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ClinicalTrials.gov public records Last synced May 22, 2026, 12:10 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 97 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Anderson Fabry Disease Clear all
Completed No phase listed Observational
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
14 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 18, 2013 · Synced May 22, 2026, 12:10 AM EDT
Approved For Marketing No phase listed Expanded access
View directory record Official record
Conditions
Fabry Disease
Interventions
Pegunigalsidase Alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
U.S. locations
11
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2024 · Synced May 22, 2026, 12:10 AM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
Iohexol
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
5 Years to 25 Years · Male only
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
5
States / cities
La Jolla, California • Decatur, Georgia • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2020 · Synced May 22, 2026, 12:10 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 7, 2025 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa), agalsidase beta
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 11 more
Source: ClinicalTrials.gov public record
Updated Sep 12, 2023 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
Migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
12 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
7
States / cities
Tampa, Florida • Atlanta, Georgia • Minneapolis, Minnesota + 4 more
Source: ClinicalTrials.gov public record
Updated Nov 29, 2021 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
Agalsidase beta
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
5 Years to 18 Years · Male only
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
3
States / cities
Decatur, Georgia • Cincinnati, Ohio • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 28, 2016 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
Baylor Research Institute
Other
Eligibility
Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 30, 2015 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal agalsidase alfa, Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
39 Years to 45 Years · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 16, 2011 · Synced May 22, 2026, 12:10 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Shire
Industry
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2021
U.S. locations
1
States / cities
Lexington, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 15, 2021 · Synced May 22, 2026, 12:10 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Cerebrovascular Accident, Fabry Disease, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1997 – 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Gaucher's Disease
Interventions
human glucocerebrosidase cDNA
Genetic
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1988 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 22, 2026, 12:10 AM EDT
Not listed No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
University of Washington
Other
Eligibility
1 Day to 90 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
3
States / cities
Decatur, Georgia • Minneapolis, Minnesota • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Aug 27, 2017 · Synced May 22, 2026, 12:10 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Fabry's Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
325 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1995 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced May 22, 2026, 12:10 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Fabry Disease, Pregnancy, Pregnancy Complications
Interventions
Not applicable- observational study
Other
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2034
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced May 22, 2026, 12:10 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
Pegunigalsidase-alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
6
States / cities
Birmingham, Alabama • Atlanta, Georgia • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 13, 2025 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
Agalsidase alfa
Drug
Lead sponsor
Shire
Industry
Eligibility
7 Years to 17 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2011
U.S. locations
13
States / cities
Tucson, Arizona • Palm Beach Gardens, Florida • Lake Charles, Louisiana + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2021 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Fabry Disease
Interventions
A (migalastat), B (migalastat), C (migalastat), D (migalastat), E (migalastat)
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Dec 17, 2013 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
Lucerastat, Placebo
Drug
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
18 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
17
States / cities
Birmingham, Alabama • Irvine, California • Oakland, California + 14 more
Source: ClinicalTrials.gov public record
Updated Aug 8, 2024 · Synced May 22, 2026, 12:10 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years and older · Male only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced May 22, 2026, 12:10 AM EDT
Terminated Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
AVR-RD-01
Drug
Lead sponsor
AVROBIO
Industry
Eligibility
16 Years to 50 Years · Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
2
States / cities
Hackensack, New Jersey • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 4, 2024 · Synced May 22, 2026, 12:10 AM EDT
Withdrawn Phase 2 Interventional
View directory record Official record
Conditions
Fabry Disease, Neuropathic Pain
Interventions
Gabapentin, placebo
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 28, 2019 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
GZ/SAR402671
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
3
States / cities
Atlanta, Georgia • Cincinnati, Ohio • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Mar 22, 2021 · Synced May 22, 2026, 12:10 AM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
DRX005B
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 22, 2026, 12:10 AM EDT