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Trackers› Clinical trials› Fabry Disease
Clinical trial register · United States · industry-sponsored

Fabry Disease Trials in the USA

Every industry-sponsored study on the ClinicalTrials.gov registry that lists a US site and is open, about to open, active-but-closed, or enrolling by invitation. This deliberately excludes academic and other non-industry sponsors — the full US registry across all sponsors runs to over 22,000 recruiting studies alone.

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Source: ClinicalTrials.gov. Complete coverage of the industry-sponsored slice — all 9094 matching records are in this view (non-industry sponsors are out of scope by design, not truncated). Synced 17 Sep 2026, 08:23 UTC
9094
Studies in scope
industry-sponsored, all four open statuses
4935
Recruiting now
open to new participants
2188
Lead sponsors
across recruiting studies
Top states
Georgia 11 Virginia 9 Alabama 7 Pennsylvania 7 California 6
Narrow the registerReset
Recruitment status
Phase
Lead sponsor
Amicus Therapeutics4 Chiesi Farmaceutici S.p.A.3 Sanofi2 Sangamo Therapeutics1 Genzyme, a Sanofi Company1
State
Georgia11 Virginia9 Alabama7 Pennsylvania7 California6
All 21 states
Target enrolment
to
14 of 9094 studies
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Status
Study
Lead sponsor
Phase
Enrol
US states
Start
Recruiting
A Study of Patients With Fabry Disease (US Specific)
NCT06906367Fabry Disease
Amicus Therapeutics
Observational
450
Alabama, Arkansas, Georgia, Missouri, New York, Pennsylvania, Texas, Virginia
2026-02-13
Recruiting
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
NCT06904261Fabry Disease
Amicus Therapeutics
Phase 3
8
Georgia, Minnesota, North Carolina, Ohio, Pennsylvania, Virginia
2026-01-08
Recruiting
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
NCT06328608Fabry Disease
Chiesi Farmaceutici S.p.A.
Phase 2/3
22
Arizona, Georgia, Iowa, Ohio, Utah, Virginia
2025-07-29
Recruiting
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
NCT06941025Fabry Disease, Pregnancy, Pregnancy Complications
Chiesi Farmaceutici S.p.A.
Observational
10
District of Columbia
2025-05-23
Recruiting
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
NCT04252066Fabry Disease
Amicus Therapeutics
Observational
20
Pennsylvania
2025-03-18
Recruiting
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
NCT06663358Fabry Disease
Chiesi Farmaceutici S.p.A.
Observational
100
Alabama, Georgia, Illinois, Iowa, Michigan, Virginia
2024-11-06
Recruiting
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
NCT06270316Fabry Disease
UniQure Biopharma B.V.
Phase 1/2
12
Alabama, Georgia, Illinois, Minnesota, New York, Pennsylvania, Utah, Virginia
2024-06-05
Active, closed
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
NCT04020055Fabry Disease
Amicus Therapeutics
Phase 3
14
Georgia, Ohio, Virginia
2022-10-31
Active, closed
A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease
NCT05280548Fabry Disease
Sanofi
Phase 3
104
Alabama, California, Georgia, Illinois, New York, Texas, Utah, Virginia
2022-05-03
Active, closed
A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease
NCT05206773Fabry Disease
Sanofi
Phase 3
122
Alabama, California, Florida, Georgia, New York, Ohio, Pennsylvania, Texas
2022-03-11
Active, closed
Long-Term Follow-up of Subjects Who Were Treated With ST-920
NCT05039866Fabry Disease, Fabry Disease, Cardiac Variant
Sangamo Therapeutics
Observational
32
California, Florida, Georgia, Iowa, Minnesota, New York, Ohio, Virginia
2021-08-16
Active, closed
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease
NCT04519749Fabry Disease
4D Molecular Therapeutics
Phase 1/2
18
California, Georgia, Pennsylvania, Virginia
2020-09-01
Active, closed
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
NCT03737214Fabry Disease
Idorsia Pharmaceuticals Ltd.
Phase 3
107
Alabama, California, Florida, Illinois, Iowa, Massachusetts, Michigan, Pennsylvania
2018-12-18
Recruiting
Fabry Disease Registry & Pregnancy Sub-registry
NCT00196742Fabry Disease
Genzyme, a Sanofi Company
Observational
9,000
Alabama, Arizona, Arkansas, California, Connecticut, District of Columbia, Florida, Georgia
2001-07-31
Rows per page Showing 1–14 of 14
1
Cite this view

Priya Life Science (2026). Clinical trial register: industry-sponsored studies with a US investigative site, filtered. Derived from ClinicalTrials.gov. Retrieved 18 September 2026. https://priyalifescience.com/clinical-trials-usa/condition/fabry-disease

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Listing here is informational and not medical advice; eligibility is determined by each study team. Compare pay for the teams running these studies in the US Pharma Salary Guide, see who's behind them in the US Company Directory, or track approvals on the FDA Approvals Tracker. For Irish, UK and EU sites, see the Ireland, UK and EU registers.