Fabry’s disease – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Tue, 02 Jun 2026 12:24:22 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Fabry’s disease – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Title: Evaluating low-dose prednisone with standard therapy to improve heart function in patients with cardiac manifestations of Fabry disease https://clinicaltrials.eu/trial/title-evaluating-low-dose-prednisone-with-standard-therapy-to-improve-heart-function-in-patients-with-cardiac-manifestations-of-fabry-disease/ Thu, 30 Apr 2026 13:08:11 +0000 https://clinicaltrials.eu/trial/title-evaluating-low-dose-prednisone-with-standard-therapy-to-improve-heart-function-in-patients-with-cardiac-manifestations-of-fabry-disease/ This clinical study focuses on patients with Fabry disease, a rare genetic condition that can affect the heart. The study will evaluate how adding low-dose prednisone (a corticosteroid medication) to standard treatments might help improve heart function in people who already receive either enzyme replacement therapy or chaperone therapy for their condition.

The research aims to determine if prednisone can reduce inflammation in the heart muscle, which is believed to be an important factor in heart problems caused by Fabry disease. Patients will receive either prednisone tablets or a placebo in addition to their regular Fabry disease treatment. The maximum daily dose of prednisone will be 5 milligrams, and the treatment period will last for 26 weeks.

During the study, doctors will monitor various aspects of heart health using different tests, including cardiac MRI (a detailed heart imaging test), echocardiography (heart ultrasound), and blood tests to measure substances that can indicate heart damage. They will also track how patients feel and how well they can perform daily activities to understand if the treatment is making a difference in their quality of life.

]]> Study of venglustat tablets to reduce neuropathic and abdominal pain in patients aged 16 and older with Fabry disease who are untreated or haven’t received treatment for 6 months https://clinicaltrials.eu/trial/study-on-the-effect-of-venglustat-on-neuropathic-and-abdominal-pain-in-patients-aged-16-and-older-with-fabry-disease/ Thu, 30 Apr 2026 13:07:54 +0000 https://clinicaltrials.eu/trial/study-on-the-effect-of-venglustat-on-neuropathic-and-abdominal-pain-in-patients-aged-16-and-older-with-fabry-disease/ This clinical trial focuses on Fabry Disease, a rare genetic condition that affects multiple parts of the body and causes various symptoms including nerve pain and stomach pain. The study tests a medication called venglustat (also known as GZ402671 or SAR402671) that is taken as tablets by mouth. Some participants will receive the actual medication while others will receive placebo tablets.

The purpose of this research is to determine how well venglustat works in treating nerve pain and abdominal pain in people with Fabry Disease who have not received previous treatment or have not been treated for at least 6 months. The study will last for 12 months, during which participants will take either venglustat tablets or placebo tablets daily.

Throughout the study, participants will be monitored for changes in their pain levels and other symptoms related to Fabry Disease. The medication will be tested at different doses (up to 15 mg per day) to evaluate its effectiveness and safety. Various aspects of the disease will be tracked, including changes in specific substances in the blood, use of pain medications, and other symptoms such as tiredness and digestive problems.

]]> A Study of Migalastat Safety and Effects in Children Aged 2 to 12 Years with Fabry Disease and Suitable Gene Changes https://clinicaltrials.eu/trial/a-study-of-migalastat-safety-and-effects-in-children-aged-2-to-12-years-with-fabry-disease-and-suitable-gene-changes/ Wed, 29 Apr 2026 15:08:02 +0000 https://clinicaltrials.eu/trial/a-study-of-migalastat-safety-and-effects-in-children-aged-2-to-12-years-with-fabry-disease-and-suitable-gene-changes/ This study involves children between 2 and 12 years old who have Fabry disease. Fabry disease is a rare inherited condition where the body cannot properly break down a certain type of fat, which then builds up in various organs and can cause health problems. The treatment being tested is migalastat hydrochloride, which is also known by its code name AT1001. This medication comes as a dispersible tablet that can be dissolved in liquid before taking it by mouth. The study will only include children who have specific changes in their genes that make them suitable for treatment with this medication.

The purpose of this study is to understand how the body of children processes migalastat and to confirm the correct dose for different age groups, as well as to evaluate the safety of this treatment in children with Fabry disease. During the study, children will receive migalastat treatment for 12 months. The highest daily dose will be 140 milligrams, and the treatment will be given by mouth. Throughout the study, doctors will collect blood samples to measure the levels of medication in the body and will monitor various aspects of health.

