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ClinicalTrials.gov public records Last synced Oct 21, 2026, 2:05 PM EDT

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Showing 97–120 of 200 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Cerebral Small Vessel Diseases Clear all
Completed Phase 2 Interventional Results available

Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes

NCT00887562
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Idebenone, Placebo
Drug · Other
Lead sponsor
Michio Hirano
Other
Eligibility
8 Years to 65 Years
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 26, 2016 · Synced Oct 21, 2026, 2:05 PM EDT
Completed Early Phase 1 Interventional

Pemetrexed Disodium in the Cerebrospinal Fluid of Patients With Leptomeningeal Metastases

NCT00424242
View directory record Official record
Conditions
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Metastatic Cancer, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Precancerous Condition, Secondary Myelofibrosis, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
Pemetrexed
Drug
Lead sponsor
Northwestern University
Other
Eligibility
18 Years to 120 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2011
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Sep 8, 2021 · Synced Oct 21, 2026, 2:05 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 21, 2026, 2:05 PM EDT
Not listed Phase 2 Interventional

Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome

NCT00068913
View directory record Official record
Conditions
MELAS Syndrome
Interventions
Dichloroacetate
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Years and older
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 21, 2026, 2:05 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers

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

NCT03678324
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 26, 2022 · Synced Oct 21, 2026, 2:05 PM EDT
Completed Phase 1 Interventional

L-Citrulline Dose Finding Safety Study in MELAS

NCT03952234
View directory record Official record
Conditions
MELAS Syndrome
Interventions
L-Citrulline
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jun 26, 2024 · Synced Oct 21, 2026, 2:05 PM 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 22, 2026 · Synced Oct 21, 2026, 2:05 PM 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 13, 2016 · Synced Oct 21, 2026, 2:05 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05368038
View directory record Official record
Conditions
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease, GM1 Gangliosidosis, Gaucher Disease, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy, Mucopolysaccharidosis II, Mucopolysaccharidosis III-B, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Niemann-Pick Disease, Type C
Interventions
Confirmatory Testing
Diagnostic Test
Lead sponsor
Albert Einstein College of Medicine
Other
Eligibility
Up to 4 Weeks
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2029
U.S. locations
9
States / cities
Brooklyn, New York • Manhasset, New York • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 28, 2026 · Synced Oct 21, 2026, 2:05 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 21, 2026, 2:05 PM EDT
Completed Phase 3 Interventional

Secondary Prevention of Small Subcortical Strokes Trial

NCT00059306
View directory record Official record
Conditions
Cerebrovascular Accident, Hypertension
Interventions
aspirin, clopidogrel, Target of Blood Pressure, placebo
Drug · Other
Lead sponsor
University of British Columbia
Other
Eligibility
30 Years and older
Enrollment
3,020 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2012
U.S. locations
39
States / cities
Mobile, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 32 more
Source: ClinicalTrials.gov public record
Updated Apr 4, 2013 · Synced Oct 21, 2026, 2:05 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Fabry Disease Gene Therapy Study

NCT04040049
View directory record Official record
Conditions
Fabry Disease, Lysosomal Storage Diseases
Interventions
FLT190
Genetic
Lead sponsor
Spur Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
4
States / cities
Los Angeles, California • New York, New York • Pittsburgh, Pennsylvania + 1 more
Source: ClinicalTrials.gov public record
Updated Sep 3, 2026 · Synced Oct 21, 2026, 2:05 PM EDT
Completed Phase 4 Interventional Results available

Pilot Study of Short-Course Glucocorticoids and Rituximab for Treatment of ANCA-Associated Vasculitis

NCT02169219
View directory record Official record
Conditions
Granulomatosis With Polyangiitis, Microscopic Polyangiitis
Interventions
Glucocorticoids, Rituximab
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 85 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 25, 2021 · Synced Oct 21, 2026, 2:05 PM EDT
Terminated Phase 2 Interventional Results available

Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)

NCT04475549
View directory record Official record
Conditions
MELAS
Interventions
IW-6463 Tablets
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 29, 2024 · Synced Oct 21, 2026, 2:05 PM 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 4, 2017 · Synced Oct 21, 2026, 2:05 PM EDT
Completed Phase 1 Interventional

Thalidomide and Docetaxel in Treating Patients With Advanced Cancer

NCT00049296
View directory record Official record
Conditions
Cancer
Interventions
docetaxel, thalidomide
Drug
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2006
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jun 11, 2010 · Synced Oct 21, 2026, 2:05 PM 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 24, 2026 · Synced Oct 21, 2026, 2:05 PM 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 15, 2026 · Synced Oct 21, 2026, 2:05 PM EDT
Recruiting Not applicable Interventional

Minimally Invasive Neuroendoscopic Ultra-Early Targeted ICH Evacuation

NCT07260916
View directory record Official record
Conditions
Basal Ganglia Intracerebral Hemorrhage
Interventions
Endoscopic ICH evacuation, Standard Medical Treatment
Procedure · Other
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 80 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
44
States / cities
Phoenix, Arizona • Tucson, Arizona • Los Angeles, California + 33 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 21, 2026, 2:05 PM EDT
Completed Phase 4 Interventional Results available

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

NCT00081497
View directory record Official record
Conditions
Fabry Disease
Interventions
agalsidase beta
Biological
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
16 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
18
States / cities
Birmingham, Alabama • Los Angeles, California • San Francisco, California + 15 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2015 · Synced Oct 21, 2026, 2:05 PM 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 4, 2008 · Synced Oct 21, 2026, 2:05 PM EDT
Active, not recruiting Not applicable Interventional

A Study to Evaluate White Matter Hyperintensities in Migraine

NCT05112289
View directory record Official record
Conditions
Migraine, Small Vessel Ischemic Disease, Multiple Sclerosis
Interventions
Brain Neuroimaging
Diagnostic Test
Lead sponsor
Mayo Clinic
Other
Eligibility
20 Years to 59 Years · Female only
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 21, 2026, 2:05 PM EDT
Completed Phase 2 Interventional Results available

Replagal Enzyme Replacement Therapy for Children With Fabry Disease

NCT00084084
View directory record Official record
Conditions
Fabry Disease
Interventions
Agalsidase alfa
Drug
Lead sponsor
Shire
Industry
Eligibility
7 Years to 17 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2011
U.S. locations
13
States / cities
Tucson, Arizona • Palm Beach Gardens, Florida • Lake Charles, Louisiana + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2021 · Synced Oct 21, 2026, 2:05 PM EDT
Recruiting No phase listed Observational

Clinical Transcriptomics in Systemic Vasculitis (CUTIS)

NCT03004326
View directory record Official record
Conditions
Cryoglobulinemic Vasculitis (CV), Drug-induced Vasculitis, Eosinophilic Granulomatosis With Polyangiitis (EGPA), IgA Vasculitis, Isolated Cutaneous Vasculitis, Granulomatosis With Polyangiitis (GPA), Microscopic Polyangiitis (MPA), Polyarteritis Nodosa (PAN), Urticarial Vasculitis, Vasculitis
Interventions
Not listed
Lead sponsor
Peter Merkel
Other
Eligibility
5 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2028
U.S. locations
8
States / cities
Los Angeles, California • Boston, Massachusetts • Rochester, Minnesota + 5 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2026 · Synced Oct 21, 2026, 2:05 PM EDT