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ClinicalTrials.gov public records Last synced Oct 13, 2026, 8:08 AM EDT

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Showing 97–120 of 1,145 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Terminated No phase listed Observational Accepts healthy volunteers

Collection of Cerebrospinal Fluid in Healthy Children

NCT01698229
View directory record Official record
Conditions
Late Infantile Neuronal Ceroid Lipofuscinosis, Batten Disease
Interventions
Group 1
Other
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years to 8 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 4, 2016 · Synced Oct 13, 2026, 8:08 AM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Gait Analysis in Neurological Disease

NCT02994719
View directory record Official record
Conditions
Parkinson's Disease, Parkinsonian Disorders, Atypical Parkinson Disease, Progressive Supranuclear Palsy, Multiple System Atrophy, Corticobasal Degeneration, Gait, Frontal, Huntington Disease, Ataxia, Spinocerebellar
Interventions
Anti-Parkinson medication, Deep Brain Stimulation
Drug · Device
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 85 Years
Enrollment
120 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 8, 2026 · Synced Oct 13, 2026, 8:08 AM EDT
Terminated Phase 1 Interventional

A Study to Assess Nomlabofusp in Adolescents and Children With Friedreich's Ataxia

NCT06681766
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Nomlabofusp, Placebo
Drug
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
2 Years to 17 Years
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Chevy Chase, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 13, 2026, 8:08 AM EDT
Completed Phase 3 Interventional Results available

Study Evaluating the Safety and Efficacy of Fixed-dose Once-daily Oral Aripiprazole in Children and Adolescents With Tourette's Disorder

NCT01727700
View directory record Official record
Conditions
Tourette's Disorder, Tic Disorder
Interventions
Aripiprazole, Placebo
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
7 Years to 17 Years
Enrollment
133 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
55
States / cities
Dothan, Alabama • Goodyear, Arizona • Tucson, Arizona + 52 more
Source: ClinicalTrials.gov public record
Updated Feb 13, 2015 · Synced Oct 13, 2026, 8:08 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 13, 2026, 8:08 AM EDT
Completed Phase 2 Interventional Results available

Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas

NCT00076102
View directory record Official record
Conditions
Neurofibromatosis 1, Neurofibroma, Plexiform
Interventions
Pirfenidone
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 21 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
15
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 23, 2018 · Synced Oct 13, 2026, 8:08 AM EDT
Completed Phase 3 Interventional Results available

The Effects Of Bronchodilator Therapy On Respiratory And Autonomic Function In Patients With Familial Dysautonomia

NCT01987219
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
Albuterol-sulphate, Ipratropium-bromide, placebo
Drug · Other
Lead sponsor
NYU Langone Health
Other
Eligibility
12 Years to 80 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 2, 2016 · Synced Oct 13, 2026, 8:08 AM EDT
Completed Not applicable Interventional

Behavioral Therapy for Tourette Syndrome

NCT03019731
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Therapist-directed Behavioral Therapy, Home-based DVD Therapy
Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
7 Years to 13 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 1, 2021 · Synced Oct 13, 2026, 8:08 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Development of Quantitative Muscle Imaging as a Biomarker of Disease Endpoints in Myotonic Dystrophy

NCT07362875
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years to 65 Years
Enrollment
75 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Winston-Salem, North Carolina
Source: ClinicalTrials.gov public record
Updated Jan 23, 2026 · Synced Oct 13, 2026, 8:08 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

NCT06948019
View directory record Official record
Conditions
HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50, Hereditary Spastic Paraplegia Type 47, Hereditary Spastic Paraplegia Type 51, Hereditary Spastic Paraplegia Type 52, SPG47, AP4B1, Neurogenetic Disorders, Neurodevelopmental Conditions, Movement Disorders, Gene Therapy
Interventions
BFB-101 (AAV9-CBh-AP4B1)
Biological
Lead sponsor
BlackfinBio Ltd
Industry
Eligibility
12 Months to 60 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 28, 2025 · Synced Oct 13, 2026, 8:08 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 13, 2026, 8:08 AM EDT
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 13, 2026, 8:08 AM EDT
Completed Not applicable Interventional Results available

Median Nerve Stimulation Pilot

NCT04731714
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders, Tic Disorder, Chronic Motor or Vocal
Interventions
Rhythmic median nerve stimulation, Arrhythmic median nerve stimulation
Device
Lead sponsor
Washington University School of Medicine
Other
Eligibility
15 Years to 64 Years
Enrollment
34 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 13, 2026, 8:08 AM EDT
Completed Phase 2 Interventional Results available

