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ClinicalTrials.gov public records Last synced Oct 4, 2026, 10:10 AM EDT

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Showing 25–48 of 344 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Central Nervous System Vascular Malformation Clear all
Recruiting Phase 2 Interventional

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of RLS-0071 in Newborns With Moderate or Severe Hypoxic-Ischemic Encephalopathy Undergoing Therapeutic Hypothermia

NCT05778188
View directory record Official record
Conditions
Hypoxic-Ischemic Encephalopathy
Interventions
RLS-0071, Placebo
Drug
Lead sponsor
ReAlta Life Sciences, Inc.
Industry
Eligibility
Up to 10 Hours
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
15
States / cities
Little Rock, Arkansas • Orange, California • San Diego, California + 11 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 4, 2026, 10:10 AM EDT
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 4, 2026, 10:10 AM EDT
Completed No phase listed Observational

VCRC Patient Contact Registry Patient-Reported Data Validation Study

NCT02190942
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated Oct 16, 2019 · Synced Oct 4, 2026, 10:10 AM EDT
Recruiting Phase 1 Interventional

Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels

NCT05241262
View directory record Official record
Conditions
Mitochondrial Disease
Interventions
N-Acetylcysteine
Drug
Lead sponsor
Michio Hirano, MD
Other
Eligibility
18 Years to 80 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 23, 2026 · Synced Oct 4, 2026, 10:10 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 6, 2012 · Synced Oct 4, 2026, 10:10 AM EDT
Recruiting No phase listed Observational

Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disorders

NCT07493096
View directory record Official record
Conditions
Neurodevelopmental Disorders, Neurodevelopmental Disorders (NDD), Neurodevelopmental Disorders and Developmental Abnormalities, Developmental Delay (Disorder), Cerebral Palsy (CP), Cerebral Palsy Hemiparetic Cerebral Palsy Spasticity Gait Disorders, Neurologic Postural Balance Impairment, Cerebral Palsy Infantile, Cerebral Palsy Spastic Hemiplegic, Cerebral Palsy, Dyskinetic, Autism Spectrum Disorder, Autism Spectrum Disorder (ASD, Hypoxic Ischemic Encephalopathy, Hypoxic Ischemic Encephalopathy (HIE), Traumatic Brain Injury (TBI), Sensory Processing Disorder, Chromosomal Abnormalities, Genetic Disorders, Down Syndrome (Trisomy 21), Fragile X Syndrome (FXS), RETT Syndrome With Proven MECP2 Mutation, Williams Syndrome, 22q11.2 Deletion Syndrome, Sensorimotor Integration
Interventions
Not listed
Lead sponsor
Healing Hope International
Other
Eligibility
4 Years to 12 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2036
U.S. locations
1
States / cities
The Woodlands, Texas
Source: ClinicalTrials.gov public record
Updated Mar 25, 2026 · Synced Oct 4, 2026, 10:10 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 9, 2021 · Synced Oct 4, 2026, 10:10 AM EDT
Completed No phase listed Observational

Genetic and Environmental Determinants That Control Metabolism in Pulmonary Hypertension

NCT02594917
View directory record Official record
Conditions
Iron-sulfur Cluster Deficiency, Pulmonary Hypertension, Friedreich Ataxia
Interventions
Right Heart Catheterization, Blood draw
Procedure · Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
18 Years to 60 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 22, 2023 · Synced Oct 4, 2026, 10:10 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

NuroSleeve Powered Brace & Stimulation System to Restore Arm Function

NCT04798378
View directory record Official record
Conditions
Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies, Arthrogryposis, Spinal Cord Injuries, Charcot-Marie-Tooth, Stroke, Weakness of Extremities as Sequela of Stroke, Weakness Due to Upper Motor Neuron Dysfunction, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, Arm Paralysis, Cerebral Palsy
Interventions
Neurosleeve
Device
Lead sponsor
Thomas Jefferson University
Other
Eligibility
4 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
2
States / cities
Wilmington, Delaware • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 9, 2025 · Synced Oct 4, 2026, 10:10 AM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Tissue Sample Study for Mitochondrial Disorders

