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ClinicalTrials.gov public records Last synced Oct 15, 2026, 4:20 PM EDT

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Showing 289–312 of 1,146 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 3 Interventional Results available

Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

NCT05089656
View directory record Official record
Conditions
Type 2 Spinal Muscular Atrophy
Interventions
OAV101, Sham control
Genetic · Procedure
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 17 Years
Enrollment
126 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
6
States / cities
Farmington, Connecticut • Chicago, Illinois • Strasburg, Pennsylvania + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 15, 2026, 4:20 PM EDT
Recruiting Phase 2Phase 3 Interventional Accepts healthy volunteers

A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset AD Caused by a Genetic Mutation

NCT05552157
View directory record Official record
Conditions
Alzheimers Disease, Dementia, Alzheimers Disease, Familial
Interventions
Remternetug (SC), Matching Placebo (Remternetug)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
280 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2034
U.S. locations
13
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Oct 15, 2026, 4:20 PM EDT
Completed Phase 2 Interventional Results available

Bevacizumab in Treating Patients With Recurrent or Progressive Meningiomas

NCT01125046
View directory record Official record
Conditions
Acoustic Schwannoma, Adult Anaplastic Meningioma, Adult Ependymoma, Adult Grade I Meningioma, Adult Grade II Meningioma, Adult Meningeal Hemangiopericytoma, Adult Papillary Meningioma, Neurofibromatosis Type 1, Neurofibromatosis Type 2, Recurrent Adult Brain Tumor
Interventions
bevacizumab
Biological
Lead sponsor
Northwestern University
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2018
U.S. locations
5
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2021 · Synced Oct 15, 2026, 4:20 PM EDT
Completed Not applicable Interventional

Investigating Circadian Rhythms in Youth With Persistent Tic Disorders

NCT03508245
View directory record Official record
Conditions
Tourette's Disorder
Interventions
Wearable short wavelength light therapy
Other
Lead sponsor
University of California, Los Angeles
Other
Eligibility
13 Years to 17 Years
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Dec 12, 2023 · Synced Oct 15, 2026, 4:20 PM EDT
Terminated No phase listed Observational

Observational Study to Assess Natural History in Cockayne Syndrome Patients

NCT00985413
View directory record Official record
Conditions
Cockayne Syndrome
Interventions
Not listed
Lead sponsor
DNage B.V.
Industry
Eligibility
1 Year to 11 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
2
States / cities
Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 23, 2011 · Synced Oct 15, 2026, 4:20 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Dominantly Inherited Alzheimer Network (DIAN)

NCT00869817
View directory record Official record
Conditions
Alzheimer's Disease
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
700 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2031
U.S. locations
8
States / cities
Jacksonville, Florida • Indianapolis, Indiana • Boston, Massachusetts + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 15, 2026, 4:20 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers Results available

Study of ALXN1840 on the Metabolism of a CYP2B6 Substrate in Healthy Participants

NCT04526210
View directory record Official record
Conditions
Wilson Disease
Interventions
ALXN1840, Bupropion Hydrochloride
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Aug 31, 2023 · Synced Oct 15, 2026, 4:20 PM EDT
Completed Phase 4 Interventional Results available

Effects of Ondansetron in Obsessive-compulsive and Tic Disorders

NCT03239210
View directory record Official record
Conditions
Obsessive-Compulsive Disorder, Tic Disorders, Tourette Syndrome
Interventions
Ondansetron, Placebo
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years to 60 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 3, 2024 · Synced Oct 15, 2026, 4:20 PM EDT
Recruiting No phase listed Observational

A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors

NCT06623890
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Omaveloxolone
Drug
Lead sponsor
Biogen
Industry
Eligibility
16 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
4
States / cities
Los Angeles, California • Aurora, Colorado • Gainesville, Florida + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced Oct 15, 2026, 4:20 PM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic Pain

NCT01633008
View directory record Official record
Conditions
Neurofibromatosis
Interventions
ACT Workshop
Behavioral
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years to 99 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 30, 2019 · Synced Oct 15, 2026, 4:20 PM EDT
Completed Not applicable Interventional Results available

Repetitive Transcranial Magnetic Stimulation to Reduce Tics

NCT01258790
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Sham Repetitive Transcranial Magnetic Stimulation, Active Repetitive Transcranial Magnetic Stimulation
Device
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
10 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 1, 2014 · Synced Oct 15, 2026, 4:20 PM EDT
Completed No phase listed Observational

Ketogenic Diet in Lafora Disease

NCT00007124
View directory record Official record
Conditions
Lafora Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 15, 2026, 4:20 PM EDT
Completed Phase 4 Interventional Results available

