of the body, leading to problems with organs like the kidneys, heart, and brain, and can cause pain. This study is testing a drug called pegunigalsidase alpha, which aims to replace the missing enzyme, helping the body clear out the harmful fatty substance.

This particular study is looking at how well the drug works and how safe it is over a longer period. It's an 'open label' study, which means everyone involved, including the patients and the doctors, will know that you are receiving the active drug. The drug is given directly into a vein through a drip every month. The main goal is to understand if this treatment is a good long-term option for managing Fabry disease.

Researchers will be carefully checking many aspects of your health throughout the study. This includes regular blood tests, checking your heart, and seeing how well your kidneys are working. They will also pay close attention to any possible side effects and how your overall quality of life is affected. This information is crucial for determining if this drug can make a real difference for people living with Fabry disease.

Key takeaways

It's a long-term study for adults with Fabry disease.
The study drug, pegunigalsidase alpha, is given by IV drip every 4 weeks.
It aims to check the safety and effectiveness of the drug over time.
Researchers will monitor heart, kidney function, and overall health.
Both men and women aged 18 and over can participate.

Who may be eligible?

This study is designed for adults aged 18 and over, with no upper age limit. Both men and women are welcome to participate. To be considered, you must have been diagnosed with Fabry disease.

Quick self-check

Are you 18 years old or older?
Do you have a confirmed diagnosis of Fabry disease?
Are you able to attend regular clinic visits for IV infusions?
Are you willing to have ongoing health checks, including blood tests and scans?

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

What does participation involve?

Taking part in this study involves receiving the study drug, pegunigalsidase alpha (PRX-102), through an IV drip once every four weeks. Each infusion lasts a certain amount of time, and you'll need to attend a clinic for each session. You will have regular check-ups, which include blood tests, physical exams, and possibly heart scans (like an echocardiogram) and brain scans (MRI).

Researchers will also ask you to fill out questionnaires about your pain levels and how your quality of life is. They'll monitor how your body reacts to the infusions and if you need any other medications to help prevent reactions. The study doesn't specify an exact total duration but implies a long-term commitment, with regular follow-up visits after each infusion.

Potential risks and benefits

Taking part in any study has potential benefits and risks. You might benefit from receiving a new treatment for your Fabry disease that could help manage your symptoms and improve your health. However, there's always a chance of side effects or allergic reactions to the study drug, or that the treatment might not work for you. All medical procedures carry some risk, such as discomfort from blood draws. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (5)

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Czechia
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Italy
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Norway
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Belgium
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Denmark

Common questions

What is Fabry disease?

Fabry disease is a rare genetic condition where a fat-like substance builds up in the body, which can harm organs like the heart, kidneys, and brain.

What is pegunigalsidase alpha?

It's a new drug being tested that aims to replace the missing enzyme in people with Fabry disease, helping their bodies clear away harmful substances.

What does 'open label' mean?

It means everyone in the study, both patients and doctors, knows that you are receiving the active study drug.

How is the drug given?

The drug is given as a drip directly into a vein (IV infusion) regularly, every four weeks.

What is a Phase 3 study?

A Phase 3 study is one of the final steps before a new drug can be approved and made widely available. It involves a larger number of people to confirm safety and effectiveness.