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ClinicalTrials.gov public records Last synced Oct 17, 2026, 4:54 AM EDT

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Showing 409–432 of 1,147 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Not applicable Interventional Results available

MN4000 for Treatment of CF and MND Patients in the Home Setting

NCT03797677
View directory record Official record
Conditions
Cystic Fibrosis, Motor Neuron Disease, Airway Clearance Impairment
Interventions
MN 4000
Device
Lead sponsor
Hill-Rom
Industry
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Aug 7, 2024 · Synced Oct 17, 2026, 4:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Biomarkers in Friedreich's Ataxia

NCT02497534
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Not listed
Lead sponsor
University of Florida
Other
Eligibility
6 Years to 70 Years
Enrollment
203 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2030
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Genetic Study of Familial and Sporadic ALS/Motor Neuron Disease, Miyoshi Myopathy and Other Neuromuscular Disorders

NCT01459302
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, PLS, Motor Neuron Disease, Lou Gehrigs Disease, Familial Disease, Amyotrophic Lateral Sclerosis, Sporadic, Muscular Dystrophy, Miyoshi Myopathy, Amyotrophic Lateral Sclerosis With Dementia
Interventions
Not listed
Lead sponsor
University of Massachusetts, Worcester
Other
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2024
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 17, 2026, 4:54 AM EDT
Terminated Phase 2 Interventional Results available

SF1126 in Recurrent or Progressive SCCHN and Mutations in PIK3CA Gene and/or PI-3 Kinase Pathway Genes

NCT02644122
View directory record Official record
Conditions
Metastatic Squamous Neck Cancer With Occult Primary Squamous Cell Carcinoma
Interventions
SF1126
Drug
Lead sponsor
Ezra Cohen
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated Jul 11, 2018 · Synced Oct 17, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Natural History Study of Patients With Neurofibromatosis Type 2

NCT00598351
View directory record Official record
Conditions
Neurofibromatosis
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
8 Years to 75 Years
Enrollment
269 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Completed Not applicable Interventional

Study Evaluating Treatment of Forefoot Pain Related to Nerve Entrapment Using the Cryo-Touch III Device

NCT01753778
View directory record Official record
Conditions
Entrapment Neuropathy
Interventions
Cryo-Touch III Device
Device
Lead sponsor
Pacira Pharmaceuticals, Inc
Industry
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
2
States / cities
Redwood City, California • Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Jan 29, 2014 · Synced Oct 17, 2026, 4:54 AM EDT
Completed No phase listed Observational

Examination of Clinical and Laboratory Abnormalities in Patients With Defective DNA Repair: Xeroderma Pigmentosum, Cockayne Syndrome, or Trichothiodystrophy

NCT00001813
View directory record Official record
Conditions
Cockayne Syndrome, Skin Neoplasms, Xeroderma Pigmentosum, Trichothiodystrophy Syndromes, Genodermatosis
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
6 Weeks to 100 Years
Enrollment
709 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety of Simvastatin in LAM and TSC

NCT02061397
View directory record Official record
Conditions
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Interventions
Simvastatin, Sirolimus Oral Product, Everolimus Oral Product
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older · Female only
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 25, 2020 · Synced Oct 17, 2026, 4:54 AM EDT
Active, not recruiting Phase 2Phase 3 Interventional

Impact of Deutetrabenazine on Functional Speech and Gait Dynamics in Huntington Disease

NCT04713982
View directory record Official record
Conditions
Huntington Disease
Interventions
Deutetrabenazine
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Recruiting Not applicable Interventional

Stem Cell Ophthalmology Treatment Study II

NCT03011541
View directory record Official record
Conditions
Retinal Disease, Age-Related Macular Degeneration, Retinitis Pigmentosa, Stargardt Disease, Optic Neuropathy, Nonarteritic Ischemic Optic Neuropathy, Optic Atrophy, Optic Nerve Disease, Glaucoma, Leber Hereditary Optic Neuropathy, Blindness, Vision Loss Night, Vision Loss Partial, Vision, Low, Retinopathy, Maculopathy, Macular Degeneration, Retina Atrophy
Interventions
Arm 1
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
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

US Benchmarking Clinical Study

NCT07215715
View directory record Official record
Conditions
HFpEF - Heart Failure With Preserved Ejection Fraction, HFmrEF, ATTR-CM (Transthyretin Amyloid Cardiomyopathy), Left Ventricular Hypertrophy, Cardiac Amyloidosis
Interventions
Not listed
Lead sponsor
eMyosound SAS
Industry
Eligibility
60 Years and older
Enrollment
150 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2026
U.S. locations
3
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 20, 2025 · Synced Oct 17, 2026, 4:54 AM EDT
Completed Phase 2 Interventional Results available

Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1

NCT01406873
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
Mexiletine, Placebo
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years to 80 Years
Enrollment
42 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jun 19, 2018 · Synced Oct 17, 2026, 4:54 AM EDT
Completed Phase 3 Interventional Results available

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

NCT04851873
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
OAV101
Genetic
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
Up to 17 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
2
States / cities
Boston, Massachusetts • St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 9, 2024 · Synced Oct 17, 2026, 4:54 AM EDT
Recruiting No phase listed Observational

Glioma Developmental and HyperActive Ras Tumor (DHART) Board

NCT05489783
View directory record Official record
Conditions
Glioma
Interventions
Not listed
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
7
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Group Drum-Based Music Therapy Intervention for Parkinson's Disease/Huntington's Disease

NCT05157074
View directory record Official record
Conditions
Parkinson Disease, Huntington Disease
Interventions
Group Drumming Music Therapy
Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 89 Years
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 30, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Terminated Phase 3 Interventional Results available

A Study to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Huntington's Disease

NCT05655520
View directory record Official record
Conditions
Huntington's Disease
Interventions
SAGE-718
Drug
Lead sponsor
Supernus Pharmaceuticals, Inc.
Industry
Eligibility
25 Years to 65 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
29
States / cities
Birmingham, Alabama • Little Rock, Arkansas • La Jolla, California + 25 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Oct 17, 2026, 4:54 AM EDT
Active, not recruiting No phase listed Observational

Natural History Study of Patients With Neurofibromatosis Type I

NCT00924196
View directory record Official record
Conditions
Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor, Plexiform Neurofibroma, Optic Glioma, Neurofibroma
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
4 Weeks and older
Enrollment
259 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

New Non-invasive Modalities for Assessing Retinal Structure and Function

NCT03475173
View directory record Official record
Conditions
Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion, Hemianopia, Leber Hereditary Optic Neuropathy, Acute Zonal Occult Outer Retinopathy
Interventions
LSFG-NAVI
Device
Lead sponsor
Randy Kardon
Other
Eligibility
18 Years to 99 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2028
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Recruiting Not applicable Interventional

Adapting RDAD for DS

NCT07425769
View directory record Official record
Conditions
Down Syndrome (DS), Alzheimer's Disease (AD)
Interventions
Remote Exercise Sessions, Caregiver Education Sessions
Behavioral
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
Not listed
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026
U.S. locations
1
States / cities
Kansas City, Kansas
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Completed Phase 2 Interventional Results available

N-acetylcysteine (NAC) for Children With Tourette Syndrome

NCT01172288
View directory record Official record
Conditions
Tourettes Syndrome, Tic
Interventions
N-Acetylcysteine (NAC), Placebo
Drug
Lead sponsor
Yale University
Other
Eligibility
8 Years to 17 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Feb 10, 2017 · Synced Oct 17, 2026, 4:54 AM EDT
Completed Not applicable Interventional

Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions

NCT02428673
View directory record Official record
Conditions
Osteopenia, Spinal Muscular Atrophy, Cerebral Palsy, Muscular Dystrophy, Spina Bifida, Rett Syndrome
Interventions
Assisted Standing Treatment Program
Other
Lead sponsor
Gillette Children's Specialty Healthcare
Other
Eligibility
3 Years to 14 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 17, 2026, 4:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis

NCT06034405
View directory record Official record
Conditions
Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation, ATTRV122I Amyloidosis, Cardiomyopathy, Hypertrophic
Interventions
Tc99-PYP or Tc99-HDMP Scan
Diagnostic Test
Lead sponsor
Columbia University
Other
Eligibility
50 Years and older
Enrollment
1,663 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2028
U.S. locations
6
States / cities
Beverly Hills, California • Boston, Massachusetts • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 17, 2026, 4:54 AM EDT
Completed Phase 2 Interventional Results available

A Pilot Study To Evaluate The Effects of Everolimus on Brain mTOR Activity and Cortical Hyperexcitability in TSC and FCD

NCT02451696
View directory record Official record
Conditions
Epilepsy, Tuberous Sclerosis Complex, Focal Cortical Dysplasia
Interventions
Everolimus
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
2 Years to 40 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 1, 2021 · Synced Oct 17, 2026, 4:54 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Retinal Imaging in Neurodegenerative Disease

NCT03233646
View directory record Official record
Conditions
Alzheimer's Disease, Mild Cognitive Impairment, Parkinson's Disease, Multiple Sclerosis, Huntington Disease, Lewy Body Dementia, Frontotemporal Dementia, Amyotrophic Lateral Sclerosis (ALS), APOE-4 Positive, Traumatic Brain Injury, Concussion, Post-Traumatic Stress Disorder, Down Syndrome, Neuro-Degenerative Disease, Normal Cognition
Interventions
Retinal and Choroidal Imaging
Device
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
2,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2026
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Oct 17, 2026, 4:54 AM EDT