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ClinicalTrials.gov public records Last synced Jul 23, 2026, 10:11 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Active, not recruiting Phase 3 Interventional

A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease

NCT03737214
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 Jul 1, 2026 · Synced Jul 23, 2026, 10:11 AM EDT
Terminated Phase 2 Interventional Results available

Safety and Efficacy Study of IFX-1 in add-on to Standard of Care in GPA and MPA

NCT03712345
View directory record Official record
Conditions
Granulomatosis With Polyangiitis (GPA), Microscopic Polyangiitis (MPA)
Interventions
IFX-1 low dose, IFX-1 high dose, Placebo
Drug
Lead sponsor
InflaRx GmbH
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
33
States / cities
Scottsdale, Arizona • Loma Linda, California • Doral, Florida + 30 more
Source: ClinicalTrials.gov public record
Updated May 25, 2022 · Synced Jul 23, 2026, 10:11 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Migalastat Food Effect Study

NCT01489995
View directory record Official record
Conditions
Fabry Disease
Interventions
A (migalastat), B (migalastat), C (migalastat), D (migalastat), E (migalastat)
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Dec 17, 2013 · Synced Jul 23, 2026, 10:11 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 Jul 23, 2026, 10:11 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 Jul 23, 2026, 10:11 AM EDT
Completed No phase listed Observational

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

NCT00106912
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 23, 2026, 10:11 AM 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 16, 2011 · Synced Jul 23, 2026, 10:11 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

An Open-label Study of Povetacicept in Autoantibody-Associated Glomerular Diseases

NCT05732402
View directory record Official record
Conditions
Lupus Nephritis, Immunoglobulin A Nephropathy, Membranous Nephropathy, Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis
Interventions
Povetacicept
Drug
Lead sponsor
Alpine Immune Sciences, Inc.
Industry
Eligibility
18 Years and older
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
18
States / cities
Phoenix, Arizona • Tucson, Arizona • Valencia, California + 14 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2025 · Synced Jul 23, 2026, 10:11 AM EDT
Recruiting Phase 3 Interventional

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

NCT06904261
View directory record Official record
Conditions
Fabry Disease
Interventions
Migalastat HCl 20 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
2 Years to 11 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
6
States / cities
Atlanta, Georgia • Minneapolis, Minnesota • Charlotte, North Carolina + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2026 · Synced Jul 23, 2026, 10:11 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 Jul 23, 2026, 10:11 AM EDT
Completed No phase listed Observational

Antiproteinuric Agents and Fabry Disease

NCT00343577
View directory record Official record
Conditions
Fabry Disease, Proteinuria
Interventions
Not listed
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
14 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Nov 18, 2013 · Synced Jul 23, 2026, 10:11 AM EDT
Terminated Not applicable Interventional Results available

Methadone, Morphine, or Oxycodone in Treating Pain in Patients With Cancer

NCT00726830
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Pain, Precancerous Condition, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
methadone hydrochloride, morphine sulfate, oxycodone hydrochloride
Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
2
States / cities
Spartanburg, South Carolina • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 23, 2020 · Synced Jul 23, 2026, 10:11 AM EDT
Completed No phase listed Observational

Development of Strategies to Increase Enrollment in Clinical Trials for Children With Cancer

NCT00002485
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Leukemia, Liver Cancer, Lymphoma, Neuroblastoma, Ovarian Cancer, Psychosocial Effects of Cancer and Its Treatment, Sarcoma, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions
psychosocial assessment and care
Procedure
Lead sponsor
Children's Oncology Group
Network
Eligibility
Up to 21 Years
Enrollment
359 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2005
U.S. locations
26
States / cities
Sacramento, California • Gainesville, Florida • Miami, Florida + 23 more
Source: ClinicalTrials.gov public record
Updated Feb 13, 2014 · Synced Jul 23, 2026, 10:11 AM EDT
Completed Phase 3 Interventional Results available

Belimumab in Remission of VASculitis

NCT01663623
View directory record Official record
Conditions
Vasculitis
Interventions
Placebo, Belimumab 10 mg/kg, Azathioprine
Biological · Drug
Lead sponsor
Human Genome Sciences Inc., a GSK Company
Industry
Eligibility
18 Years and older
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
22
States / cities
Birmingham, Alabama • Mobile, Alabama • Tucson, Arizona + 18 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2018 · Synced Jul 23, 2026, 10:11 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Androgenetic Alopecia in Fabry Disease

NCT01295008
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Baylor Research Institute
Other
Eligibility
18 Years to 64 Years · Male only
Enrollment
107 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2015
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 12, 2016 · Synced Jul 23, 2026, 10:11 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 Jul 23, 2026, 10:11 AM EDT
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 Jul 23, 2026, 10:11 AM 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 Mar 18, 2026 · Synced Jul 23, 2026, 10:11 AM EDT
Completed Early Phase 1 Interventional

Ketones & Mitochondrial Heteroplasmy

NCT01252979
View directory record Official record
Conditions
MELAS Syndrome, Mitochondrial Diseases
Interventions
Medium-Chain Triglycerides
Dietary Supplement
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 5, 2012 · Synced Jul 23, 2026, 10:11 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy (CADASIL) Study

NCT05677880
View directory record Official record
Conditions
CADASIL
Interventions
Study Procedures
Other
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
18 Years and older
Enrollment
660 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2027
U.S. locations
12
States / cities
Los Angeles, California • San Francisco, California • Denver, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Jan 14, 2026 · Synced Jul 23, 2026, 10:11 AM EDT
Not yet recruiting Not applicable Interventional

Development and Efficacy of a Novel, Cost-Effective Gait Training Device Utilized at Home for Stroke Survivors

NCT07250425
View directory record Official record
Conditions
Stroke, Neurological Diseases or Conditions, Hemiparesis;Poststroke/CVA, Gait Disorders, Neurologic, Post Stroke Fatigue, Motor Recovery, Walking Difficulty, Balance Impairment, Falls Prevention, Hemiparesis, Mobility Limitation
Interventions
Rise&Walk InHome by Healing Innovations
Device
Lead sponsor
Healing Innovations
Other
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
West Orange, New Jersey
Source: ClinicalTrials.gov public record
Updated Mar 8, 2026 · Synced Jul 23, 2026, 10:11 AM EDT
Terminated Phase 2 Interventional Results available

PR3-AAV Resilient Remission or PRRR

NCT05376319
View directory record Official record
Conditions
Granulomatosis With Polyangiitis, Microscopic Polyangiitis, ANCA Associated Vasculitis
Interventions
Obinutuzumab, Rituximab
Drug
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
3
States / cities
Boston, Massachusetts • Rochester, Minnesota • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Jul 23, 2026, 10:11 AM 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 12, 2017 · Synced Jul 23, 2026, 10:11 AM EDT