Fabry disease: open trials in United States
13 open trials have a site in United States. Registry data; only the trial team decides who can take part.
NCT07778667
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Trial Treatment · Phase 3 · Idorsia Pharmaceuticals Ltd.
Emory University School of Medicine, Atlanta · University of Alabama of Birmingham, Birmingham · Ann & Robert H. Lurie Childrens Hospital of Chicago, Chicago · Dallas Nephrology Associates (DNA) - Dallas Landry Office Practice, Dallas · UF Health Center for Pediatric Neuromuscular and Rare Diseases, Gainesville · The University of Minnesota, Fairview Health, Minneapolis · UCSF Benioff Childens Hospital, Oakland · Hospital of University Pennsylvania, Translational Medicine and Human Genetics, Philadelphia
Registry updated 2026-11-03
NCT04252066
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Research · Amicus Therapeutics
Amicus Therapeutics, Inc. Pregnancy Registry, Philadelphia
Registry updated 2026-10-28
NCT06906367
A Study of Patients With Fabry Disease (US Specific)
Research · Amicus Therapeutics
Emory Genetics, Atlanta · UAB Nephrology Research Clinic at Paula Building, Birmingham · Renal Disease Research Institute, Dallas · Lysosomal and Rare Disorders Research and Treatment Center, Inc, Fairfax · Arkansas Children's Hospital, Little Rock · New York-Presbyterian Morgan Stanley Children's Hospital - Columbia University Medical Center, New York · UPMC Children's Hospital of Pittsburgh, Pittsburgh · Washington University School of Medicine, St Louis
Registry updated 2026-10-28
NCT06904261
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Trial Treatment · Phase 3 · Amicus Therapeutics
Emory Genetics, Atlanta · Atrium Health/Levine Children's Hospital, Charlotte · Cincinnati Children's Hospital Medical Center, Cincinnati · Lysosomal and Rare Disorders Research and Treatment Center, Inc, Fairfax · University of Minnesota/Masonic Children's Hospital, Minneapolis · UPMC Children's Hospital of Pittsburgh, Pittsburgh
Registry updated 2026-10-22
NCT00196742
Fabry Disease Registry & Pregnancy Sub-registry
Research · Genzyme, a Sanofi Company
University Of Michigan Pediatrics- Site Number : 840107, Ann Arbor · University Of Colorado At Denver Genetics- Site Number : 840068, Aurora · Baltimore · Ochsner Health Center - Baton Rouge- Site Number : 840120, Baton Rouge · University Of Alabama Birmingham- Nephrology- Site Number : 840073, Birmingham · University Of Alabama Birmingham- Nephrology- Site Number : 840018, Birmingham · Brigham & Women's Hospital- Site Number : 840103, Boston · Massachusetts General Hospital-Genetics- Site Number : 840062, Boston · Boston Children's Hospital- Site Number : 840092, Boston · Atrium Health - Carolinas Medical Center- Site Number : 840065, Charlotte · University Of Virginia School Of Medicine-Pediatrics- Site Number : 840078, Charlottesville · Rush University Medical Center Genetics- Site Number : 840079, Chicago · Ann & Robert H. Lurie Children's Hospital of Chicago- Site Number : 840013, Chicago · Cincinnati Children's Hospital Medical Center (Cincinnati Children's Hospital)- Site Number : 840033, Cincinnati · University Hospitals Cleveland Medical Center- Site Number : 840119, Cleveland · Cleveland Clinic - Cleveland- Site Number : 840048, Cleveland · University Of Missouri Health System Department Of Genetics- Site Number : 840031, Columbia · Ohio State University Department Of Genetics- Site Number : 840097, Columbus · Nationwide Children's Hospital- Site Number : 840091, Columbus · University of Miami- Site Number : 840006, Coral Gables · University of Miami Sylvester Comprehensive Cancer Center - Coral Springs- Site Number : 840075, Coral Springs · Baylor Institute of Metabolic Disease- Site Number : 840058, Dallas · Geisinger Medical Center- Site Number : 840111, Danville · Emory University School Of Medicine- Human Genetics- Site Number : 840060, Decatur · Children's Hospital of Michigan- Site Number : 840066, Detroit · Lsd Data Registry Site Llc- Site Number : 840094, Dublin · Duke University Medical Center Genetics Dept- Site Number : 840045, Durham · O & O Alpan- Site Number : 840025, Fairfax · Cook Children's Medical Center- Site Number : 840024, Fort Worth · University Of Florida Dept Of Genetics- Site Number : 840083, Gainesville · Corewell Health - Helen DeVos Children's Hospital (HDVCH)- Site Number : 