Study on the Long-Term Safety and Effectiveness of Pegunigalsidase Alfa for Adults with Fabry Disease

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What is this study about?

This clinical trial is focused on studying Fabry disease, a rare genetic condition caused by a deficiency of an enzyme called alpha-galactosidase A. The study is evaluating a treatment called pegunigalsidase alfa, also known by its code name PRX-102. This treatment is administered as an intravenous infusion, which means it is given directly into a vein, every four weeks. The main goal of the study is to assess the long-term safety and effectiveness of this treatment in patients with Fabry disease.

Participants in the study will receive the treatment regularly over a period of time, and their health will be monitored to observe any changes. This includes checking various health indicators such as kidney function, heart health, and levels of certain substances in the blood. The study will also look at how the treatment affects the participants’ quality of life and their ability to perform physical activities. Additionally, the study will monitor for any side effects or reactions to the treatment.

The study is designed to provide valuable information about the potential benefits and risks of using pegunigalsidase alfa for treating Fabry disease. By participating in this study, researchers hope to gather data that could lead to improved treatment options for individuals living with this condition. The study is expected to continue until 2025, allowing for a comprehensive evaluation of the treatment’s long-term impact.

1 joining the study

Participation begins after completing a previous study, PB-102-F50.

Informed consent is required to participate.

If applicable, use of effective contraception is necessary for two weeks after the treatment ends.

2 treatment administration

The medication used is pegunigalsidase alfa, administered as a concentrate for solution for infusion.

The infusion is given intravenously every four weeks.

The dosage is 2 mg per kg of body weight.

3 safety assessments

Regular monitoring includes clinical laboratory tests and physical examinations.

The injection site is assessed for any reactions.

Electrocardiography and brain MRI are conducted to monitor health status.

Any adverse events related to the treatment are recorded.

The ability to reduce or stop pre-medication for infusion reactions is evaluated.

4 efficacy assessments

The study measures kidney function using the estimated glomerular filtration rate (eGFRCKD-EPI).

Heart health is assessed by measuring the left ventricular mass index through an echocardiogram.

Blood tests measure plasma Lyso-Gb3 and Gb3 concentrations.

Urine tests check the protein/creatinine ratio.

The frequency of pain medication use is monitored.

Exercise tolerance is evaluated through a stress test.

Pain levels are assessed using the Short Form Brief Pain Inventory.

The Mainz Severity Score Index is used to evaluate disease severity.

Quality of life is measured using the EQ-5D-5L questionnaire.

5 study duration

The study is expected to continue until December 1, 2025.

Who Can Join the Study?

The patient must have completed a previous study called PB-102-F50.
The patient must sign a document called an informed consent, which means they agree to participate in the study after understanding what it involves.
Female patients and male patients whose partners can become pregnant must agree to use a reliable method of birth control. This includes methods like hormonal birth control (pills, patches, or rings) combined with a barrier method like a condom, or using devices like an intrauterine device (IUD). Other options include having a partner who has had a vasectomy or choosing not to have sex. Birth control should be used for 2 weeks after stopping the treatment.

Who Cannot Join the Study?

Patients who are not adults cannot participate. This means only individuals who are 18 years or older can join.
Patients who do not have Fabry disease cannot participate. Fabry disease is a genetic condition caused by a lack of a specific enzyme called α-galactosidase A.
Patients who are unable to follow the study procedures or attend the required visits cannot participate.
Patients who have any other medical condition that might interfere with the study cannot participate. This means if you have another health issue that could affect the study results, you may not be eligible.
Patients who are pregnant or breastfeeding cannot participate. This is to ensure the safety of both the mother and the baby.
Patients who are currently participating in another clinical trial cannot participate. This is to avoid any interference with the study results.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Azienda Ospedaliera Universitaria Federico II Di Napoli	Naples	Italy

Other Sites

Site Name	City	Country
Vseobecna Fakultni Nemocnice V Praze	Prague	Czechia
Rigshospitalet	Copenhagen	Denmark
Ubkugdjuio Og Ablguoj	Edegem	Belgium
Hmupp Bsrcxw Hp	Bergen	Norway

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	18.02.2019
Czechia	Not recruiting	18.02.2019
Denmark	Not recruiting	18.02.2019
Italy	Not recruiting	18.02.2019
Norway	Not recruiting	18.02.2019

Trial locations

Investigated drugs:

Pegunigalsidase Alfa

Pegunigalsidase Alpha (PRX-102) is a medication used in this clinical trial to treat patients with Fabry disease. It is administered through an intravenous infusion every four weeks. The purpose of this study is to evaluate the long-term safety and effectiveness of this treatment in managing the symptoms of Fabry disease.

Investigated diseases:

Fabry's disease

Fabry Disease – Fabry disease is a rare genetic disorder caused by a deficiency of the enzyme alpha-galactosidase A. This enzyme deficiency leads to the accumulation of a fatty substance called globotriaosylceramide in the body’s cells. Over time, this buildup can cause a range of symptoms, including pain, particularly in the hands and feet, as well as skin rashes. The disease can also affect the heart, kidneys, and nervous system, leading to complications in these organs. Symptoms often begin in childhood or adolescence and can vary widely in severity among individuals. As the disease progresses, it can lead to more significant health issues, impacting the quality of life.

Trial ID:

2024-516735-27-00

Protocol code:

CLI-06657AA1-03

NCT ID:

NCT03614234

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Long-Term Safety and Effectiveness of Pegunigalsidase Alfa for Adults with Fabry Disease

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?