During the 12 months of treatment, doctors will regularly check for any side effects or unwanted reactions to the medication. They will also monitor changes in laboratory test results, vital signs such as blood pressure and heart rate, physical examination findings, body weight and height, heart function using tests like electrocardiograms and echocardiograms, and development in older children. The study will track how the medication affects various complications of Fabry disease that the children may have, such as problems with the heart, kidneys, nervous system, or other organs.

]]> Study comparing venglustat tablets with standard therapy for adult patients with Fabry disease and left ventricular hypertrophy https://clinicaltrials.eu/trial/study-comparing-venglustat-tablets-with-standard-therapy-for-adult-patients-with-fabry-disease-and-left-ventricular-hypertrophy/ Wed, 29 Apr 2026 15:07:21 +0000 https://clinicaltrials.eu/trial/study-comparing-venglustat-tablets-with-standard-therapy-for-adult-patients-with-fabry-disease-and-left-ventricular-hypertrophy/ This study focuses on patients with Fabry disease, a rare genetic condition that affects various organs in the body. The research aims to evaluate a new medication called venglustat compared to standard treatments including agalsidase alfa, agalsidase beta, and migalastat in treating patients who have an enlarged heart muscle, also known as left ventricular hypertrophy.

The study will compare two groups of patients over 18 months. One group will receive venglustat tablets taken by mouth, while the other group will continue their current standard treatment. The standard treatments are given either as an infusion into a vein or as oral capsules, depending on the specific medication.

Throughout the study, patients will undergo various medical examinations including cardiac MRI scans to measure heart muscle size, heart function tests, kidney function tests, and assessments of various symptoms related to Fabry disease. The study will also monitor the safety of the treatment by checking for any side effects that may occur during the treatment period.

]]> Long-Term Safety Study of ST-920 Gene Therapy for Patients with Fabry Disease https://clinicaltrials.eu/trial/long-term-safety-study-of-st-920-gene-therapy-for-patients-with-fabry-disease/ Wed, 29 Apr 2026 15:04:01 +0000 https://clinicaltrials.eu/trial/long-term-safety-study-of-st-920-gene-therapy-for-patients-with-fabry-disease/ This clinical trial focuses on Fabry Disease, a genetic condition that affects the body’s ability to break down certain fatty substances. The study involves a treatment called ST-920, which is a type of gene therapy. This therapy uses a specially designed virus to deliver a gene that helps produce an enzyme called alpha-galactosidase A, which is missing or not working properly in people with Fabry Disease.

The purpose of the study is to evaluate the long-term safety of ST-920. Participants in this study have previously received the ST-920 treatment in an earlier trial. The study will monitor these participants over an extended period to observe any potential side effects or adverse events that may occur as a result of the treatment.

Throughout the study, participants will undergo regular check-ups and assessments to ensure their well-being and to gather data on the treatment’s safety. This long-term follow-up is crucial to understanding how the treatment affects patients over time and to ensure that it remains a safe option for managing Fabry Disease.

]]> Study on the Long-Term Safety and Effectiveness of Pegunigalsidase Alfa for Adults with Fabry Disease https://clinicaltrials.eu/trial/study-on-the-long-term-safety-and-effectiveness-of-pegunigalsidase-alfa-for-adults-with-fabry-disease/ Wed, 29 Apr 2026 14:59:32 +0000 https://clinicaltrials.eu/trial/study-on-the-long-term-safety-and-effectiveness-of-pegunigalsidase-alfa-for-adults-with-fabry-disease/ 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.

]]> Study on the Safety and Effects of Pegunigalsidase Alfa for Children and Adolescents with Fabry Disease https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-pegunigalsidase-alfa-for-children-and-adolescents-with-fabry-disease/ Wed, 29 Apr 2026 14:59:25 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-effects-of-pegunigalsidase-alfa-for-children-and-adolescents-with-fabry-disease/ This clinical trial is focused on studying Fabry disease, a rare genetic condition that affects the body’s ability to break down a specific type of fat, leading to a buildup in various organs. The study is investigating a treatment called PRX-102, which is a form of enzyme replacement therapy. This treatment is given as a concentrate for solution, which is then infused into the bloodstream through a vein. The main goal of the study is to learn about the safety and effects of PRX-102 in children and adolescents aged 2 to less than 18 years who have been diagnosed with Fabry disease.

