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All 38 Fabry Disease trials and their US sites
Enter your ZIP code above to sort them by how far their nearest open site is from you.
- Fabry Disease Registry & Pregnancy Sub-registryMost sites in California, New York and FloridaRecruitingGenzyme, a Sanofi Company74US sites
34 states - Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry DiseaseMost sites in Alabama, California and GeorgiaRecruitingPhase 1/2Ages 18-65UniQure Biopharma B.V.10US sites
10 states - A Study of Patients With Fabry Disease (US Specific)Most sites in Alabama, Arkansas and GeorgiaRecruitingAges 18+Amicus Therapeutics8US sites
8 states - A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry DiseaseMost sites in Alabama, California and FloridaRecruitingPhase 3Ages 18-60Idorsia Pharmaceuticals8US sites
8 states - A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry PatientsMost sites in Alabama, Georgia and IllinoisRecruitingAges 18+Chiesi Farmaceutici S.p.A.6US sites
6 states - A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA VariantsMost sites in Georgia, Minnesota and North CarolinaRecruitingPhase 3Ages 2-11Amicus Therapeutics6US sites
6 states - A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry DiseaseMost sites in Arizona, Georgia and IowaRecruitingPhase 2/3Ages 2-17Chiesi Farmaceutici S.p.A.6US sites
6 states - Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease StudyOnly in MinnesotaRecruitingAges 1-75University of Minnesota2US sites
- A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and BreastfeedingOnly in PennsylvaniaRecruitingAmicus Therapeutics1US site
- Cardiovascular Multimodality Imaging StudyOnly in New YorkRecruitingAges 18+Montefiore Medical Center1US site
- Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and BreastfeedingOnly in District of ColumbiaRecruitingChiesi Farmaceutici S.p.A.1US site
- 4D-310 in Adults With Fabry Disease and Cardiac InvolvementRecruitingPhase 1/2Ages 18-654D Molecular TherapeuticsNo US site yet
- A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry DiseaseRecruitingAges 7+TakedaNo US site yet
- A Study of Replagal in Children and Adults With Fabry Disease in IndiaRecruitingPhase 4ShireNo US site yet
- Anderson-Fabry Disease Fitness Improvement TrainingRecruitingAges 18+General University Hospital, PragueNo US site yet
- Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry DiseaseRecruitingAges 18+Mackay Memorial HospitalNo US site yet
- Canadian Fabry Disease Initiative (CFDI) National RegistryRecruitingAges 5-85Canadian Fabry Research ConsortiumNo US site yet
- Characterization of Patients With Cardiomyopathy to Identify Critical Patients Candidates for Cardiac TransplantationRecruitingAges 12+IRCCS Azienda Ospedaliero-Universitaria di BolognaNo US site yet
- Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational StudyRecruitingAges 18+Technical University of MunichNo US site yet
- CVI Alterations in FD: a Prospective, Multicenter, Observational Cohort StudyRecruitingAges 18+China National Center for Cardiovascular DiseasesNo US site yet
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Who runs the Fabry Disease trials in the US, and what stage they are in
Of the 11 Fabry Disease trials with a US site, 3 are in Phase 3, the last stage before a company can ask the FDA for approval. One more is an earlier Phase 1 or 2 test of safety and dosing. The remaining 7 have no drug phase, such as studies that follow people over time or test a device or a way of giving care. Companies run 9 of them, and universities, hospitals, foundations and government groups run 2. The sponsors with the most are Amicus Therapeutics (3) and Chiesi Farmaceutici S.p.A. (3). The most recently listed one first appeared on ClinicalTrials.gov on August 21, 2026.
Fabry Disease trials in the US, state by state
Of the 11 Fabry Disease trials with a US site, 6 accept children under 18. Their open sites are in 33 states and Washington, DC, most often Georgia, Minnesota and Ohio. A trial counts once in each state where it has an open site, and some trials accept both children and adults.
Show the other 24 states
US hospitals running the most Fabry Disease trials
Centers that run trials for a rare condition often see more people with it than most clinics do, so this list can also help you find doctors who know Fabry Disease.
