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ClinicalTrials.gov public records Last synced Oct 7, 2026, 4:09 AM EDT

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Showing 25–48 of 1,144 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed No phase listed Observational Accepts healthy volunteers

Neuroimaging of Dystonia

NCT02046447
View directory record Official record
Conditions
Primary Cervical Dystonia, DYT 1 Dystonia
Interventions
Primary Cervical Dystonia (Trihexyphenidyl), Controls Primary Cervical Dystonia (Trihexyphenidyl), DYT 1 Dystonia (Healthy Control), DYT 1 Dystonia
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
7 Years to 70 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 22, 2017 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Neurobiology and Treatment of Reading Disability in NF-1

NCT00624234
View directory record Official record
Conditions
Neurofibromatosis Type 1, Reading Disabilities
Interventions
Tutoring Program I, Tutoring Program II
Behavioral
Lead sponsor
Vanderbilt University
Other
Eligibility
8 Years to 17 Years
Enrollment
184 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2017
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 8, 2025 · Synced Oct 7, 2026, 4:09 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Parkinsonism in Spinocerebellar Ataxia Type 6

NCT01934998
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 6
Interventions
Not listed
Lead sponsor
University of Chicago
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2015
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Apr 28, 2015 · Synced Oct 7, 2026, 4:09 AM EDT
Recruiting Phase 2 Interventional

Sapanisertib and Serabelisib (PIKTOR) With Paclitaxel and a Substudy With an Insulin-Suppressing Diet in Patients With Advanced/Recurrent Endometrial Cancer

NCT06463028
View directory record Official record
Conditions
Endometrial Cancer
Interventions
Sapanisertib, Serabelisib, Paclitaxel
Drug
Lead sponsor
Faeth Therapeutics
Industry
Eligibility
18 Years and older · Female only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
18
States / cities
La Jolla, California • San Francisco, California • Miami Beach, Florida + 15 more
Source: ClinicalTrials.gov public record
Updated Dec 24, 2025 · Synced Oct 7, 2026, 4:09 AM EDT
Terminated Phase 3 Interventional Results available

Tanezumab In Osteoarthritis Of The Hip Or Knee

NCT00985621
View directory record Official record
Conditions
Osteoarthritis
Interventions
tanezumab 10 mg, tanezumab 5 mg, oxycodone, placebo
Biological · Drug · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
614 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
66
States / cities
Birmingham, Alabama • Mobile, Alabama • Phoenix, Arizona + 54 more
Source: ClinicalTrials.gov public record
Updated May 14, 2021 · Synced Oct 7, 2026, 4:09 AM EDT
Recruiting No phase listed Observational

Investigations of Juvenile Neuronal Ceroid Lipofuscinosis

NCT03307304
View directory record Official record
Conditions
Juvenile Neuronal Ceroid Lipofuscinosis (CLN3), Batten Disease
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
1 Week to 100 Years
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2050
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 5, 2026 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)

NCT01070316
View directory record Official record
Conditions
Epilepsy, Tuberous Sclerosis Complex
Interventions
Everolimus
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
2 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2016
U.S. locations
2
States / cities
Cincinnati, Ohio • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 13, 2017 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Not applicable Interventional Results available

Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials

NCT03531814
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibroma, Plexiform
Interventions
Medication Event Monitoring System (MEMS^TM), Questionnaires
Behavioral · Other
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 59 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 11, 2024 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Phase 1 Interventional

Dose Escalation of RMC-5552 Monotherapy in Relapsed/Refractory Solid Tumors

NCT04774952
View directory record Official record
Conditions
Solid Tumors
Interventions
RMC-5552
Drug
Lead sponsor
Revolution Medicines, Inc.
Industry
Eligibility
18 Years and older
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
8
States / cities
Irvine, California • Sacramento, California • San Francisco, California + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2025 · Synced Oct 7, 2026, 4:09 AM EDT
Recruiting No phase listed Observational

Longitudinal Endpoint Assessment of Disease Burden in HD

NCT06475898
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Huntington Study Group
Network
Eligibility
18 Years and older
Enrollment
600 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Sep 10, 2025 · Synced Oct 7, 2026, 4:09 AM EDT
Terminated Phase 2 Interventional Results available

Open Label Extension Study To Investigate Long Term Safety, Tolerability And Efficacy Of Pf-02545920 In Subjects With Huntington's Disease Who Completed Study A8241021

NCT02342548
View directory record Official record
Conditions
Huntington's Disease
Interventions
20 mg BID of PF-02545920
Drug
Lead sponsor
Pfizer
Industry
Eligibility
30 Years to 66 Years
Enrollment
188 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
17
States / cities
Birmingham, Alabama • Scottsdale, Arizona • Irvine, California + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 23, 2018 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Phase 1Phase 2 Interventional

Long Term Follow Up for RAD001 Therapy of Angiomyolipomata in Patients With Tuberous Sclerosis (TSC) and Sporadic Lymphangioleiomyomatosis (LAM)

