Clinical trials
Fabry Disease clinical trials
Below are recruiting fabry disease clinical trials, each written for real people, not researchers. We’re tracking 47 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT04440254Recruiting
Long ECG monitoring for Fabry disease heart rhythm
This study checks how your heart’s electrical rhythm looks over a longer period using an ECG (a heart rhythm recording) in people with Fabry disease. It may help doctors understand heart rhythm changes better so future care can be more tailored and safer.
Paris, ParisAges 18 years+ - NCT05698901Recruiting
Testing heart blood tests and scans to monitor Fabry disease
This study checks whether certain heart-related blood tests and heart scans can help monitor people with Fabry disease. It may be useful for both people who have never received enzyme replacement therapy and those who have.
TaipeiAges 18 years+ - NCT05710692RecruitingPhase 2/Phase 3
Study drug PRX-102 for Fabry disease in Japanese patients
This trial tests whether PRX-102 is safe and effective for treating Fabry disease in Japanese participants. It also measures how the drug moves through the body and affects body systems, to see if it helps your symptoms and organ function.
Chikushino-shi, FukuokaAges 13–70 - NCT06081062RecruitingPhase 3
Testing Fabagal in people with Fabry disease
This trial is testing a new enzyme replacement therapy called Fabagal for people with Fabry disease who have not had prior treatment. It aims to see if it helps with kidney, heart, or brain problems caused by the disease.
ManilaAges 8 years+ - NCT06114329RecruitingPhase 2
Oral AL01211 for untreated men with Fabry disease
This study tests an oral medication called AL01211 for men who have classic Fabry disease but have never been treated before. The goal is to see if it is safe and if it can help reduce the build-up of fatty substances in the body.
Guangzhou, GuangdongAges 18–60 - NCT06207552RecruitingEarly Phase 1
Gene therapy study for children with Fabry disease
This study tests a new gene therapy for children aged 7 to 17 with Fabry disease. The treatment aims to fix the faulty gene that causes the condition, which may help reduce symptoms and prevent organ damage.
ShanghaiAges 7–18 - NCT06270316RecruitingPhase 1/Phase 2
Gene therapy for classic Fabry disease
This trial tests a new gene therapy called AMT-191 for men with classic Fabry disease who still have symptoms despite standard enzyme replacement therapy. It aims to see if the treatment is safe and can improve symptoms.
Birmingham, AlabamaAges 18–50 - NCT06325488Recruiting
Kidney Health Study in Fabry Disease
This study uses MRI scans to look at how Fabry disease affects the kidneys, including scarring, swelling, and oxygen levels. It may help researchers understand kidney problems better and find new ways to monitor the disease.
CopenhagenAges 18 years+ - NCT06328608RecruitingPhase 2/Phase 3
PRX-102 for children with Fabry disease
This study tests a new enzyme replacement therapy called PRX-102 for children with Fabry disease. It aims to see if the drug is safe and helps manage symptoms like pain and eye or skin changes.
Phoenix, ArizonaAges 2–17 - NCT06539624Recruiting
Testing a new gene therapy for Fabry disease
This trial is testing a new treatment called EXG110 for people with Fabry disease. It's designed to see if it's safe and if it can help. You may be able to join if you have a confirmed diagnosis and at least one symptom of Fabry disease.
Shanghai, Shanghai MunicipalityAges 7 years+ - NCT06776419Recruiting
Heart inflammation and scarring in Fabry disease
This study uses advanced heart scans (PET/CT and MRI) to look at inflammation, blood vessel health, and scarring in the hearts of people with Fabry disease. It aims to better understand how Fabry disease affects the heart and find new ways to track or treat these changes.
CopenhagenAges 18 years+ - NCT07187440Recruiting
Study of Fabry disease treatment in Chinese children and adults
This study tests an enzyme replacement therapy (agalsidase alfa) for Fabry disease in Chinese children and adults. It's an observational study that follows patients who receive the treatment as part of their regular care.
Hefei, AnhuiAges 7 years+ - NCT01581424Recruiting
Study how kidney findings change in Fabry disease
This study looks at the natural course of Fabry disease in the kidneys and how different kidney test results relate to each other. It may help doctors better understand kidney progression and treatment outcomes, including for people who have or have not had enzyme replacement therapy.
Minneapolis, MinnesotaAges 1–75 - NCT03362164Recruiting
Studying heart involvement in people with Fabry disease
This study looks at how the heart is affected in adults with Fabry disease. It may help doctors better understand heart-related changes in order to guide care.
Würzburg, BavariaAges 18 years+ - NCT06880250Enrolling by invitation
Enzyme therapy study for Fabry disease patients
This trial tests if enzyme replacement therapy works well and is safe for Fabry disease. It is for people who are already prescribed this treatment.
AstrakhanAges 8–65 - NCT00455104Recruiting
Fabry disease registry for Canadians and eligible adults
This study is a national registry that collects health and medical information from people with Fabry disease. It helps researchers better understand the condition and plan future care and studies.
Calgary, AlbertaAges 5–85 - NCT05067868RecruitingPhase 4
Replagal for Fabry disease in children and adults in India
This trial studies Replagal (enzyme replacement therapy) for people with Fabry disease who have not used it before. It aims to see how the treatment works and how safely it can be used in children and adults.
KolkataAges Any age - NCT06806813Recruiting
Italian Fabry disease heart registry
This study is creating a registry of people with Fabry disease to better understand how it affects the heart. By joining, you help doctors learn more about the condition and improve care for future patients.
Chieti, Abruzzo/ChietiAges 2 years+ - NCT06906367Recruiting
Long-term study of Fabry disease treatments in the US
This study looks at how well available Fabry disease treatments (like migalastat or ERT) work over time in real life. It may help doctors understand who benefits most from these medicines.
Birmingham, AlabamaAges 18 years+ - NCT03869554Recruiting
Screening for Fabry disease in people with kidney biopsy results
This study tests a screening approach to find Fabry disease in adults whose kidney biopsy findings are unclear or suggest certain kidney patterns. If Fabry disease is missed, it can delay targeted treatment, so screening may help identify people who could benefit from earlier care.
AngersAges 18 years+ - NCT07560956Enrolling by invitation
Quality of Life in Adults With Untreated Fabry Disease
This study looks at how Fabry disease affects daily life in adults who haven't started treatment yet. Researchers want to understand your experiences and challenges to improve care for people with this rare genetic condition.
GothenburgAges 18 years+ - NCT06226987Recruiting
Heart imaging study for Fabry disease
This study uses special MRI scans to look at how Fabry disease affects the heart. It may help researchers understand the disease better.
Cambridge, CambridgeshireAges 18 years+ - NCT05629559RecruitingPhase 1/Phase 2
Study drug 4D-310 for Fabry disease with heart problems
This Phase 1/2 study tests how safe and potentially effective 4D-310 is for adults with Fabry disease who have heart involvement. It may help people who can’t tolerate standard treatments like enzyme replacement therapy (ERT) or migalastat, or who still have disease progression.
MelbourneAges 18–65 - NCT06884358Recruiting
Heart and exercise capacity in Fabry disease
This study looks at how Anderson-Fabry disease affects your heart and ability to exercise. Researchers want to understand this better by testing your heart function and how well you can do physical activity.
Brescia, BresciaAges Any age
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Fabry Disease trials by city
Studies with a site in or near these metro areas.
Fabry Disease trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for fabry disease?
- Yes. Clin2 currently lists 47 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.