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ClinicalTrials.gov public records Last synced Jul 23, 2026, 10:19 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Alpha Galactosidase A Deficiency Clear all
Completed Not applicable Interventional Results available

Evaluation of the Gastrointestinal Manifestation of Fabry's Disease

NCT02798458
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 Jul 23, 2026, 10:19 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan

NCT02985710
View directory record Official record
Conditions
Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
Interventions
Sudoscan, Skin biopsy, QSART
Device · Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
8 Years to 70 Years
Enrollment
102 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2020
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 6, 2021 · Synced Jul 23, 2026, 10:19 AM EDT
Completed Phase 4 Interventional Results available

A Study Evaluating Glycosphingolipid Clearance in Patients Treated With Agalsidase Alfa Who Switch to Agalsidase Beta

NCT01650779
View directory record Official record
Conditions
Fabry Disease
Interventions
Agalsidase beta
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Male only
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
6
States / cities
Coral Springs, Florida • Decatur, Georgia • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 9, 2014 · Synced Jul 23, 2026, 10:19 AM EDT
Completed Phase 3 Interventional Results available

Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

NCT04049760
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
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
5
States / cities
Tampa, Florida • Atlanta, Georgia • Minneapolis, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Jul 23, 2026, 10:19 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

NCT04046224
View directory record Official record
Conditions
Fabry Disease
Interventions
ST-920
Biological
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
9
States / cities
Irvine, California • Tampa, Florida • Atlanta, Georgia + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Jul 23, 2026, 10:19 AM EDT
Completed Phase 3 Interventional Results available

Open Label Extension Study of 1 mg/kg Pegunigalsidase Alfa Every 2 Weeks in Patients With Fabry Disease

NCT03566017
View directory record Official record
Conditions
Fabry Disease
Interventions
pegunigalsidase alfa
Drug
Lead sponsor
Chiesi Farmaceutici S.p.A.
Industry
Eligibility
18 Years to 60 Years
Enrollment
97 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 11 more
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Jul 23, 2026, 10:19 AM EDT
Withdrawn No phase listed Observational

Evaluation of Phenotypic Variability in Fabry Disease

NCT03145779
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Year and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 2, 2020 · Synced Jul 23, 2026, 10:19 AM EDT
Enrolling by invitation No phase listed Observational

Long-Term Follow-up of Subjects Who Were Treated With ST-920

NCT05039866
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 Jul 23, 2026, 10:19 AM EDT
Completed Phase 3 Interventional

A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

NCT00074971
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2004
U.S. locations
14
States / cities
Los Angeles, California • San Francisco, California • Coral Springs, Florida + 11 more
Source: ClinicalTrials.gov public record
Updated Dec 3, 2013 · Synced Jul 23, 2026, 10:19 AM EDT
Terminated Phase 4 Interventional Results available

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

NCT00230607
View directory record Official record
Conditions
Fabry Disease, Alpha Galactosidase A Deficiency
Interventions
agalsidase beta
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
Not listed
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2024
U.S. locations
3
States / cities
Decatur, Georgia • Fairfax, Virginia • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 9, 2024 · Synced Jul 23, 2026, 10:19 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

PET Scans in Normal Volunteers and Patients With Fabry Disease

NCT00005111
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 Jul 23, 2026, 10:19 AM EDT
Completed Phase 2 Interventional Results available

A Study of Fabrazyme in Pediatric Patients With Fabry Disease

NCT00074958
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta)
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
7 Years to 15 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2005
U.S. locations
1
States / cities
Tucson, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 1, 2015 · Synced Jul 23, 2026, 10:19 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Dose-ranging Study of PRX-102 in Adult Fabry Disease Patients

NCT01678898
View directory record Official record
Conditions
Fabry Disease
Interventions
PRX-102
Drug
Lead sponsor
Protalix
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
9
States / cities
Sacramento, California • Atlanta, Georgia • Iowa City, Iowa + 6 more
Source: ClinicalTrials.gov public record
Updated Sep 12, 2023 · Synced Jul 23, 2026, 10:19 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

NCT06065605
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
2
States / cities
Rockville, Maryland • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Oct 3, 2023 · Synced Jul 23, 2026, 10:19 AM EDT
Completed Phase 3 Interventional

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

NCT03614234
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
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
7
States / cities
Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more
Source: ClinicalTrials.gov public record
Updated May 21, 2026 · Synced Jul 23, 2026, 10:19 AM EDT
Completed Phase 4 Interventional Results available

A Prospective Study to Investigate Safety and Tolerability of Shorter Infusion of Fabrazyme

NCT06019728
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 Jul 23, 2026, 10:19 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Investigating Lysosomal Storage Diseases in Minority Groups

NCT02120235
View directory record Official record
Conditions
Lysosomal Storage Disorders, Gaucher Disease, Fabry Disease, Pompe Disease, Niemann-Pick Disease
Interventions
Not listed
Lead sponsor
O & O Alpan LLC
Other
Eligibility
1 Day to 100 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2018
U.S. locations
1
States / cities
Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Apr 3, 2017 · Synced Jul 23, 2026, 10:19 AM EDT
Completed Phase 3 Interventional Results available

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

NCT02795676
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 Jul 23, 2026, 10:19 AM EDT
Completed Phase 3 Interventional Results available

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

NCT00864851
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Biological
Lead sponsor
Shire
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
4
States / cities
Tucson, Arizona • Iowa City, Iowa • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 8, 2021 · Synced Jul 23, 2026, 10:19 AM EDT
Completed No phase listed Observational

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

NCT05186324
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 Jul 23, 2026, 10:19 AM EDT
Completed Phase 2 Interventional

Alternative Dosing and Regimen of Replagal to Treat Fabry Disease

NCT00075244
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 Jul 23, 2026, 10:19 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Vasodilation in Patients With Fabry's Disease

NCT00001774
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 Jul 23, 2026, 10:19 AM EDT
Completed Phase 4 Interventional

Replagal Enzyme Replacement Therapy for Adults With Fabry Disease

NCT00097890
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
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 Jul 23, 2026, 10:19 AM EDT
Terminated No phase listed Observational

Cardiovasculorenal Phenotyping in Fabry Disease Through Noninvasive Testing

NCT05699265
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 Jul 23, 2026, 10:19 AM EDT