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ClinicalTrials.gov public records Last synced May 22, 2026, 3:49 AM EDT

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

Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Fabry Disease Clear all
Terminated No phase listed Observational
View directory record Official record
Conditions
ESRD, Fabry Disease, Rare Diseases, Kidney Diseases, End Stage Renal Disease, Genetic Diseases, X-Linked
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 65 Years
Enrollment
338 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 23, 2021 · Synced May 22, 2026, 3:49 AM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
agalsidase beta
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
18
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 15 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2015 · Synced May 22, 2026, 3:49 AM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
Lucerastat
Drug
Lead sponsor
Idorsia Pharmaceuticals Ltd.
Industry
Eligibility
18 Years and older
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2029
U.S. locations
14
States / cities
Birmingham, Alabama • Irvine, California • Oakland, California + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced May 22, 2026, 3:49 AM EDT
Completed Phase 1Phase 2 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa)
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
5
States / cities
Gainesville, Florida • Iowa City, Iowa • Baltimore, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 12, 2023 · Synced May 22, 2026, 3:49 AM EDT
Recruiting Phase 2Phase 3 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102 1 mg/kg every two weeks
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
2 Years to 17 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2031
U.S. locations
6
States / cities
Phoenix, Arizona • Atlanta, Georgia • Iowa City, Iowa + 3 more
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced May 22, 2026, 3:49 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Fabry Disease
Interventions
AT1001 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
4
States / cities
Costa Mesa, California • Miami, Florida • Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Aug 2, 2017 · Synced May 22, 2026, 3:49 AM EDT
Recruiting Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Fabry Disesase
Interventions
HM15421/GC1134A
Drug
Lead sponsor
GC Biopharma Corp
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
6
States / cities
Los Angeles, California • Kansas City, Kansas • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced May 22, 2026, 3:49 AM EDT
Completed Phase 3 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Biological
Lead sponsor
Shire
Industry
Eligibility
18 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Iowa City, Iowa • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Jun 7, 2021 · Synced May 22, 2026, 3:49 AM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
Venglustat (GZ402671), Agalsidase alfa, Agalsidase beta (GZ419828), Migalastat
Drug
Lead sponsor
Sanofi
Industry
Eligibility
18 Years to 65 Years
Enrollment
104 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
8
States / cities
Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced May 22, 2026, 3:49 AM EDT
Completed Not applicable Interventional Results available
View directory record Official record
Conditions
Fabry's Disease
Interventions
Smartpill, Endoscopic Mucosal Resection
Device · Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 70 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 26, 2023 · Synced May 22, 2026, 3:49 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
Interview
Other
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
Not listed
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 20, 2023 · Synced May 22, 2026, 3:49 AM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal (agalsidase alfa)
Biological
Lead sponsor
Shire
Industry
Eligibility
7 Years to 17 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
5
States / cities
Decatur, Georgia • Durham, North Carolina • Dallas, Texas + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced May 22, 2026, 3:49 AM EDT
Completed Phase 4 Interventional Results available
View directory record Official record
Conditions
Fabry's Disease
Interventions
AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, Dexamethasone, Montelukast, Loratadine, Cetirizine, Fexofenadine
Drug
Lead sponsor
Sanofi
Industry
Eligibility
2 Years to 65 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
5
States / cities
Santa Monica, California • Atlanta, Georgia • Grand Rapids, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced May 22, 2026, 3:49 AM EDT
Completed Phase 2 Interventional
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 22, 2026, 3:49 AM EDT
Terminated No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
Measures of arterial stiffness and endothelial function, Ambulatory blood pressure monitoring, Cardiopulmonary exercise testing (CPET), Serum and urine biomarkers
Diagnostic Test
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
8 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 18, 2025 · Synced May 22, 2026, 3:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
18 Years and older
Enrollment
2,724 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2017
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Feb 12, 2018 · Synced May 22, 2026, 3:49 AM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Non-ischemic Cardiomyopathy, Cardiomyopathies, Hypertrophic Cardiomyopathy, Right Ventricular Arrhythmogenic Cardiomyopathy, Cardiac Amyloidosis, Anderson Fabry Disease, Sarcoidosis, Cancer Therapy-related Cardiac Dysfunction, Ventricular Arrythmia, Heart Failure
Interventions
Not listed
Lead sponsor
Montefiore Medical Center
Other
Eligibility
18 Years and older
Enrollment
5,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
1
States / cities
The Bronx, New York
Source: ClinicalTrials.gov public record
Updated Feb 13, 2025 · Synced May 22, 2026, 3:49 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Lysosomal Storage Disorders
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
1 Day to 100 Years
Enrollment
80 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2022
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Feb 22, 2021 · Synced May 22, 2026, 3:49 AM EDT
Terminated Phase 1Phase 2 Interventional
View directory record Official record
Conditions
Fabry Disease, Lysosomal Storage Diseases
Interventions
FLT190
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2023 · Synced May 22, 2026, 3:49 AM EDT
Completed No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Fabry Disease, Healthy
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2000 – 2001
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced May 22, 2026, 3:49 AM EDT
Completed No phase listed Observational
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
7 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 3, 2017 · Synced May 22, 2026, 3:49 AM EDT
Terminated Phase 3 Interventional Results available
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat hydrochloride
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years and older
Enrollment
85 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
11
States / cities
Atlanta, Georgia • Chicago, Illinois • Kansas City, Kansas + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 1, 2018 · Synced May 22, 2026, 3:49 AM EDT
Enrolling by invitation No phase listed Observational
View directory record Official record
Conditions
Fabry Disease, Fabry Disease, Cardiac Variant
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2039
U.S. locations
8
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated May 24, 2025 · Synced May 22, 2026, 3:49 AM EDT