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ClinicalTrials.gov public records Last synced Oct 21, 2026, 4:02 PM EDT

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Showing 721–744 of 1,148 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 2 Interventional

Thalamic Deep Brain Stimulation for Tourette Syndrome

NCT00311909
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Thalamic deep brain stimulation
Device
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated May 12, 2022 · Synced Oct 21, 2026, 4:02 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

NCT05394064
View directory record Official record
Conditions
AMN, AMN Gene Mutation, X-ALD
Interventions
SBT101 Treatment
Genetic
Lead sponsor
SwanBio Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 21, 2026, 4:02 PM EDT
Completed Phase 2 Interventional

Idebenone to Treat Friedreich's Ataxia

NCT00229632
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Idebenone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
9 Years to 17 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 18, 2019 · Synced Oct 21, 2026, 4:02 PM EDT
Terminated Phase 2 Interventional

PTC299 for Treatment of Neurofibromatosis Type 2

NCT00911248
View directory record Official record
Conditions
Neurofibromatosis 2
Interventions
PTC299
Drug
Lead sponsor
PTC Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 12, 2019 · Synced Oct 21, 2026, 4:02 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Intravitreal ERT to Prevent Retinal Disease Progression in Children With CLN2

NCT05152914
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis Type 2
Interventions
Cerliponase Alfa
Drug
Lead sponsor
David L Rogers, MD
Other
Eligibility
24 Months to 72 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 30, 2026 · Synced Oct 21, 2026, 4:02 PM EDT
Completed No phase listed Observational

Genetics of Obsessive-Compulsive Disorder

NCT00001548
View directory record Official record
Conditions
Obsessive Compulsive Disorder
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
18 Years and older
Enrollment
1,067 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 6, 2017 · Synced Oct 21, 2026, 4:02 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Pilot Study of Minocycline in Huntington's Disease

NCT00277355
View directory record Official record
Conditions
Huntington Disease
Interventions
minocycline, Matching placebo
Drug
Lead sponsor
Merit Cudkowicz
Network
Eligibility
18 Years and older
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2008
U.S. locations
11
States / cities
Birmingham, Alabama • Englewood, Colorado • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Apr 19, 2013 · Synced Oct 21, 2026, 4:02 PM EDT
Recruiting No phase listed Observational

A Study Of Deep Learning For Echo Analysis, Tracking, And Evaluation

NCT07308704
View directory record Official record
Conditions
Cardiomyopathy, Ischemic Heart Disease, Cardiac Amyloidosis
Interventions
Transthoracic Echocardiography (TTE)
Diagnostic Test
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
10,040,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 21, 2026, 4:02 PM 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 21, 2026, 4:02 PM EDT
Completed No phase listed Observational

Neurofibromatosis Type 1 (NF1) and Tibial Dysplasia

NCT00303368
View directory record Official record
Conditions
Neurofibromatosis Type 1, Tibial Dysplasia
Interventions
Not listed
Lead sponsor
Shriners Hospitals for Children
Other
Eligibility
Not listed
Enrollment
395 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2016
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Feb 25, 2019 · Synced Oct 21, 2026, 4:02 PM EDT
Recruiting Not applicable Interventional

Neuromodulation to Enhance Motor Function in HSP

NCT07417943
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
transcutaneous spinal cord stimulation
Device
Lead sponsor
Rahul Sachdeva
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Oct 21, 2026, 4:02 PM EDT
Completed Phase 3 Interventional Results available

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)

NCT02652780
View directory record Official record
Conditions
Optic, Atrophy, Hereditary, Leber
Interventions
GS010, Sham Intravitreal Injection
Biological · Device
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
3
States / cities
Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 21, 2026, 4:02 PM EDT
Recruiting No phase listed Observational

