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ClinicalTrials.gov public records Last synced Oct 6, 2026, 12:56 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 97 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Anderson Fabry Disease Clear all

Conditions: Fabry Disease
Interventions: migalastat HCl 150 mg; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 14 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 3
States / cities: Atlanta, Georgia • Cleveland, Ohio • Fairfax, Virginia

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

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

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

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

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

Conditions: Fabry Disease
Interventions: agalsidase alfa; Biological
Lead sponsor: Shire; Industry
Eligibility: Not listed
U.S. locations: 25
States / cities: Tucson, Arizona • La Jolla, California • Los Angeles, California + 21 more

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

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

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

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

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

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

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

Conditions: Fabry Disease
Interventions: Functional MRI and fast bound-pool fraction imaging; Other
Lead sponsor: University of Utah; Other
Eligibility: 18 Years and older
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2022
U.S. locations: 1
States / cities: Salt Lake City, Utah

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

Conditions: Fabry Disease
Interventions: migalastat HCl 150 mg; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 84 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2019
U.S. locations: 7
States / cities: Atlanta, Georgia • Kansas City, Kansas • Grand Rapids, Michigan + 4 more

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

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

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

Conditions: Fabry Disease
Interventions: migalastat HCl; Drug
Lead sponsor: Amicus Therapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 3
States / cities: Decatur, Georgia • New York, New York • Dallas, Texas

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

Conditions: Fabry
Interventions: biomarkers; Diagnostic Test
Lead sponsor: Lysosomal and Rare Disorders Research and Treatment Center, Inc.; Other
Eligibility: 18 Years to 70 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2022
U.S. locations: 1
States / cities: Fairfax, Virginia

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

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A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

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

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

Treatment Protocol of Replagal for Patients With Fabry Disease

Immune Response in Subjects With Fabry Disease Who Are Switching From Agalsidase Alfa to Agalsidase Beta

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

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

Study to Collect Data on Fabry Disease Patients With Enhanceable Alpha-Galactosidase A Activity

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

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

Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

Evaluation of Phenotypic Variability in Fabry Disease

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

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

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

Registry of Fabry Disease - A Multicenter Observational Study

Open-label Long-term Safety Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease Who Have Completed a Previous AT1001 Study

Vasodilation in Patients With Fabry's Disease

Inflammatory Pathways and Cardiac Growth Factors Associated With Fabry Disease Cardiomyopathy

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