NCT01803906
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Disease, Melas, Kearns Sayer, NARP, MNGIE, LHON, Mitochondrial Depletion Syndrome, Leigh's Disease
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
6,900 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 4, 2026, 10:10 AM EDT
Completed No phase listed Observational

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

NCT04804566
View directory record Official record
Conditions
Fabry Disease
Interventions
This is a non-interventional study
Other
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023
U.S. locations
1
States / cities
Eagan, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 3, 2024 · Synced Oct 4, 2026, 10:10 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 19, 2013 · Synced Oct 4, 2026, 10:10 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease

NCT06065605
View directory record Official record
Conditions
Fabry Disease
Interventions
Not listed
Lead sponsor
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Other
Eligibility
18 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2025
U.S. locations
2
States / cities
Rockville, Maryland • Fairfax, Virginia
Source: ClinicalTrials.gov public record
Updated Oct 4, 2023 · Synced Oct 4, 2026, 10:10 AM EDT
Completed Phase 4 Interventional Accepts healthy volunteers Results available

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

NCT01831934
View directory record Official record
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
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 4, 2026, 10:10 AM 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 4, 2026, 10:10 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 31, 2014 · Synced Oct 4, 2026, 10:10 AM 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 4, 2026, 10:10 AM EDT
Completed No phase listed Observational

Prediction of Brain Injury in Premature Infants

NCT02814383
View directory record Official record
Conditions
Intraventricular Hemorrhage, Periventricular Leukomalacia, Brain Injury, Extreme Prematurity
Interventions
Brain Monitoring Platform
Other
Lead sponsor
Baylor College of Medicine
Other
Eligibility
0 Hours to 12 Hours
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2025
U.S. locations
3
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 4, 2026, 10:10 AM EDT
Completed Phase 2 Interventional Results available

Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable Meningioma

NCT00589784
View directory record Official record
Conditions
CNS Cancer, Meningioma, Intracranial Hemangiopericytoma, Hemangioblastoma, Neurofibromatosis
Interventions
Sunitinib
Drug
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
6
States / cities
Boston, Massachusetts • Basking Ridge, New Jersey • Commack, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 25, 2016 · Synced Oct 4, 2026, 10:10 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

NCT01694940
View directory record Official record
Conditions
Mitochondrial Disorders, Mitochondrial Genetic Disorders, Mitochondrial Diseases, Disorder of Mitochondrial Respiratory Chain Complexes, Deletion and Duplication of Mitochondrial DNA
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2026
U.S. locations
16
States / cities
San Diego, California • Stanford, California • Aurora, Colorado + 12 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 4, 2026, 10:10 AM EDT
Completed No phase listed Observational

Effect of Ischemic Strokes on Recovery From Intracerebral Hemorrhages

NCT01417117
View directory record Official record
Conditions
Hemorrhage; Intracerebral, Nontraumatic, Ischemic Strokes, Diffusion Weighted Imaging Lesions
Interventions
Not listed
Lead sponsor
Rush University Medical Center
Other
Eligibility
19 Years to 79 Years
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Oct 20, 2017 · Synced Oct 4, 2026, 10:10 AM EDT
Completed Phase 3 Interventional Results available

Silent Cerebral Infarct Transfusion Multi-Center Clinical Trial

NCT00072761
View directory record Official record
Conditions
Sickle Cell Anemia, Stroke
Interventions
transfusion therapy
Procedure
Lead sponsor
Washington University School of Medicine
Other
Eligibility
5 Years to 14 Years
Enrollment
196 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2013
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Feb 8, 2016 · Synced Oct 4, 2026, 10:10 AM EDT
Not yet recruiting Not applicable Interventional

iBCI Optimization for Veterans With Paralysis

NCT05470478
View directory record Official record
Conditions
Spinal Cord Injury, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked-in Syndrome, Muscular Dystrophy
Interventions
Mobile neural decoding platform (mobile iBCI)
Device
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
18 Years to 80 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Providence, Rhode Island
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Oct 4, 2026, 10:10 AM EDT