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Fabry Disease clinical trials recruiting in the U.S.

10 studies for Fabry Disease are recruiting participants at sites across the United States.

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Recently updated studies

Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease

RecruitingPhase 1/2
Ages 18 to 65 · Men · Updated Sep 24, 2026 · NCT06270316

A Study of Patients With Fabry Disease (US Specific)

RecruitingObservational study
Ages 18 and older · All sexes · Updated Sep 9, 2026 · NCT06906367

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

RecruitingPhase 3
Ages 2 to 11 · All sexes · Updated Sep 1, 2026 · NCT06904261

Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study

RecruitingObservational study
Ages 1 to 75 · All sexes · Updated Aug 14, 2026 · NCT01581424

Cardiovascular Multimodality Imaging Study

RecruitingObservational study
Ages 18 and older · All sexes · Updated Aug 10, 2026 · NCT06610019

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

RecruitingPhase 2/3
Ages 2 to 17 · All sexes · Updated Aug 10, 2026 · NCT06328608

Fabry Disease Registry & Pregnancy Sub-registry

RecruitingObservational study
All ages · All sexes · Updated Jun 23, 2026 · NCT00196742

A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

RecruitingObservational study
Ages 18 and older · All sexes · Updated Nov 14, 2025 · NCT06663358

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

RecruitingObservational study
All ages · Women · Updated Jul 8, 2025 · NCT04252066

From ClinicalTrials.gov, data retrieved Sep 29, 2026. Each study sets its own eligibility; the study team decides who can join.