Fabry disease trials in United States

In United States 13 Also abroad 48

13 trials · From ClinicalTrials.gov, checked 11 November 2026. Ask the study team whether a trial fits you.

Phase 3Taking people
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
Where
Atlanta, Birmingham, Chicago, Dallas, Gainesville, Minneapolis, Oakland, Philadelphia
Age
18 years to 60 years
People
16 worldwide
Run by
Idorsia Pharmaceuticals Ltd.
See details and how to joinNCT07778667 · EU 2026-525207-27-00
No phaseTaking people
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Where
Philadelphia
People
20
Run by
Amicus Therapeutics
See details and how to joinNCT04252066
No phaseTaking people
A Study of Patients With Fabry Disease (US Specific)
Where
Atlanta, Birmingham, Dallas, Fairfax, Little Rock, New York, Pittsburgh, St Louis
Age
18 years and over
People
450
Run by
Amicus Therapeutics
See details and how to joinNCT06906367
Phase 3Taking people
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
Where
Atlanta, Charlotte, Cincinnati, Fairfax, Minneapolis, Pittsburgh
Age
2 years to 11 years
People
8 worldwide
Run by
Amicus Therapeutics
See details and how to joinNCT06904261
No phaseTaking people
Fabry Disease Registry & Pregnancy Sub-registry
Where
Ann Arbor, Aurora, Baltimore, Baton Rouge, Birmingham, Boston, Charlotte, Charlottesville, Chicago, Cincinnati, Cleveland, Columbia, Columbus, Coral Gables, Coral Springs, Dallas, Danville, Decatur, Detroit, Dublin, Durham, Fairfax, Fort Worth, Gainesville, Grand Rapids, Greenville, Hackensack, Helena, Hershey, Huntington, Indianapolis, Iowa City, Jacksonville, Kansas City, Las Vegas, Little Rock, Los Angeles, Louisville, Milwaukee, Minneapolis, Morristown, Nashville, New Brunswick, New Haven, New Hyde Park, New Orleans, New York, Norfolk, Oakland, Omaha, Orange, Paterson, Philadelphia, Phoenix, Pittsburgh, Portland, Providence, Rochester, Sacramento, Salt Lake City, San Diego, San Francisco, Seattle, Springfield, Stanford, St Louis, Syracuse, Tacoma, Tampa, Tucson, Valhalla, Washington D.C.
People
9,000 worldwide
Run by
Genzyme, a Sanofi Company
See details and how to joinNCT00196742
Phase 1/2Taking people
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
Where
Atlanta, Birmingham, Chicago, Cincinnati, Fairfax, Los Angeles, Minneapolis, New York, Pittsburgh, Salt Lake City
Age
18 years to 65 years
People
20
Run by
UniQure Biopharma B.V.
See details and how to joinNCT06270316
No phaseTaking people
Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study
Where
Minneapolis
Age
1 year to 75 years
People
50
Run by
University of Minnesota
See details and how to joinNCT01581424
Phase 2/3Taking people
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Where
Atlanta, Cincinnati, Fairfax, Iowa City, Phoenix, Salt Lake City
Age
2 years to 17 years
People
22 worldwide
Run by
Chiesi Farmaceutici S.p.A.
See details and how to joinNCT06328608 · EU 2022-503128-29-00
No phaseTaking people
Cardiovascular Multimodality Imaging Study
Where
The Bronx
Age
18 years and over
People
10,000
Run by
Montefiore Medical Center
See details and how to joinNCT06610019
No phaseEnrolling by invitation
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
Where
Brooklyn, Manhasset, New York, Queens, The Bronx
Age
Up to 4 weeks
People
100,000
Run by
Albert Einstein College of Medicine
See details and how to joinNCT05368038
No phaseTaking people
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
Where
Washington D.C.
People
10 worldwide
Run by
Chiesi Farmaceutici S.p.A.
See details and how to joinNCT06941025
No phaseTaking people
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
Where
Atlanta, Birmingham, Chicago, Fairfax, Grand Rapids, Iowa City
Age
18 years and over
People
100 worldwide
Run by
Chiesi Farmaceutici S.p.A.
See details and how to joinNCT06663358
Phase 1/2Taking people
A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
Where
Cincinnati, Fairfax, Kansas City, Los Angeles, Minneapolis, Pittsburgh
Age
18 years and over
People
18 worldwide
Run by
GC Biopharma Corp
See details and how to joinNCT06858397