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ClinicalTrials.gov public records Last synced Jul 23, 2026, 10:03 AM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Central Nervous System Vascular Malformation Clear all
Not listed No phase listed Observational Accepts healthy volunteers

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

NCT02000310
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Feb 22, 2021 · Synced Jul 23, 2026, 10:03 AM EDT
Completed No phase listed Observational

International Ataxia Rating Scale in Younger Patients

NCT01942850
View directory record Official record
Conditions
Ataxia Telangiectasia
Interventions
Not listed
Lead sponsor
Quince Therapeutics S.p.A.
Industry
Eligibility
6 Years to 18 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Dec 30, 2014 · Synced Jul 23, 2026, 10:03 AM EDT
Completed Phase 1Phase 2 Interventional

Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD).

NCT06051448
View directory record Official record
Conditions
Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MERRF
Interventions
Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN)
Behavioral
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 28, 2024 · Synced Jul 23, 2026, 10:03 AM EDT
Completed Phase 2 Interventional

A Phase 2b Study of Zagociguat in Patients With MELAS

NCT06402123
View directory record Official record
Conditions
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
Interventions
zagociguat 15mg, zagociguat 30mg, Placebo
Drug
Lead sponsor
Tisento Therapeutics
Industry
Eligibility
18 Years to 75 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
10
States / cities
La Jolla, California • Aurora, Colorado • Atlanta, Georgia + 5 more
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 23, 2026, 10:03 AM EDT
Terminated Phase 3 Interventional Results available

Stroke With Transfusions Changing to Hydroxyurea

NCT00122980
View directory record Official record
Conditions
Hemochromatosis, Cerebrovascular Accident, Anemia, Sickle Cell, Hematologic Diseases
Interventions
Red Cell Transfusions, Iron Chelation, Hydroxyurea, Phlebotomy
Procedure · Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
5 Years to 18 Years
Enrollment
134 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
28
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Jacksonville, Florida + 23 more
Source: ClinicalTrials.gov public record
Updated Jan 17, 2013 · Synced Jul 23, 2026, 10:03 AM EDT
Completed Phase 2 Interventional

Alpha-Galactosidase A Replacement Therapy for Fabry Disease

NCT00048906
View directory record Official record
Conditions
Fabry Disease
Interventions
DRX005B
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 23, 2026, 10:03 AM EDT
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, 10:03 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Optimizing BCI-FIT: Brain Computer Interface - Functional Implementation Toolkit

NCT04468919
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Brainstem Stroke, Muscular Dystrophies, Parkinson's Disease and Parkinsonism, Multiple System Atrophy, Brain Tumor Adult, Spinal Cord Injuries, Locked-in Syndrome
Interventions
BCI-FIT multi-modal access, BCI-FIT adaptive signal modeling, BCI-FIT active querying, BCI-FIT language modeling
Behavioral
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years to 89 Years
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated May 15, 2025 · Synced Jul 23, 2026, 10:03 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Risk Factors, Costs, and Impacts of ED Boarding

NCT07532564
View directory record Official record
Conditions
Stroke (CVA) or TIA, Diabetes, Asthma (Diagnosis), COPD (Chronic Obstructive Pulmonary Disease), Sepsis, Hypertension, Heart Failure, Pneumonia, Urinary Tract Infection (Diagnosis), Chest Pain, Psychiatric Disorder, Sickle Cell Disease (SCD)
Interventions
Not listed
Lead sponsor
University of Maryland, Baltimore
Other
Eligibility
18 Years and older
Enrollment
30,486 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Jul 23, 2026, 10:03 AM EDT
Recruiting Not applicable Interventional

Interfacing With NeuroTechnology to Expand Neural Throughput (INTENT)

NCT07521930
View directory record Official record
Conditions
Tetraplegia/Tetraparesis, Amyotrophic Lateral Sclerosis (ALS), Muscular Disorders, Atrophic, Brain Stem Stroke, Spinal Cord Injuries (SCI), Muscular Dystrophies, Muscular Atrophy, Progressive, Pontine Hemorrhage, Pontine Infarction
Interventions
INTENT Neural Interface System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Jul 23, 2026, 10:03 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

NCT01931644
View directory record Official record
Conditions
All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions
Not listed
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 100 Years
Enrollment
17,667 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 17, 2024 · Synced Jul 23, 2026, 10:03 AM EDT
Not listed Phase 2 Interventional

MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia

NCT04887311
View directory record Official record
Conditions
Ataxia Telangiectasia Louis-Bar, Ataxia Telangiectasia in Children, Ataxia Telangiectasia
Interventions
MBM-01
Drug
Lead sponsor
Matrix Biomed, Inc.
Industry
Eligibility
4 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 16, 2021 · Synced Jul 23, 2026, 10:03 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, 10:03 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, 10:03 AM EDT
Completed No phase listed Observational

Quality of Life in Younger Leukemia and Lymphoma Survivors

NCT00956475
View directory record Official record
Conditions
Anxiety Disorder, Cancer Survivor, Fatigue, Leukemia, Long-term Effects Secondary to Cancer Therapy in Adults, Lymphoma, Lymphoproliferative Disorder, Pain, Psychosocial Effects of Cancer and Its Treatment, Small Intestine Cancer
Interventions
telephone-based intervention, questionnaire administration, assessment of therapy complications, fatigue assessment and management, psychosocial assessment and care, quality-of-life assessment
Behavioral · Other · Procedure
Lead sponsor
Case Comprehensive Cancer Center
Other
Eligibility
18 Years to 35 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 23, 2011 · Synced Jul 23, 2026, 10:03 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, 10:03 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, 10:03 AM EDT
Completed Phase 1Phase 2 Interventional

Doxorubicin Hydrochloride Liposome and Bortezomib in Treating Patients With Refractory Hematologic Cancer or Malignant Solid Tumor or Metastatic Breast Cancer

NCT00237627
View directory record Official record
Conditions
Breast Cancer, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Ovarian Cancer, Unspecified Adult Solid Tumor, Protocol Specific
Interventions
PS-341, Doxil, Velcade
Drug
Lead sponsor
UNC Lineberger Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
107 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2010
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated May 17, 2012 · Synced Jul 23, 2026, 10:03 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, 10:03 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, 10:03 AM EDT
Recruiting Not applicable Interventional

Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia (BG-Speech-02)

NCT06094205
View directory record Official record
Conditions
Anarthria, Dysarthria, Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked-in Syndrome, Muscular Dystrophies
Interventions
BrainGate Neural Interface System
Device
Lead sponsor
Leigh R. Hochberg, MD, PhD.
Other
Eligibility
18 Years to 80 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Nov 30, 2025 · Synced Jul 23, 2026, 10:03 AM EDT
Completed Phase 2 Interventional Results available

Phase II Study of Everolimus (RAD001) in Children and Adults With Neurofibromatosis Type 2

NCT01419639
View directory record Official record
Conditions
Neurofibromatosis Type II
Interventions
Everolimus (RAD001) , Afinitor®
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
3 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 17, 2017 · Synced Jul 23, 2026, 10:03 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Complex Eye Movements in Parkinson's Disease and Related Movement Disorders

NCT04925622
View directory record Official record
Conditions
Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, Corticobasal Degeneration, Parkinsonian Disorders, Essential Tremor, Vascular Parkinsonism, Multiple System Atrophy, Parkinson Variant, Parkinsonian Syndrome, Huntington Disease
Interventions
Complex eye movement exam
Diagnostic Test
Lead sponsor
Saccadous, Inc.
Other
Eligibility
30 Years and older
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated May 3, 2022 · Synced Jul 23, 2026, 10:03 AM EDT