- Emory University School of MedicineAtlanta, Georgia3 trials
- UPMC Children's Hospital of PittsburghPittsburgh, Pennsylvania3 trials
- Cincinnati Children's Hospital Medical CenterCincinnati, Ohio2 trials
- Emory GeneticsAtlanta, Georgia2 trials
- Lysosomal and Rare Disorders Research and Treatment Center, Inc.Fairfax, Virginia2 trials
- Amicus Therapeutics, Inc. Pregnancy RegistryPhiladelphia, Pennsylvania1 trial
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois1 trial
- Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013Chicago, Illinois1 trial
- Ann & Robert H. Lurie Childrens Hospital of ChicagoChicago, Illinois1 trial
- Arkansas Children's HospitalLittle Rock, Arkansas1 trial
Help getting to a Fabry Disease trial far from home
Ask the study team whether the trial pays back travel, parking and hotel costs, because many sponsors do. The FDA's guidance on paying research volunteers says paying back these costs does not count as pressuring someone to join.
These nonprofits also help patients reach care, and each one sets its own rules.
- Mercy Medical Angels
- Pays for gas cards, ground rides and airline tickets anywhere in the US, and it lists clinical trials as a reason to travel. Household income at or below 300% of the federal poverty guidelines, and usually a trip of 50 miles or more each way.
- Air Charity Network
- Volunteer pilots in all 50 states fly patients for free in small private planes, and members also fly people taking part in clinical trials. You must be able to walk and sit upright for the flight, show financial need and have a backup plan if weather cancels it.
- Ronald McDonald House
- Gives families a room near the hospital while a child gets care, through 394 House programs around the world. Families of a sick child, with rules set by each local chapter.
- Healthcare Hospitality Network
- Keeps a map of hospitality houses near hospitals, and most member houses charge little or nothing to patients and families. Each house sets its own rules, so check with the house near your study site.
Program details were checked on each program's website on October 24, 2026.
Questions about finding a Fabry Disease trial near you
Are there Fabry Disease clinical trials near me?
As of November 13, 2026, 38 Fabry Disease clinical trials are recruiting now and 5 are not yet recruiting. In the US, 11 of them have at least 1 open site, across 33 states and Washington, DC. Enter your ZIP code on this page to rank every one of them by the distance to its closest open site, with the sites shown on a map.
Which states have Fabry Disease clinical trial sites?
Recruiting Fabry Disease trials list open sites in 33 states and Washington, DC. Georgia, Pennsylvania and Virginia have the most, and the table on this page lists every state.
Are there Fabry Disease trials for children near me?
Of the 11 Fabry Disease trials with a US site, 6 accept children under 18. Their open sites are in 33 states and Washington, DC, most often Georgia, Minnesota and Ohio. Each trial sets its own age limits, so check the ages listed on a trial before you contact it.
Who pays for travel to a Fabry Disease clinical trial?
Many trial sponsors pay back travel, parking and hotel costs, so ask the study team before you rule out a far-away site. The FDA's guidance says paying back these costs does not count as pressuring someone to join. Charities such as Mercy Medical Angels and Air Charity Network can also help with rides and flights.
What if there is no Fabry Disease trial near me?
Some trials send staff to your home or use a local lab, so fewer visits happen at the study site. You can also sign up on this page to get an email when a Fabry Disease trial opens a new site or changes status.
How is the distance to a trial site measured?
Distances are straight-line miles from the center of your ZIP code to each study site's listed location, not driving distance. Your browser looks up the ZIP code's coordinates with a free map service, Zippopotam.us, and the ZIP code is never sent to or stored by Trial Friend.
FDA-approved for Fabry Disease: Elfabrio (pegunigalsidase alfa), Fabrazyme (agalsidase beta), Galafold (migalastat).
Clinical trials near you for related conditions
Source: ClinicalTrials.gov, trials with status Recruiting or Not yet recruiting. Refreshed every 6 hours; counts as of November 13, 2026. Sites that ClinicalTrials.gov lists as closed or withdrawn are left out. Children means the trial's age group includes people under 18.