Independent directory Public ClinicalTrials.gov records United States
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Showing 1–24 of 200 matching trials from the live ClinicalTrials.gov search.
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Terminated Phase 1Phase 2 Interventional

A Fabry Disease Gene Therapy Study

NCT04040049
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
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
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
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
migalastat hydrochloride, agalsidase
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years to 74 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
8
States / cities
Los Angeles, California • Aurora, Colorado • Decatur, Georgia + 5 more
Conditions
Fabry Disease
Interventions
migalastat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Recruiting Not applicable Interventional

Neurologic Stem Cell Treatment Study

NCT02795052
Conditions
Neurologic Disorders, Nervous System Diseases, Neurodegenerative Diseases, Neurological Disorders, Stroke, Traumatic Brain Injury, Cadasil, Chronic Traumatic Encephalopathy, Cerebral Infarction, Cerebral Ischemia, Cerebral Stroke, Cerebral Hemorrhage, Parkinson, Multi-System Degeneration, MSA - Multiple System Atrophy, Progressive Supranuclear Palsy, ALS, Amyotrophic Lateral Sclerosis, Neuropathy, Diabetic Neuropathies, Alzheimer Disease, Dementia, Frontotemporal Dementia, Lewy Body Disease, Cognitive Impairment, Lewy Body Variant of Alzheimer Disease
Interventions
Intravenous and Intranasal BMSC
Procedure
Lead sponsor
MD Stem Cells
Industry
Eligibility
18 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
2
States / cities
Westport, Connecticut • Coral Springs, Florida
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
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
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
Conditions
Fabry Disease
Interventions
4D-310
Biological
Lead sponsor
4D Molecular Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2030
U.S. locations
4
States / cities
La Jolla, California • Atlanta, Georgia • Pittsburgh, Pennsylvania + 1 more
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
Conditions
Behcet's Disease, Churg-Strauss Syndrome, Giant Cell Arteritis, Wegener Granulomatosis, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu's Arteritis
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
Not listed
Enrollment
198 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Tampa, Florida
Conditions
Eosinophilic Granulomatosis With Polyangiitis (EGPA), Churg-Strauss Syndrome (CSS), Giant Cell Arteritis, Granulomatosis With Polyangiitis, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu Arteritis
Interventions
Naltrexone Hydrochloride, Placebo Comparator
Drug · Other
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
6
States / cities
Boston, Massachusetts • Rochester, Minnesota • Cleveland, Ohio + 3 more
Conditions
Fabry Disease
Interventions
PRX-102 (pegunigalsidase alfa), agalsidase beta
Biological
Lead sponsor
Protalix
Industry
Eligibility
18 Years to 60 Years
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
15
States / cities
Birmingham, Alabama • Phoenix, Arizona • Orange, California + 11 more
Conditions
Lupus Erythematosus, Systemic Lupus Erythematosus, SLE (Systemic Lupus), Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis, Granulomatous Polyangiitis, Microscopic Polyangiitis
Interventions
SC291
Biological
Lead sponsor
Sana Biotechnology
Industry
Eligibility
18 Years to 75 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
5
States / cities
Aurora, Colorado • Atlanta, Georgia • Baltimore, Maryland + 2 more
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
No Longer Available No phase listed Expanded access

EPI-743 for Mitochondrial Respiratory Chain Diseases

NCT01370447
Conditions
Mitochondrial Diseases
Interventions
EPI-743
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
1 Year and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
16
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 12 more
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
Recruiting No phase listed Observational

VCRC Tissue Repository

NCT02967068
Conditions
Aortitis, Cutaneous Vasculitis, Eosinophilic Granulomatosis With Polyangiitis, Giant Cell Arteritis, Granulomatosis With Polyangiitis (Wegener's), Henoch-Schonlein Purpura, IgA Vasculitis, Microscopic Polyangiitis, Polyarteritis Nodosa, Takayasu Arteritis, Churg-Strauss Syndrome
Interventions
Not listed
Lead sponsor
University of Pennsylvania
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2028
U.S. locations
6
States / cities
Boston, Massachusetts • Cleveland, Ohio • Philadelphia, Pennsylvania + 2 more
Active, not recruiting No phase listed Observational Accepts healthy volunteers

BRINK (BRain In Kidney Disease) Memory Study 2.0

NCT04975464
Conditions
Chronic Kidney Diseases, Dementia, Dementia, Vascular, Stroke, Alzheimer Disease, Cognitive Decline, Cognitive Impairment, Cognitive Dysfunction, Kidney Diseases, Diabetes, Cerebral Atrophy, Small Vessel Ischemic Disease
Interventions
Not listed
Lead sponsor
Anne Murray
Other
Eligibility
50 Years and older
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2026
U.S. locations
2
States / cities
Minneapolis, Minnesota
Conditions
Brain and Central Nervous System Tumors, Breast Cancer, Kidney Cancer, Lung Cancer, Lymphoma, Melanoma (Skin), Ovarian Cancer, Small Intestine Cancer, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
carboxyamidotriazole, paclitaxel
Drug
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1994 – 2006
U.S. locations
2
States / cities
Bethesda, Maryland
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
Brain and Central Nervous System Tumors, Lymphoma, Lymphoproliferative Disorder, Small Intestine Cancer
Interventions
autologous immunoglobulin idiotype-KLH conjugate vaccine, sargramostim, methotrexate, thiotepa, radiation therapy
Biological · Drug · Radiation
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 120 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2008
U.S. locations
1
States / cities
Dallas, Texas