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Study comparing venglustat tablets with standard therapy for adult patients with Fabry disease and left ventricular hypertrophy

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What is this study about?

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.

1 Initial assessment and group assignment

Your participation begins after signing the informed consent form

You will be randomly assigned to one of two groups: either receiving venglustat tablets or continuing your current standard treatment (if any)

Your current treatment may include agalsidase alfa, agalsidase beta, or migalastat

2 Treatment period

The study will last for 18 months

If assigned to venglustat group: you will receive venglustat tablets for oral use

If assigned to standard care group: you will continue receiving your current medication through either oral capsules or intravenous infusion, depending on your treatment

3 Regular monitoring

Your heart will be examined using cardiac magnetic resonance imaging (MRI)

Regular heart ultrasound (echocardiography) will be performed

Your kidney function will be monitored through blood tests

Eye examinations will check your lens clarity

You will complete questionnaires about tiredness and mood changes

Blood samples will be taken to measure medication levels in your body

4 Safety monitoring

Any side effects or health changes will be recorded throughout the study

Regular check-ups will monitor your overall health

The study will track changes in symptoms related to Fabry disease, including tiredness and leg swelling

5 Study completion

Final assessments will be conducted at the end of the 18-month period

All test results will be compared with your initial measurements

The main focus will be on changes in your heart muscle thickness as measured by MRI

Who Can Join the Study?

  • Age between 18 and 65 years old
  • Must have a confirmed diagnosis of Fabry disease and previous symptoms related to this condition
  • Can be either currently receiving treatment with agalsidase alfa, agalsidase beta, or migalastat (medications used to treat Fabry disease), or not receiving any treatment
  • Must have left ventricular hypertrophy (a condition where the heart’s main pumping chamber becomes thickened)
  • If female, must not be pregnant or breastfeeding
  • Must use appropriate contraception methods during the study
  • If male, must not donate sperm during the study period
  • Must be willing and able to sign an informed consent form before any study procedures begin

Who Cannot Join the Study?

  • Age below 18 years or above 65 years
  • Patients with severe heart conditions other than those related to Fabry disease
  • Pregnant or breastfeeding women
  • Patients currently participating in other clinical trials
  • Patients with severe kidney dysfunction (poor kidney function)
  • History of allergic reactions to similar medications
  • Patients who cannot follow the study protocol due to physical or mental conditions
  • Patients taking medications that could interfere with the study drug
  • Patients with uncontrolled high blood pressure
  • Inability to provide informed consent
  • History of substance abuse within the past year
  • Any condition that, in the opinion of the study doctor, would make participation unsafe
  • Patients who have had major surgery within the last 3 months
  • Severe liver problems or abnormal liver function tests

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi Lodz Poland
Center For Pediatric And Adolescent Medicine Of The Johannes Gutenberg University Mainz Mainz Germany
Azienda Ospedaliera Universitaria Federico II Di Napoli Naples Italy
Medical University Of Graz Graz Austria

Other Sites

Site Name City Country Status
Hospital General Universitario Gregorio Maranon Madrid Spain
University General Hospital Of Heraklion Heraklion Greece
SphinCS GmbH Hochheim Am Main Germany
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Charite Universitaetsmedizin Berlin KöR Berlin Germany
Vseobecna Fakultni Nemocnice V Praze Prague Czechia
Rigshospitalet Copenhagen Denmark
Hospital Alvaro Cunqueiro Vigo Spain
University General Hospital Attikon General Hospital Of West Attica H Agia Varvara Chaidari Greece
Riqxgxxpwhdddivo Hstxbdud Garches France
Lklmy Gvfsobp Hjxacauz Of Akwtnt Athens Greece
Hiior Bhraze Hf Bergen Norway
Aytzusnwt Ulk Amsterdam The Netherlands
Umfleagnqrzrmrmufhjff Wpwfmihyx Avc Wuerzburg Germany
Aeewjxj Ugysd Snzurnozy Ljpqor Dq Brarkts Bologna Italy
Knvqfdpgb Soafrge Skyzzbfldyoeyqa io Jfhj Pajqo Iv Cracow Poland

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not recruiting
03.05.2022
Czechia Czechia
Not recruiting
03.05.2022
Denmark Denmark
Not recruiting
03.05.2022
France France
Not recruiting
03.05.2022
Germany Germany
Not recruiting
03.05.2022
Greece Greece
Not recruiting
03.05.2022
Italy Italy
Not recruiting
03.05.2022
Norway Norway
Not recruiting
03.05.2022
Poland Poland
Not recruiting
03.05.2022
Spain Spain
Not recruiting
03.05.2022
The Netherlands The Netherlands
Not recruiting
03.05.2022

Trial locations

Investigated drugs:

Venglustat is an investigational oral medication being studied for treating Fabry disease. It works by reducing the buildup of harmful substances in the body’s cells, particularly in the heart. This medication aims to help patients with Fabry disease who have developed heart problems, specifically left ventricular hypertrophy (enlarged heart muscle).

Standard of care Fabry therapies refers to the currently approved and commonly used treatments for Fabry disease. These treatments help manage the symptoms and complications of Fabry disease by replacing the missing enzyme or reducing the accumulation of harmful substances in the body’s tissues and organs.

Investigated diseases:

Fabry Disease – A rare genetic disorder that affects the body’s ability to break down specific types of fats called glycolipids. The disease causes these fatty substances to build up in blood vessels, tissues, and organs throughout the body. This accumulation typically begins in childhood and gradually increases over time, affecting multiple body systems. The condition is inherited and primarily affects the heart, kidneys, nervous system, and skin. As the disease progresses, it can lead to decreased function in affected organs and tissues. The disease affects males more severely than females, though both can develop symptoms.

Trial ID:
2023-509715-91-00
Protocol code:
EFC16158 – CARAT
NCT ID:
NCT05280548
Trial Phase:
Therapeutic confirmatory (Phase III)

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