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ClinicalTrials.gov public records Last synced Oct 21, 2026, 2:42 PM EDT

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Showing 697–720 of 1,148 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed No phase listed Observational

Validating Pain Scales in Children and Young Adults

NCT01639950
View directory record Official record
Conditions
Sickle Cell Disease, Solid Tumor, Leukemia, Neurofibromatosis
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
252 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2023
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 21, 2024 · Synced Oct 21, 2026, 2:42 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Brain Dynamics Involved in Generating Tics and Controlling Voluntary Movement

NCT00056420
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
21 Years to 65 Years
Enrollment
54 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2003 – 2009
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 21, 2026, 2:42 PM EDT
Recruiting No phase listed Observational

Clinical and Neuropsychological Investigations in Batten Disease

NCT01873924
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis, Neuronal Ceroid Lipofuscinosis CLN1, Neuronal Ceroid Lipofuscinosis CLN2, Neuronal Ceroid Lipofuscinosis CLN3, Neuronal Ceroid Lipofuscinosis CLN5, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN7, Neuronal Ceroid Lipofuscinosis CLN8, Neuronal Ceroid Lipofuscinosis CLN10, Batten Disease
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2035
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Sep 12, 2025 · Synced Oct 21, 2026, 2:42 PM EDT
Completed Phase 3 Interventional Results available

Ecopipam Tablets to Study Tourette's Disorder in Children, Adolescents and Adults

NCT05615220
View directory record Official record
Conditions
Tourette Disorder
Interventions
Ecopipam Hydrochloride
Drug
Lead sponsor
Emalex Biosciences Inc.
Industry
Eligibility
6 Years and older
Enrollment
216 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
56
States / cities
Dothan, Alabama • Little Rock, Arkansas • Anaheim, California + 41 more
Source: ClinicalTrials.gov public record
Updated Jun 12, 2026 · Synced Oct 21, 2026, 2:42 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Safety and Efficacy Studies of Panobinostat and Bicalutamide in Patients With Recurrent Prostate Cancer After Castration

NCT00878436
View directory record Official record
Conditions
Prostate Cancer, Prostatic Neoplasms
Interventions
Panobinostat, Bicalutamide
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years and older · Male only
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
4
States / cities
New Brunswick, New Jersey • Lake Success, New York • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 7, 2018 · Synced Oct 21, 2026, 2:42 PM EDT
Completed No phase listed Observational

Clinical and Molecular Correlations in Spinocerebellar Ataxia Type 10 (SCA10)

NCT00004306
View directory record Official record
Conditions
Hereditary Ataxia
Interventions
Not listed
Lead sponsor
Office of Rare Diseases (ORD)
NIH
Eligibility
3 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2009
U.S. locations
1
States / cities
Galveston, Texas
Source: ClinicalTrials.gov public record
Updated Mar 6, 2012 · Synced Oct 21, 2026, 2:42 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Characterization of the Cardiac Phenotype of Friedreich's Ataxia (FRDA)

NCT02316314
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
Cardiac magnetic resonance imaging (CMR), Exercise-stress test, Echocardiogram (ECHO), Cardiac-related blood studies
Procedure
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
12 Years to 50 Years
Enrollment
100 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 21, 2026, 2:42 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Observational Study of Neuromuscular Function in CMT Type 1&2 and Healthy Controls

NCT04980807
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
Not listed
Lead sponsor
NMD Pharma A/S
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 7, 2022 · Synced Oct 21, 2026, 2:42 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Proof-of-Concept Safety Study of CPP-109 (Vigabatrin) for Treatment Refractory Tourette's Disorder

NCT01585207
View directory record Official record
Conditions
Tourette's Disorder
Interventions
vigabatrin
Drug
Lead sponsor
Barbara J. Coffey
Other
Eligibility
18 Years to 35 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 19, 2018 · Synced Oct 21, 2026, 2:42 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Phase Ib/II Study of BYL719 and Cetuximab in Recurrent or Metastatic Head and Neck Squamous Cell Carcinoma

NCT01602315
View directory record Official record
Conditions
Recurrent Head and Neck Squamous Cell Carcinoma, Metastatic Head and Neck Squamous Cell Carcinoma
Interventions
BYL719 as film-coated (FC) whole tablets, BYL719 as dispersible tablets (DT), cetuximab, BYL719 drink suspension
Drug · Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
179 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
14
States / cities
Sacramento, California • San Francisco, California • Aurora, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Dec 29, 2020 · Synced Oct 21, 2026, 2:42 PM EDT
Completed No phase listed Observational

Effect of Fasting on the Size of Abdominal Lymphatic Tumors in Women

NCT00552955
View directory record Official record
Conditions
Lymphangioleiomyomas, Tuberous Sclerosis, Lymphangioleiomyomatosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 80 Years · Female only
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2016
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 6, 2019 · Synced Oct 21, 2026, 2:42 PM EDT
Completed Phase 1Phase 2 Interventional

