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ClinicalTrials.gov public records Last synced Oct 8, 2026, 1:02 PM EDT

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Showing 49–72 of 97 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Anderson Fabry Disease Clear all
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 22, 2026 · Synced Oct 8, 2026, 1:02 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function

NCT01730469
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 3, 2017 · Synced Oct 8, 2026, 1:02 PM 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 13, 2023 · Synced Oct 8, 2026, 1:02 PM EDT
Completed No phase listed Observational

Registry of Fabry Disease - A Multicenter Observational Study

NCT00055016
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2008
U.S. locations
2
States / cities
Bethesda, Maryland • Cambridge, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 8, 2026, 1:02 PM EDT
Completed Phase 4 Interventional Results available

A Study of the Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

NCT00074984
View directory record Official record
Conditions
Fabry Disease
Interventions
Fabrazyme (agalsidase beta), Placebo
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
82 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2004
U.S. locations
19
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 16 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2013 · Synced Oct 8, 2026, 1:02 PM EDT
Completed No phase listed Observational

Establishment of Biomarkers for Fabry Disease

NCT01165697
View directory record Official record
Conditions
Fabry Disease
Interventions
Fluorescein angiography
Other
Lead sponsor
Ohio State University
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 13, 2017 · Synced Oct 8, 2026, 1:02 PM EDT
Terminated No phase listed Observational

Kidney Information Network for Disease Research and Education

NCT03321604
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 24, 2021 · Synced Oct 8, 2026, 1:02 PM 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 4, 2008 · Synced Oct 8, 2026, 1:02 PM EDT
Completed Phase 2 Interventional Results available

Evaluation of the Long-term Safety, Pharmacodynamics, and Exploratory Efficacy of GZ/SAR402671 in Treatment-Naïve Adult Male Patients With Fabry Disease

NCT02489344
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 23, 2021 · Synced Oct 8, 2026, 1:02 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Gene Therapy for Gaucher's and Fabry Disease Using Viruses and Blood-Forming Cells

NCT00001234
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 4, 2008 · Synced Oct 8, 2026, 1:02 PM EDT
Completed Phase 1 Interventional

An Open-Label Maintenance Study of the Enzyme Replacement Therapy Replagal in Patients With Fabry Disease

NCT00357786
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 17, 2011 · Synced Oct 8, 2026, 1:02 PM EDT
Completed Phase 3 Interventional Results available

Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

NCT01124643
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 8, 2021 · Synced Oct 8, 2026, 1:02 PM 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 4, 2008 · Synced Oct 8, 2026, 1:02 PM EDT
Recruiting No phase listed Observational

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

NCT01581424
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
1 Year to 75 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2030
U.S. locations
2
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Aug 14, 2026 · Synced Oct 8, 2026, 1:02 PM EDT
Recruiting Phase 1Phase 2 Interventional

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

NCT06270316
View directory record Official record
Conditions
Fabry Disease
Interventions
AMT-191
Drug
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
10
States / cities
Birmingham, Alabama • Los Angeles, California • Atlanta, Georgia + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 8, 2026, 1:02 PM EDT
Completed Phase 2 Interventional

Alpha-Galactosidase A Replacement Therapy for Fabry Disease

NCT00048906
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 4, 2008 · Synced Oct 8, 2026, 1:02 PM EDT
Completed Phase 2 Interventional Results available

Drug-Drug Interaction Study Between AT1001 (Migalastat Hydrochloride) and Agalsidase in Participants With Fabry Disease

NCT01196871
View directory record Official record
Conditions
Fabry Disease
Interventions
Migalastat HCl, Agalsidase Beta, Agalsidase Alfa
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2012
U.S. locations
5
States / cities
Birmingham, Alabama • Decatur, Georgia • Iowa City, Iowa + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 19, 2018 · Synced Oct 8, 2026, 1:02 PM 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 7, 2021 · Synced Oct 8, 2026, 1:02 PM EDT
Completed No phase listed Observational

Fabry Outcome Survey (FOS)

NCT03289065
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 16, 2021 · Synced Oct 8, 2026, 1:02 PM EDT
Completed Phase 3 Interventional Results available

A Study of Two Fabrazyme (Agalsidase Beta) Dosing Regimens in Treatment-naïve, Male Pediatric Patients Without Severe Symptoms

NCT00701415
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 29, 2016 · Synced Oct 8, 2026, 1:02 PM EDT
Recruiting Phase 2Phase 3 Interventional

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

NCT06328608
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 Aug 10, 2026 · Synced Oct 8, 2026, 1:02 PM 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 2, 2015 · Synced Oct 8, 2026, 1:02 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Fabry Screening Study

NCT01019629
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 13, 2018 · Synced Oct 8, 2026, 1:02 PM 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 3, 2020 · Synced Oct 8, 2026, 1:02 PM EDT