Study to Assess the Efficacy and Safety of Raxone in LHON Patients

NCT02774005
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy (LHON)
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
9
States / cities
Phoenix, Arizona • Palo Alto, California • Stanford, California + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2023 · Synced Oct 15, 2026, 4:20 PM EDT
Recruiting No phase listed Observational

Study of the Disease Process of Lymphangioleiomyomatosis

NCT00001465
View directory record Official record
Conditions
Lung Disease, Pneumothorax, Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
Toshibia Aquilion One CT
Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
16 Years to 100 Years
Enrollment
2,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1995
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 15, 2026, 4:20 PM EDT
Completed No phase listed Observational

Using Positron Emission Tomography to Predict Intracranial Tumor Growth in Neurofibromatosis Type II Patients

NCT01222728
View directory record Official record
Conditions
Neoplasms, Nervous System Disease, Vestibular Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 50 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 15, 2026, 4:20 PM EDT
No Longer Available No phase listed Expanded access

3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)

NCT02012933
View directory record Official record
Conditions
Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenia (CM)
Interventions
3,4-diaminopyridine
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
2 Years and older
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 11, 2019 · Synced Oct 15, 2026, 4:20 PM EDT
Terminated Phase 3 Interventional Results available

Efficacy and Safety Study of Fosmetpantotenate (RE-024) in PKAN Participants

NCT03041116
View directory record Official record
Conditions
Pantothenate Kinase-Associated Neurodegeneration
Interventions
Fosmetpantotenate, Placebo
Drug
Lead sponsor
Travere Therapeutics, Inc.
Industry
Eligibility
6 Years to 65 Years
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
9
States / cities
Irvine, California • Washington D.C., District of Columbia • Miami, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2021 · Synced Oct 15, 2026, 4:20 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

NCT05481879
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
DYNE-101, Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
8
States / cities
Stanford, California • Gainesville, Florida • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 15, 2026, 4:20 PM EDT
Terminated Not applicable Interventional Results available

Thalamic Deep Brain Stimulation for the Treatment of Refractory Tourette Syndrome

NCT01817517
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Medtronic Activa Deep Brain Stimulation System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
15 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2023
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 15, 2026, 4:20 PM EDT
Completed Phase 4 Interventional Results available

Open Label Trial of Aripiprazole in Children and Adolescents With Tourette's Disorder

NCT00241176
View directory record Official record
Conditions
Tourette's Syndrome, Tic Disorders
Interventions
Aripiprazole
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
7 Years to 18 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 9, 2016 · Synced Oct 15, 2026, 4:20 PM EDT
Completed Phase 2 Interventional

Premanifest Huntington's Disease: Creatine Safety & Tolerability Extension Study

NCT01411150
View directory record Official record
Conditions
Huntington's Disease
Interventions
Creatine Monohydrate
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
26 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Charlestown, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 10, 2014 · Synced Oct 15, 2026, 4:20 PM EDT
Not listed Not applicable Interventional

Study of an Intervention to Improve Problem List Accuracy and Use

NCT01105923
View directory record Official record
Conditions
Attention Deficit Disorder With Hyperactivity, Asthma, COPD, Breast Cancer, Coronary Artery Disease, Congestive Heart Failure, Diabetes, Glaucoma, Hemophilia, Hypertension, Hyperthyroidism, Hypothyroidism, Myasthenia Gravis, Osteoporosis, Osteopenia, Renal Failure, Renal Insufficiency, Sickle Cell Disease, Stroke
Interventions
MAPLE
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2017
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 2, 2015 · Synced Oct 15, 2026, 4:20 PM EDT
Completed Phase 1 Interventional

Safety Study of TPI-287 to Treat CBS and PSP

NCT02133846
View directory record Official record
Conditions
Primary Four Repeat Tauopathies (4RT), Corticobasal Syndrome (CBS), Progressive Supranuclear Palsy (PSP), Corticobasal Degeneration (CBD)
Interventions
TPI 287 2 mg/m2, TPI-287 20 mg/m2, Placebo, TPI-287 6.3 mg/m2
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
50 Years to 85 Years
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
2
States / cities
Birmingham, Alabama • San Francisco, California
Source: ClinicalTrials.gov public record
Updated Apr 15, 2020 · Synced Oct 15, 2026, 4:20 PM EDT
Terminated Phase 4 Interventional Results available

The Role of F-18 Florbetapir in the Early Detection of Cardiac Amyloidosis

NCT03040427
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
F-18 florbetapir
Drug
Lead sponsor
The Cleveland Clinic
Other
Eligibility
18 Years to 95 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 27, 2021 · Synced Oct 15, 2026, 4:20 PM EDT