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

NCT01238250
View directory record Official record
Conditions
16P11.2 Deletion Syndrome, 16p11.2 Duplications, 1Q21.1 Deletion, 1Q21.1 Microduplication Syndrome (Disorder), ACTL6B, ADNP, AHDC1, ANK2, ANKRD11, ARID1B, ASH1L, BCL11A, CHAMP1, CHD2, CHD8, CSNK2A1, CTBP1, CTNNB1 Gene Mutation, CUL3, DDX3X, DNMT3A, DSCAM, DYRK1A, FOXP1, GRIN2A, GRIN2B, HIVEP2-Related Intellectual Disability, HNRNPH2, KATNAL2, KDM5B, KDM6B, KMT2C Gene Mutation, KMT2E, KMT5B, MBD5, MED13L, PACS1, PPP2R5D-Related Intellectual Disability, PTCHD1, REST, SCN2A Encephalopathy, SETBP1 Gene Mutation, SETD5, SMARCA4 Gene Mutation, SMARCC2, STXBP1 Encephalopathy With Epilepsy, SYNGAP1-Related Intellectual Disability, TBR1, ARHGEF9, HNRNPU, PPP3CA, PPP2R1A, SLC6A1, 2p16.3 Deletions, 5q35 Deletions, 5q35 Duplications, 7q11.23 Duplications, 15Q13.3 Deletion Syndrome, 16p11.2 Triplications, 16P12.2 Microdeletion, 16P13.11 Microdeletion Syndrome (Disorder), 17Q12 Microdeletion Syndrome (Disorder), 17Q12 Duplication Syndrome, 17Q21.31 Deletion Syndrome, 17q21.3 Duplications, ACTB, ADSL, AFF2, ALDH5A1, ANK3, ARX, ATRX Gene Mutation, AUTS2 Syndrome, BCKDK, BRSK2, CACNA1C, CAPRIN1, CASK, CASZ1, CHD3, CIC, CNOT3, CREBBP Gene Mutation, CSDE1, CTCF, DEAF1, DHCR7, DLG4, EBF3, EHMT1, EP300 Gene Mutation, GIGYF1, GRIN1, GRIN2D, IQSEC2-Related Syndromic Intellectual Disability, IRF2BPL, KANSL1, KCNB1, KDM3B, NEXMIF, KMT2A, MBOAT7, MEIS2, MYT1L, NAA15, NBEA, NCKAP1, NIPBL, NLGN2, NLGN3, NLGN4X, NR4A2, NRXN1, NRXN2, NSD1 Gene Mutation, PHF21A, PHF3, PHIP, PSMD12, RELN, RERE, RFX3, RIMS1, RORB, SCN1A, SETD2 Gene Mutation, SHANK2, SIN3A, SLC9A6, SON, SOX5, SPAST, SRCAP, TAOK1, TANC2, TCF20, TLK2, TRIO, TRIP12, UPF3B, USP9X, VPS13B, WAC, WDFY3, ZBTB20, ZNF292, ZNF462, 2Q37 Deletion Syndrome, 9q34 Duplications, 15q15 Deletions, 15Q24 Deletion, NR3C2, SYNCRIP, 2q37.3 Deletion, 6q16 Deletion, 15q11.2 BP1-BP2 Deletion, 16p13.3 Deletion, 17Q11.2 Microduplication Syndrome (Disorder), 17p13.3, Xq28 Duplication, CLCN4, CSNK2B, DYNC1H1, EIF3F, GNB1, MED13, MEF2C, RALGAPB, SCN1B, YY1, Xp11.22 Duplication, PACS2, MAOA, MAOB, HNRNPC, HNRNPD, HNRNPK, HNRNPR, HNRNPUL2, 5P Deletion Syndrome, TCF7L2 Gene Mutation, HECW2, PPM1D, RNU4-2, SNAP25, FOXP2, ITSN1
Interventions
Not listed
Lead sponsor
Simons Searchlight
Other
Eligibility
Not listed
Enrollment
100,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2050
U.S. locations
2
States / cities
Boston, Massachusetts • Lewisburg, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Oct 21, 2026, 4:02 PM EDT
Not listed No phase listed Observational

Exercise Effect on Transthyretin Stability

NCT04986826
View directory record Official record
Conditions
Amyloidosis Transthyretin, Athlete Heart
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years and older
Enrollment
24 participants
Timeline
2021
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Aug 3, 2021 · Synced Oct 21, 2026, 4:02 PM EDT
Completed Phase 2 Interventional Results available