840019, Grand Rapids · Infusion Associates - Grand Rapids- Site Number : 840050, Grand Rapids · Greenwood Genetic Center - Greenville- Site Number : 840055, Greenville · Joseph M. Sanzari Children'S Hospital- Site Number : 840101, Hackensack · Shodair Children'S Hospital- Site Number : 840090, Helena · Penn State University Medical Center-Neurology- Site Number : 840104, Hershey · NYU Langone Health - Perlmutter Cancer Center - Huntington- Site Number : 840040, Huntington · Indianapolis University School Of Medicine- Site Number : 840027, Indianapolis · University of Iowa- Site Number : 840032, Iowa City · University Of Florida Pediatrics Genetics- Site Number : 840121, Jacksonville · University of Kansas Medical Center- Site Number : 840071, Kansas City · Children's Specialty Center - Las Vegas- Site Number : 840008, Las Vegas · University of Arkansas for Medical Sciences- Site Number : 840113, Little Rock · Arkansas Children's Hospital- Site Number : 840109, Little Rock · University Of California Los Angeles- Site Number : 840088, Los Angeles · Southern California Permanente Medical Group- Site Number : 840108, Los Angeles · Usc Health Sciences Center Dept Of Genetics- Site Number : 840082, Los Angeles · University of Louisville - Health Sciences Center- Site Number : 840030, Louisville · Milwaukee · Children's Hospitals and Clinics of Minnesota- Site Number : 840114, Minneapolis · M Health Fairview University of Minnesota Medical Center - West Bank- Site Number : 840076, Minneapolis · Children's Hospitals and Clinics of Minnesota- Site Number : 840046, Minneapolis · Atlantic Health System- Site Number : 840099, Morristown · Vanderbilt University Medical Center - Medical Laboratories- Site Number : 840049, Nashville · Saint Peter's University Hospital - New Brunswick- Site Number : 840016, New Brunswick · Yale University School of Medicine- Site Number : 840047, New Haven · Northwell Health- Site Number : 840102, New Hyde Park · Tulane University School of Medicine- Site Number : 840001, New Orleans · Icahn School of Medicine at Mount Sinai- Site Number : 840080, New York · Metropolitan Hospital Center- Site Number : 840110, New York · Children's Hospital of The King's Daughters- Site Number : 840072, Norfolk · UCSF Benioff Children's Hospital - Oakland- Site Number : 840029, Oakland · University of Nebraska Medical Center- Site Number : 840084, Omaha · Children's Hospital of Orange County- Site Number : 840074, Orange · University of California Irvine Medical Center- Site Number : 840036, Orange · St. Joseph's Regional Medical Center - Paterson- Site Number : 840057, Paterson · The Children's Hospital of Philadelphia- Site Number : 840034, Philadelphia · Children'S Hospital Of Philadelphia Hup Medical Genetics- Site Number : 840089, Philadelphia · Phoenix Children's Hospital- Site Number : 840003, Phoenix · UPMC Children's Hospital of Pittsburgh- Site Number : 840023, Pittsburgh · Maine Medical Partners Pediatric Specialty Care- Site Number : 840064, Portland · Oregon Health & Science University (OHSU)- Site Number : 840116, Portland · Oregon Health & Science University (OHSU)- Site Number : 840038, Portland · Rhode Island Hospital Genetics- Site Number : 840053, Providence · University of Rochester Medical Center- Site Number : 840105, Rochester · Kaiser Permanente Sacramento Medical Center- Site Number : 840042, Sacramento · Uc Davis Mind Institute- Site Number : 840010, Sacramento · University of Utah- Site Number : 840043, Salt Lake City · University of California at San Diego- Site Number : 840007, San Diego · University of California San Francisco - Parnassus Campus- Site Number : 840051, San Francisco · University of Washington Medical Center- Site Number : 840059, Seattle · Seattle Children's Hospital- Site Number : 840028, Seattle · Baystate Medical Center- Site Number : 840002, Springfield · Stanford University Medical Center- Site Number : 840022, Stanford · Washington University- Site Number : 840100, St Louis · SUNY Upstate Medical University - Site Number : 840052, Syracuse · MultiCare Mary Bridge Children's Health Center- Site Number : 840115, Tacoma · University Of Florida-Genetics- Site Number : 840096, Tampa · University of Arizona- Site Number : 840015, Tucson · New York Medical College- Site Number : 840039, Valhalla · Children's National Medical Center - Washington- Site Number : 840067, Washington D.C.