NCT00792766
View directory record Official record
Conditions
Tuberous Sclerosis, Angiolipoma
Interventions
everolimus (RAD001)
Drug
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 18, 2013 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Transmembrane Electromyography (TM-EMG) for the Assessment of Neuromuscular Function in the Oropharynx

NCT03986671
View directory record Official record
Conditions
Obstructive Sleep Apnea, Amyotrophic Lateral Sclerosis, Muscular Dystrophies, Healthy
Interventions
Transmembrane EMG Oropharynx Probe
Device
Lead sponsor
Powell Mansfield Inc.
Other
Eligibility
18 Years to 70 Years
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2021
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Jun 21, 2022 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Not applicable Interventional Results available

Open-label MNS for Tourette Syndrome

NCT05016765
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorder, Chronic Motor or Vocal
Interventions
Active, self-directed electrical stimulation of the median nerve
Device
Lead sponsor
Washington University School of Medicine
Other
Eligibility
15 Years to 65 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Oct 5, 2023 · Synced Oct 7, 2026, 4:09 AM EDT
Completed No phase listed Observational

Down Syndrome Biomarker Initiative (DSBI)

NCT02141971
View directory record Official record
Conditions
Down Syndrome, Alzheimer's Disease
Interventions
Not listed
Lead sponsor
Michael Rafii
Other
Eligibility
30 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
La Jolla, California
Source: ClinicalTrials.gov public record
Updated May 2, 2017 · Synced Oct 7, 2026, 4:09 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Intracardiac Flow Assessment in Cardiac Amyloidosis

NCT05379101
View directory record Official record
Conditions
Amyloidosis Cardiac, Healthy Adults
Interventions
Cardiac magnetic resonance image (CMR), Transthoracic Echocardiogram (TTE), Six-minute Walk Test
Diagnostic Test
Lead sponsor
Mayo Clinic
Other
Eligibility
40 Years and older
Enrollment
81 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Not applicable Interventional

Respiratory Training in Friedreich's Ataxia

NCT06539598
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Respiratory Strength Training (RST)
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Nov 3, 2025 · Synced Oct 7, 2026, 4:09 AM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Evaluating Mitochondrial Dysfunction in Patients With Neurofibromatosis Type 1

NCT05912400
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Blood draw, FACIT-F and Pain Scales
Diagnostic Test · Other
Lead sponsor
University of Arkansas
Other
Eligibility
18 Years and older
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2024
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated May 8, 2025 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904
View directory record Official record
Conditions
Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Gaucher's Disease, Fucosidosis, Wolman Disease, Niemann-Pick Disease, Batten Disease, GM1 Gangliosidosis, Tay Sachs Disease, Sandhoff Disease
Interventions
Stem Cell Transplant, Busulfan, Cyclophosphamide, Antithymocyte Globulin
Procedure · Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Not listed
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1995 – 2010
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Dec 28, 2017 · Synced Oct 7, 2026, 4:09 AM EDT
Not listed Not applicable Interventional Accepts healthy volunteers

Open Trial of Telehealth Group for Improving Peer Relationships (PEERS) in NF1

NCT04856514
View directory record Official record
Conditions
Neurofibromatosis 1, Social Skills
Interventions
PEERS
Behavioral
Lead sponsor
University of Wisconsin, Milwaukee
Other
Eligibility
12 Years to 17 Years
Enrollment
8 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2021
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Apr 27, 2021 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Not applicable Interventional

Time-Restricted Eating in Huntington's Disease: A Clinical Pilot Study

NCT06490367
View directory record Official record
Conditions
Huntington Disease
Interventions
Time-Restricted Eating Diet
Behavioral
Lead sponsor
Oregon Health and Science University
Other
Eligibility
21 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jun 4, 2025 · Synced Oct 7, 2026, 4:09 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Charcot-Marie-Tooth Disease (CMT) Biological Sample Collection for IPSC Generation and Biobanking

NCT06203093
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Healthy
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
5 Years and older
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Phase 1 Study To Evaluate The Pharmacokinetics, Safety, And Tolerability Of Dimebon [PF-01913539] In Japanese And Western Healthy Subjects

NCT00825084
View directory record Official record
Conditions
Alzheimer's Disease, Huntington's Disease
Interventions
Dimebon
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 55 Years
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009
U.S. locations
1
States / cities
Glendale, California
Source: ClinicalTrials.gov public record
Updated Apr 26, 2011 · Synced Oct 7, 2026, 4:09 AM EDT
Completed Phase 1 Interventional

An Implant for Hearing Loss Due to Removal of Neurofibromatosis 2 Tumors

NCT00030043
View directory record Official record
Conditions
Neurofibromatosis 2
Interventions
Penetrating auditory brainstem implant
Device
Lead sponsor
FDA Office of Orphan Products Development
Federal
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
3
States / cities
Los Angeles, California • Pasadena, California • Englewood, Colorado
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 7, 2026, 4:09 AM EDT