A Phase II Study of Single Agent MEK162 in Patients With Advanced Melanoma

NCT01320085
View directory record Official record
Conditions
BRAF or NRAS Mutant Metastatic Melanoma
Interventions
MEK162
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2023
U.S. locations
10
States / cities
Fayetteville, Arkansas • Tampa, Florida • Portland, Oregon + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2024 · Synced Oct 21, 2026, 4:02 PM EDT
Completed Phase 2 Interventional

Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

NCT00457808
View directory record Official record
Conditions
Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
Rapamycin, sirolimus
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2006
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Apr 9, 2007 · Synced Oct 21, 2026, 4:02 PM EDT
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Derivation of Human Induced Pluripotent Stem (iPS) Cells to Heritable Cardiac Arrhythmias

NCT02413450
View directory record Official record
Conditions
Inherited Cardiac Arrythmias, Long QT Syndrome (LQTS), Brugada Syndrome (BrS), Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT), Early Repolarization Syndrome (ERS), Arrhythmogenic Cardiomyopathy (AC, ARVD/C), Hypertrophic Cardiomyopathy (HCM), Dilated Cardiomyopathy (DCM), Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Normal Control Subjects
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years to 85 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2031
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 21, 2026, 4:02 PM 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 21, 2026, 4:02 PM EDT
Completed No phase listed Observational

Incidence of Malignant Peripheral Nerve Sheath Tumor (MPNST) Development in Participants With Neurofibromatosis Type 1 (NF1) Receiving and Not Receiving Medical Therapies Directed at Plexiform Neurofibromas (PN)

NCT04774289
View directory record Official record
Conditions
Neurofibromatosis 1, Peripheral Nerve Neoplasms, Malignant
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month and older
Enrollment
296 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 19, 2024 · Synced Oct 21, 2026, 4:02 PM EDT
Completed Phase 2 Interventional

A Double-Blind, Placebo-Controlled, Dose Exploration Study of CTI-1601 in Adult Subjects With Friedreich's Ataxia

NCT05579691
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
CTI-1601, Placebo
Biological · Other
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Nov 29, 2024 · Synced Oct 21, 2026, 4:02 PM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb

NCT06300996
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy 4, SMA
Interventions
Spinal Cord Stimulator (Octopolar Medtronic Vectris Leads)
Device
Lead sponsor
Marco Capogrosso
Other
Eligibility
16 Years to 65 Years
Enrollment
3 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2029
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 26, 2025 · Synced Oct 21, 2026, 4:02 PM EDT
Completed Phase 4 Interventional

Treatment of Obsessive-Compulsive Disorder

NCT00000373
View directory record Official record
Conditions
Obsessive-Compulsive Disorder
Interventions
olanzapine + fluoxetine, placebo + fluoxetine
Drug
Lead sponsor
University of Florida
Other
Eligibility
14 Years to 70 Years
Enrollment
74 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1992 – 2003
U.S. locations
2
States / cities
Gainesville, Florida • Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Nov 27, 2013 · Synced Oct 21, 2026, 4:02 PM EDT
Completed Phase 1 Interventional

Safety, Pharmacokinetics, and Pharmacodynamics of NBI-98854 in Children and Adolescents With Tourette Syndrome

NCT02256475
View directory record Official record
Conditions
Tourette Syndrome
Interventions
NBI-98854
Drug
Lead sponsor
Neurocrine Biosciences
Industry
Eligibility
6 Years to 18 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
10
States / cities
San Diego, California • Kissimmee, Florida • St. Petersburg, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jan 7, 2016 · Synced Oct 21, 2026, 4:02 PM EDT
Completed Phase 1Phase 2 Interventional

Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease

NCT00541164
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Coenzyme Q10, Coenzyme Q10
Drug · Dietary Supplement
Lead sponsor
Memorial Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Johnstown, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 15, 2013 · Synced Oct 21, 2026, 4:02 PM EDT