Participants in the study will receive the treatment and be monitored over a period of time to assess how their bodies respond. The study will look at various factors, including how the treatment affects the symptoms of Fabry disease, such as pain and organ function. The study will also monitor for any side effects or reactions to the treatment. Participants will be grouped into different age categories to better understand how the treatment works in different age groups.

Throughout the study, regular check-ups will be conducted to track the participants’ health and development. This includes physical exams, blood tests, and other assessments to ensure the treatment is working as intended and to identify any potential issues early on. The study aims to provide valuable information on the use of PRX-102 in young patients with Fabry disease, potentially leading to improved treatment options in the future.

]]> Study on Long-Term Safety of Oral Lucerastat for Adults with Fabry Disease https://clinicaltrials.eu/trial/study-on-long-term-safety-of-oral-lucerastat-for-adults-with-fabry-disease/ Wed, 29 Apr 2026 14:58:13 +0000 https://clinicaltrials.eu/trial/study-on-long-term-safety-of-oral-lucerastat-for-adults-with-fabry-disease/ This clinical trial focuses on studying the long-term safety and tolerability of a medication called Lucerastat in adults with Fabry disease. Fabry disease is a rare genetic disorder that affects the body’s ability to break down a certain type of fat, leading to a buildup that can cause a variety of symptoms, including pain, kidney problems, and heart issues. Lucerastat, also known by its code name ACT-434964, is taken orally in the form of a hard capsule.

The purpose of this study is to observe how well patients tolerate Lucerastat over an extended period. Participants in the study will take Lucerastat daily, with a maximum dose of 2000 milligrams per day, for up to 72 weeks. Throughout the study, researchers will monitor participants for any side effects or adverse reactions to the medication. This study is open-label, meaning that both the participants and the researchers know that Lucerastat is being administered, and it is a single-arm study, which means all participants receive the same treatment without a comparison group.

By participating in this study, researchers aim to gather valuable information on the long-term effects of Lucerastat in managing Fabry disease. This information could help in understanding the potential benefits and risks of using Lucerastat as a treatment option for individuals living with this condition. The study is designed to ensure that participants’ health and safety are closely monitored throughout the trial period.

]]> Study on the Safety and Tolerability of ST-920 Gene Therapy for Patients with Fabry Disease https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-st-920-gene-therapy-for-patients-with-fabry-disease/ Wed, 29 Apr 2026 14:27:21 +0000 https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-st-920-gene-therapy-for-patients-with-fabry-disease/ This clinical trial is focused on studying Fabry Disease, a genetic condition that affects the body’s ability to break down a specific type of fat, leading to a buildup in various organs. The study is testing a new treatment called ST-920, which is a type of gene therapy. This treatment uses a specially designed virus to deliver a healthy copy of a gene that helps produce an enzyme called alpha-galactosidase A, which is missing or not working properly in people with Fabry Disease.

The purpose of the study is to assess the safety and tolerability of ST-920. Participants in the study will receive a single dose of this treatment through an injection into a vein. The study will monitor participants over a period of time to see how their bodies respond to the treatment. This includes regular check-ups and tests to ensure the treatment is safe and to observe any changes in the symptoms of Fabry Disease.

Throughout the study, researchers will collect information on any side effects that may occur and will perform various tests, such as blood tests and imaging scans, to monitor the health of participants. The study aims to provide valuable information on whether ST-920 can be a safe and effective treatment option for people living with Fabry Disease.

]]> Stowarzyszenie Rodzin z Chorobą Fabry’ego https://clinicaltrials.eu/organisation/stowarzyszenie-rodzin-z-choroba-fabryego/ Wed, 29 Apr 2026 13:34:18 +0000 https://clinicaltrials.eu/organisation/stowarzyszenie-rodzin-z-choroba-fabryego/ Stowarzyszenie Rodzin z Chorobą Fabry’ego (Association of Families with Fabry Disease) was founded in 2002 with the primary goal of integrating families affected by Fabry disease. The association is dedicated to supporting these families and plays a crucial role in advocating for improved access to existing therapies for all patients. They actively work to shorten the path to treatment, ensuring that individuals with Fabry disease can access the care they need more efficiently.

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