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Showing 1–24 of 97 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Filters: Condition: Fabry's Disease Clear all
Completed Phase 3 Interventional Results available

Study of the Effects of Oral AT1001 (Migalastat Hydrochloride) in Patients With Fabry Disease

NCT00925301
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat hydrochloride, Placebo
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years to 74 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
13
States / cities
Los Angeles, California • San Francisco, California • Decatur, Georgia + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2018 · Synced Sep 25, 2026, 8:04 AM EDT
Completed Phase 2 Interventional

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

NCT00071877
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 25, 2026, 8:04 AM EDT
Completed Phase 2 Interventional Results available

A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease

NCT00214500
View directory record Official record
Conditions
Fabry Disease
Interventions
migalastat HCl
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 55 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
5
States / cities
Los Angeles, California • Decatur, Georgia • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 29, 2018 · Synced Sep 25, 2026, 8:04 AM 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 22, 2021 · Synced Sep 25, 2026, 8:04 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 Sep 25, 2026, 8:04 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 Sep 25, 2026, 8:04 AM 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 23, 2026 · Synced Sep 25, 2026, 8:04 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 Sep 25, 2026, 8:04 AM EDT
Completed Phase 2 Interventional Results available

Safety Study of Replagal® Therapy in Children With Fabry Disease

NCT01363492
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 Sep 25, 2026, 8:04 AM EDT
Recruiting No phase listed Observational

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

NCT06941025
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 Sep 25, 2026, 8:04 AM EDT
Approved For Marketing No phase listed Expanded access

Open-Label Expanded Access Treatment With Pegunigalsidase Alfa for Fabry Disease Patients

NCT04552691
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 Sep 25, 2026, 8:04 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 Sep 25, 2026, 8:04 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 Sep 25, 2026, 8:04 AM EDT
Completed No phase listed Observational

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

NCT04804566
View directory record Official record
Conditions
Fabry Disease
Interventions
This is a non-interventional study
Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Eagan, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Sep 25, 2026, 8:04 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

NCT03454893
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 Sep 25, 2026, 8:04 AM 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 7, 2021 · Synced Sep 25, 2026, 8:04 AM EDT
Recruiting No phase listed Observational

A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

NCT06663358
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 Sep 25, 2026, 8:04 AM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

NCT03425539
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 Sep 25, 2026, 8:04 AM 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 18, 2018 · Synced Sep 25, 2026, 8:04 AM EDT
Completed No phase listed Observational Results available

A Study to Describe the Experience of Both Patients and Their Clinicians in the Treatment of Fabry Disease With Enzyme Replacement Therapy.

NCT04281537
View directory record Official record
Conditions
Fabry Disease
Interventions
Enzyme Replacement Therapy (ERT)
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
82 participants
Timeline
2020 – 2022
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Sep 25, 2026, 8:04 AM EDT
Terminated Phase 3 Interventional Results available

Open-Label Phase 3 Long-Term Safety Study of Migalastat

NCT01458119
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 Sep 25, 2026, 8:04 AM EDT
Recruiting No phase listed Observational

Cardiovascular Multimodality Imaging Study

NCT06610019
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
10,000 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 Aug 9, 2026 · Synced Sep 25, 2026, 8:04 AM EDT
Completed Phase 2 Interventional Results available

Dosing Study of Replagal in Patients With Fabry Disease

NCT00068107
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 Sep 25, 2026, 8:04 AM EDT