Study of MK-3475 (Pembrolizumab) in Patients With Microsatellite Unstable (MSI) Tumors (Cohort D)

NCT04098068
View directory record Official record
Conditions
High Tumor Mutation Burden, High TMB (Tumor Mutation Burden), MSS (Microsatellite Stable)
Interventions
MK-3475
Drug
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
3
States / cities
Baltimore, Maryland • New York, New York • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 30, 2025 · Synced Oct 13, 2026, 8:08 AM EDT
Recruiting Not applicable Interventional

Investigating Changes in Premonitory Urges During Habit Reversal Training for Tics

NCT06825520
View directory record Official record
Conditions
Tics, Tourette Disorder, Tourette Syndrome in Children, Tourette Syndrome in Adolescence
Interventions
Habit Reversal Training
Behavioral
Lead sponsor
University of Utah
Other
Eligibility
8 Years to 17 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated May 28, 2025 · Synced Oct 13, 2026, 8:08 AM EDT
Not listed Not applicable Interventional

A Clinical Study to Evaluate the Safety and Efficacy of T92 in Pediatric Patients With Tourette Syndrome

NCT05188274
View directory record Official record
Conditions
Tourette Syndrome in Children, Tourette Syndrome in Adolescence
Interventions
T92, Placebo
Dietary Supplement
Lead sponsor
Tasly Pharmaceuticals, Inc.
Industry
Eligibility
6 Years to 17 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jan 12, 2022 · Synced Oct 13, 2026, 8:08 AM EDT
Completed No phase listed Observational

Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

NCT01203085
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
Up to 21 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2025
U.S. locations
9
States / cities
Palo Alto, California • Hartford, Connecticut • Orlando, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 7, 2025 · Synced Oct 13, 2026, 8:08 AM EDT
Completed Phase 3 Interventional Results available

Safety and Tolerability of Once-daily Oral Aripiprazole in Children and Adolescents With Tourette's Disorder

NCT01727713
View directory record Official record
Conditions
Tourette's Disorder, Tic Disorder
Interventions
Aripiprazole
Drug
Lead sponsor
Otsuka Pharmaceutical Development & Commercialization, Inc.
Industry
Eligibility
7 Years to 18 Years
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
26
States / cities
Dothan, Alabama • Goodyear, Arizona • Orange, California + 23 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2015 · Synced Oct 13, 2026, 8:08 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Study of Electrical Impedance Myography (EIM) in ALS

NCT02011204
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Charcot-Marie-Tooth Disease, Multiple Sclerosis
Interventions
Electrical impedance myography (EIM)
Device
Lead sponsor
Skulpt, Inc.
Industry
Eligibility
35 Years to 80 Years
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
6
States / cities
Phoenix, Arizona • Miami, Florida • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 11, 2016 · Synced Oct 13, 2026, 8:08 AM EDT
Completed Phase 3 Interventional Results available

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

NCT01960348
View directory record Official record
Conditions
TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
Interventions
patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
13
States / cities
La Mesa, California • Orange, California • Denver, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2024 · Synced Oct 13, 2026, 8:08 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Investigation of Neurofeedback With Real-Time fMRI in Healthy Volunteers and Patients With Hyperkinetic Movement Disorders

NCT00885040
View directory record Official record
Conditions
Movement Disorder, Tourette Syndrome, Healthy Volunteer
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 60 Years
Enrollment
59 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 24, 2019 · Synced Oct 13, 2026, 8:08 AM EDT
Completed No phase listed Observational

Study of Disease Severity in Adults With Neurofibromatosis Type 1 (NF1)

NCT00111384
View directory record Official record
Conditions
Neurofibromatosis Type 1, Legius Syndrome
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
2 Years to 100 Years
Enrollment
313 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 31, 2026 · Synced Oct 13, 2026, 8:08 AM EDT
Completed Not applicable Interventional Results available

Transcranial Magnetic Stimulation in Spino-Cerebellar Ataxia

NCT01975909
View directory record Official record
Conditions
Spinocerebellar Ataxia
Interventions
Transcranial Magnetic Stimulation
Device
Lead sponsor
Beth Israel Deaconess Medical Center
Other
Eligibility
18 Years to 70 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 30, 2017 · Synced Oct 13, 2026, 8:08 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Analysis of Data Collected From Individuals Administered Neurobehavioral Assessments

NCT01445314
View directory record Official record
Conditions
HIV, Brain Tumors, Neurofibromatosis Type 1
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month and older
Enrollment
598 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2024
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 16, 2024 · Synced Oct 13, 2026, 8:08 AM EDT