Registry updated 2026-10-14
NCT06270316
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
Trial Treatment · Phase 1/2 · UniQure Biopharma B.V.
Emory University School of Medicine, Atlanta · The Kirklin Clinic of University of Alabama Birmingham Hospital, Birmingham · Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago · Children's Hospital Medical Center, Cincinnati · Lysosomal & Rare Disorders Research and Treatment Center, Inc, Fairfax · University of California Los Angeles (UCLA)., Los Angeles · MHealth Fairview University of Minnesota Medical Center East Bank, Minneapolis · NYC Health + Hospitals/Metropolitan, New York · UPMC Children's Hospital of Pittsburgh, Pittsburgh · University of Utah, Clinical and Translational Sciences Institute, Salt Lake City
Registry updated 2026-09-24
NCT01581424
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
Research · University of Minnesota
University of Minnesota, Minneapolis · Universtity of Minnesota, Department of Pediatric Nephrology, Minneapolis
Registry updated 2026-08-14
NCT06328608
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Trial Treatment · Phase 2/3 · Chiesi Farmaceutici S.p.A.
Emory Genetics Clinical Trials Center, Atlanta · Cincinnati Children's Hospital Medical Center, Cincinnati · Lysosomal and Rare Disorders Research and Treatment Center, Inc, Fairfax · University of Iowa, Iowa City · Phoenix Children's, Phoenix · University of Utah, Salt Lake City
Registry updated 2026-08-10
NCT06610019
Cardiovascular Multimodality Imaging Study
Research · Montefiore Medical Center
Montefiore Health System, The Bronx
Registry updated 2026-08-10
NCT05368038
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Research · Albert Einstein College of Medicine
Maimonides Medical Center, Brooklyn · NYU Langone Hospital - Brooklyn, Brooklyn · North Shore University Hospital, Manhasset · NYU Langone Health - Tisch Hospital, New York · Mount Sinai West, New York · Mount Sinai Hospital, New York · Long Island Jewish Medical Center, Queens · Jack D. Weiler Hospital, The Bronx · ScreenPlus Coordinating Core, Children's Hospital at Montefiore, The Bronx
Registry updated 2026-07-28
NCT06941025
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 Breastfeeding
Research · Chiesi Farmaceutici S.p.A.
No physical study sites - Decentralized, web-based registry, Washington D.C.
Registry updated 2026-03-19
NCT06663358
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
Research · Chiesi Farmaceutici S.p.A.
Emory University School of Medicine, Atlanta · University of Alabama at Birmingham, Birmingham · Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago · Lysosomal & Rare Disorder Research & Treatment Center (LRDRTC), Fairfax · Infusion Associates, Grand Rapids · University of Iowa Hospitals and Clinics, Iowa City
Registry updated 2025-11-14
NCT06858397
A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
Trial Treatment · Phase 1/2 · GC Biopharma Corp
Children's Hospital Medical Center, Cincinnati · Lysosomal and Rare Disorders Research and Treatment Center, Fairfax · University of Kansas, School of Medicine, Kansas City · David Geffen School of Medicine UCLA, UCLA Health, Los Angeles · University of Minnesota, Minneapolis · University of Pittsburgh Medical Center Children's Hoispital of Pittsburgh, Pittsburgh
Registry updated 2025-11-04
All Fabry disease trials and the map
This page is not medical advice. Talk to your doctor before contacting a trial.