Clinical trials
Fabry Disease clinical trials
Below are recruiting fabry disease clinical trials, each written for real people, not researchers. We’re tracking 46 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT06904261RecruitingPhase 3
Study of migalastat for children with Fabry disease
This trial tests if the oral drug migalastat is safe and works for children aged 2 to under 12 with a specific type of Fabry disease. It may be an option if your child has not been on enzyme therapy recently and has a compatible gene variant.
Atlanta, GeorgiaAges 2–11 - NCT00196742Recruiting
Fabry disease registry and pregnancy follow-up
This study collects health information from people with Fabry disease and specifically from pregnant people with Fabry disease. It helps researchers better understand the condition and how pregnancy affects health, using information you already experience in routine care.
Birmingham, AlabamaAges Any age - NCT04252066Recruiting
Pregnancy and breastfeeding study for women with Fabry disease
This study follows pregnant and breastfeeding women with Fabry disease (and their infants) to better understand what happens during pregnancy and early life. You may or may not be taking migalastat, and the goal is to gather real-world information that could help future care.
Philadelphia, PennsylvaniaAges Any age - NCT06941025Recruiting
Safety of Elfabrio in pregnant and breastfeeding women with Fabry disease
This study follows women with Fabry disease who took the medicine Elfabrio during pregnancy or breastfeeding to see if it is safe for them and their babies. It helps doctors learn more about how this treatment affects mothers and infants.
Washington D.C., District of ColumbiaAges Any age - NCT07235709Recruiting
Study of agalsidase alfa for heart inflammation in Fabry disease
This trial tests whether agalsidase alfa can reduce heart inflammation in people with Fabry disease who have heart problems. You may qualify if you have not had this treatment in the past year.
SeoulAges 15–75 - NCT07495410Enrolling by invitation
Exercise training for Anderson-Fabry disease
This study tests whether a supervised exercise and physiotherapy program can improve fitness, stamina, and daily life for people with Anderson-Fabry disease. It is designed for people who are medically stable and able to join a gentle, progressive training program.
PragueAges 18 years+ - NCT07277361Recruiting
Quality of life study for Fabry disease patients aged 65+
This study looks at the quality of life of people aged 65 and older with Fabry disease. It includes patients who are receiving specific treatment and those who are not, to compare their experiences.
Paris, FranceAges 65 years+ - NCT07336394Recruiting
Study on rare heart muscle diseases using advanced MRI
This study uses a special heart MRI to better diagnose and understand rare heart muscle diseases. It may help doctors predict risks and make more accurate treatment decisions.
BeijingAges Any age - NCT06858397RecruitingPhase 1/Phase 2
Study of a new drug for Fabry disease
This study tests a new investigational drug called HM15421/GC1134A for people with Fabry disease. It aims to see if the drug is safe and tolerable, and to learn how well it works. You may be eligible if you have Fabry disease with symptoms, have not had treatment for at least 6 months, and your kidneys are functioning well.
Los Angeles, CaliforniaAges 18 years+ - NCT07575347Recruiting
Gum Disease and Rare Kidney Disorders Study
This study explores whether people with certain rare kidney diseases or chronic kidney problems have more gum disease than others. Researchers will examine your teeth and gums to understand the connection and help improve care for people with kidney conditions.
BucharestAges 18 years+ - NCT05473637Recruiting
Study causes of small vessel brain disease in stroke and memory issues
This trial looks at people who may have “small vessel” problems in the brain, which can cause stroke, memory trouble, walking changes, movement symptoms, or headaches. It may help researchers better understand the genetic and non-genetic causes, which could improve future diagnosis and treatment.
TaipeiAges 18 years+ - NCT06663358Recruiting
Safety and effectiveness of Elfabrio in Fabry disease patients
This study is for people with Fabry disease who are taking or planning to take the medication Elfabrio (pegunigalsidase alfa). It will look at how safe and effective the treatment is in real-world use.
Birmingham, AlabamaAges 18 years+ - NCT05368038Enrolling by invitation
Newborn screening program for babies up to 4 weeks old
This trial tests a flexible newborn screening process that looks for certain conditions early in life. It may help by catching problems sooner, when treatment can be started earlier.
Brooklyn, New YorkAges Up to 4 weeks - NCT06935578Recruiting
A network for rare brain vessel diseases
This study creates a large Italian network to help diagnose and understand rare cerebrovascular diseases like CADASIL, Fabry's disease, and Moyamoya. It aims to improve care for people with these conditions.
Acquaviva delle Fonti, BAAges 18 years+ - NCT06512571Recruiting
Heart changes in Fabry disease: an observational study
This study looks at people with Fabry disease, a genetic condition that can cause the heart muscle to thicken. Researchers want to understand how the heart's blood vessels change over time, which may help improve care for Fabry disease.
BeijingAges 18 years+ - NCT07109375Recruiting
Long-term study of pegunigalsidase alfa for Fabry disease
This study observes how well the medication pegunigalsidase alfa works over a long period in people with Fabry disease. It helps doctors learn more about managing the condition in everyday life.
ArezzoAges 18 years+ - NCT04856059Recruiting
MRI study for early heart changes in Fabry disease
This study looks at early structural and tissue changes in the heart using a special type of MRI scan in adults with Fabry disease. It may help doctors understand changes earlier and better track heart involvement over time.
Toronto, OntarioAges 18 years+ - NCT06813443Recruiting
Studying cardiomyopathy to find who needs a heart transplant
This study is looking at people with cardiomyopathy to understand which patients are most in need of a heart transplant. By joining, you will help doctors learn more about critical cases and improve care for heart muscle disease.
Bologna, Emilia-Romagna/BolognaAges 12 years+ - NCT06065852Recruiting
National registry for rare kidney diseases
This study creates a registry to collect health information from people with rare kidney diseases. It aims to improve understanding and future treatments by tracking patient experiences.
Bristol, South WestAges Any age - NCT06607471Recruiting
Study on non-ischemic heart muscle diseases
This study looks at different types of heart muscle diseases that are not caused by blocked arteries (non-ischemic cardiomyopathies). It aims to better understand how to diagnose, predict outcomes, and treat these conditions by gathering information from many patients over time.
Milan, MilanoAges 18 years+ - NCT06610019Recruiting
Heart imaging study using MRI
This study uses heart MRI scans to learn more about cardiovascular health in adults. If you are 18 or older and have been referred for a heart MRI at Montefiore, you may be able to join.
The Bronx, New YorkAges 18 years+ - NCT07382128Recruiting
Heart MRI to Understand Hypertrophic Cardiomyopathy
This trial uses a special heart MRI to better understand different types of hypertrophic cardiomyopathy (thick heart muscle). It may help doctors tell apart various forms of the condition and guide treatment.
Bologna, BOAges 18 years+
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Common questions
- Are there clinical trials for fabry disease?
- Yes. Clin2 currently lists 46 recruiting fabry disease studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a fabry disease trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a fabry disease trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.