Minocycline in Patients With Huntington's Disease

NCT00029874
View directory record Official record
Conditions
Huntington's Disease
Interventions
Minocycline
Drug
Lead sponsor
FDA Office of Orphan Products Development
Federal
Eligibility
18 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2015 · Synced Oct 21, 2026, 2:42 PM EDT
Completed Phase 2 Interventional

Safety and Tolerability Study of Phenylbutyrate in Huntington's Disease (PHEND-HD)

NCT00212316
View directory record Official record
Conditions
Huntington's Disease
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
8
States / cities
Birmingham, Alabama • San Diego, California • Iowa City, Iowa + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 15, 2012 · Synced Oct 21, 2026, 2:42 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Doxycycline in Cutaneous Schwannoma (NF2)

NCT05521048
View directory record Official record
Conditions
Neurofibromatosis Type 2
Interventions
Doxycycline Injection [Doxy]
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
8 Years and older
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 21, 2026, 2:42 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Rocky Mountain Alzheimer's Disease Center Longitudinal Biomarker and Clinical Phenotyping Study

NCT02612376
View directory record Official record
Conditions
Alzheimer Disease, Down Syndrome, Mild Cognitive Impairment
Interventions
Not listed
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years and older
Enrollment
800 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2025
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Jun 3, 2025 · Synced Oct 21, 2026, 2:42 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Inherited Retinal Degenerative Disease Registry

NCT02435940
View directory record Official record
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced Oct 21, 2026, 2:42 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Determine Range of Tissue Frataxin Concentrations and Other Potential Biomarkers

NCT05028764
View directory record Official record
Conditions
Friedreich Ataxia, Healthy Volunteers
Interventions
Buccal Swabs, Blood Draws and Skin Punch Biopsy
Diagnostic Test
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years to 50 Years
Enrollment
60 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Feb 24, 2022 · Synced Oct 21, 2026, 2:42 PM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Mutations Associated With Parkinson s Disease

NCT01547832
View directory record Official record
Conditions
Parkinson's Disease
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 80 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 29, 2018 · Synced Oct 21, 2026, 2:42 PM EDT
Completed Phase 1 Interventional Results available

Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)

NCT01682811
View directory record Official record
Conditions
Neurofibromatoses
Interventions
Part 1 Levulan injection, Part 1 Levulan surface application, Part 1 Levulan surface application twice, Part 1 Levulan surface application twice with microneedling, Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 1, Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 2, Levulan (5-aminolevulinic acid) photodynamic therapy - Dose level 3
Drug
Lead sponsor
Harry T Whelan, MD
Other
Eligibility
18 Years to 90 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jun 18, 2021 · Synced Oct 21, 2026, 2:42 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

I-123 Brain Studies of Serotonin Metabolism in Psychiatric Patients and Normal Volunteers

NCT00001771
View directory record Official record
Conditions
Healthy, Mental Disorder, Obsessive Compulsive Disorder, Schizophrenia, Tourette Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
Not listed
Enrollment
112 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 21, 2026, 2:42 PM EDT
Completed Phase 1 Interventional

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

NCT05573698
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
DT-216, Placebo
Drug
Lead sponsor
Design Therapeutics, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
32 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 Feb 29, 2024 · Synced Oct 21, 2026, 2:42 PM EDT
Completed No phase listed Observational Accepts healthy volunteers Results available

Examination of Quantitative Electroencephalographic (QEEG) Biomarkers in Huntington's Disease

NCT00670709
View directory record Official record
Conditions
Huntington's Disease
Interventions
Not listed
Lead sponsor
University of California, Los Angeles
Other
Eligibility
21 Years to 85 Years
Enrollment
42 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2008
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Nov 9, 2023 · Synced Oct 21, 2026, 2:42 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)

NCT05462106
View directory record Official record
Conditions
Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease, Alzheimer's Disease
Interventions
Placebo (Study Part 1a), ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a, ACI-24.060 with an additional adjuvant at Dose D in Study Part 1b, Placebo (Study Part 2), ACI-24.060 at Dose A in Study Part 2, ACI-24.060 at Dose B in Study Part 2, ACI-24.060 at Dose C in Study Part 2, Placebo (Study Part 1b), ACI-24.060 with an additional adjuvant at Dose E in Study Part 1b
Biological
Lead sponsor
AC Immune SA
Industry
Eligibility
35 Years to 85 Years
Enrollment
304 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
13
States / cities
Phoenix, Arizona • Lady Lake, Florida • Orlando, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 21, 2026, 2:42 PM EDT
Completed Phase 2 Interventional

Effectiveness of Behavior Therapy and Psychosocial Therapy for the Treatment of Tourette Syndrome and Chronic Tic Disorder

NCT00231985
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders
Interventions
Habit reversal therapy, Supportive therapy
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
16 Years and older
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
3
States / cities
New Haven, Connecticut • Boston, Massachusetts • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Feb 9, 2012 · Synced Oct 21, 2026, 2:42 PM EDT