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ClinicalTrials.gov public records Last synced Oct 21, 2026, 2:41 PM EDT

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Showing 121–144 of 200 matching trials from the live ClinicalTrials.gov search.
Conditions
CADASIL
Interventions
Not listed
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
New York, New York
Completed Phase 3 Interventional Results available

Belimumab in Remission of VASculitis

NCT01663623
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
Completed Phase 4 Interventional Accepts healthy volunteers Results available

Responses to Influenza Vaccine in Patients With Mitochondrial Disorders (MELAS)

NCT01831934
Conditions
MELAS Syndrome
Interventions
Fluzone®
Biological
Lead sponsor
Stanford University
Other
Eligibility
13 Years to 65 Years
Enrollment
22 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2012
U.S. locations
1
States / cities
Stanford, California
Conditions
Cerebrovascular Disease, Ischemic Stroke, Transient Ischemic Attack, Intracerebral Hemorrhage, Aneurysmal Subarachnoid Hemorrhage, Vascular Dementia, Anoxic Brain Injury, Unruptured Intracranial Aneurysm, Carotid Artery Stenosis Symptomatic, Asymptomatic Carotid Artery Stenosis, Non-Aneurysmal Perimesencephalic Subarachnoid Haemorrhage, Cerebral Venous Thrombosis, Moyamoya Disease, Fibromuscular Dysplasia, Subarachnoid Hemorrhage, Leukoaraiosis, Arteriovenous Fistula, Reversible Cerebral Vasoconstriction Syndrome, CADASIL
Interventions
Not listed
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
72 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
1
States / cities
Jacksonville, Florida
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
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, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
educational intervention
Other
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years to 120 Years
Enrollment
583 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
4
States / cities
Detroit, Michigan • Cleveland, Ohio • Philadelphia, Pennsylvania
Recruiting No phase listed Observational

Vasculitis Pregnancy Registry

NCT02593565
Conditions
Vasculitis, Behcet's Disease, CNS Vasculitis, Cryoglobulinemic Vasculitis, Eosinophilic Granulomatosis With Polyangiitis (EGPA), Churg-Strauss Syndrome (CSS), Granulomatosis With Polyangiitis (GPA), Wegener's Granulomatosis, IgA Vasculitis, Henoch-Schoenlein Purpura (HSP), Microscopic Polyangiitis (MPA), Polyarteritis Nodosa (PAN), Takayasu Arteritis (TAK), Urticarial Vasculitis, Systemic Vasculitis
Interventions
Online questionnaires
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years to 50 Years · Female only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2027
U.S. locations
1
States / cities
Tampa, Florida
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
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
Cancer
Interventions
entinostat
Drug
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2008
U.S. locations
3
States / cities
Bethesda, Maryland
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
Conditions
Cancer
Interventions
Fentanyl sublingual spray, Placebo
Drug
Lead sponsor
INSYS Therapeutics Inc
Industry
Eligibility
18 Years and older
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2010
U.S. locations
1
States / cities
Chandler, Arizona
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
Fabry Disease, Neuropathic Pain
Interventions
Gabapentin, placebo
Drug
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2014
U.S. locations
1
States / cities
Minneapolis, Minnesota
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
Conditions
Leukodystrophy, White Matter Disease, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts 1, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus-Merzbacher-Like Disease, 1, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Up to 18 Years
Enrollment
236 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study - Mitochondrial Disease

NCT01532791
Conditions
MELAS or m.3243 A>G Mitochondrial DNA Mutation Carrier
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
4 Years and older
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2026
U.S. locations
1
States / cities
New York, New York
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
Conditions
Brain and Central Nervous System Tumors, Breast Cancer, Gastrointestinal Carcinoid Tumor, Islet Cell Tumor, Lung Cancer, Lymphoma, Melanoma (Skin), Neoplastic Syndrome
Interventions
yttrium Y 90-edotreotide
Radiation
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
Tampa, Florida
Not listed No phase listed Observational Accepts healthy volunteers

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

NCT03812042
Conditions
Lysosomal Storage Diseases
Interventions
Enzyme assay and molecular sequencing
Diagnostic Test
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
Not listed
Enrollment
100,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2019
U.S. locations
1
States / cities
Fairfax, Virginia
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
Conditions
Autoimmune Diseases, Non-small Cell Lung Cancer, Rheumatoid Arthritis, Psoriasis, Giant Cell Arteritis, Polymyalgia Rheumatica, Systemic Lupus Erythematosus, Crohn Disease, Multiple Sclerosis, Ulcerative Colitis
Interventions
Nivolumab
Drug
Lead sponsor
Alliance Foundation Trials, LLC.
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
9
States / cities
Chicago, Illinois • Boston, Massachusetts • Ann Arbor, Michigan + 6 more
Conditions
Eosinophilic Granulomatosis With Polyangiitis (Churg-Strauss) (EGPA), Churg-Strauss Syndrome (CSS), Granulomatosis With Polyangiitis (Wegener's) (GPA), Wegener Granulomatosis (WG), Microscopic Polyangiitis (MPA), ANCA-Associated Vasculitis (AAV), Vasculitis
Interventions
Online questionnaire
Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older · Female only
Enrollment
280 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
2
States / cities
Tampa, Florida • Philadelphia, Pennsylvania
Conditions
Cancer
Interventions
cyproheptadine hydrochloride, placebo
Drug · Other
Lead sponsor
University of South Florida
Other
Eligibility
2 Years to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
14
States / cities
Long Beach, California • Hartford, Connecticut • Wilmington, Delaware + 10 more