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ClinicalTrials.gov public records Last synced Oct 24, 2026, 11:23 AM EDT

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Showing 1057–1080 of 1,148 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 2 Interventional Results available

Study Assessing the Efficacy and Safety of Alpelisib Plus Fulvestrant or Letrozole, Based on Prior Endocrine Therapy, in Patients With PIK3CA Mutant, HR+, HER2- Advanced Breast Cancer Who Have Progressed on or After Prior Treatments

NCT03056755
View directory record Official record
Conditions
Breast Cancer
Interventions
Alpelisib, Fulvestrant, Letrozole, Goserelin, Leuprolide
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 99 Years
Enrollment
383 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
25
States / cities
Gilbert, Arizona • Phoenix, Arizona • Anaheim, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 24, 2026, 11:23 AM 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 24, 2026, 11:23 AM EDT
Terminated Phase 3 Interventional

Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E)

NCT00712426
View directory record Official record
Conditions
Huntington's Disease
Interventions
Creatine Monohydrate, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
553 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
38
States / cities
Birmingham, Alabama • Irvine, California • Sacramento, California + 35 more
Source: ClinicalTrials.gov public record
Updated Mar 11, 2016 · Synced Oct 24, 2026, 11:23 AM EDT
Completed Early Phase 1 Interventional Accepts healthy volunteers

Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic Pain

NCT01633008
View directory record Official record
Conditions
Neurofibromatosis
Interventions
ACT Workshop
Behavioral
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
12 Years to 99 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2017
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 30, 2019 · Synced Oct 24, 2026, 11:23 AM EDT
Recruiting Phase 3 Interventional

Pridopidine Phase 3 Study in Huntington's Disease

NCT07609108
View directory record Official record
Conditions
Huntington Disease
Interventions
Pridopidine, Placebo
Drug
Lead sponsor
Prilenia
Industry
Eligibility
23 Years to 65 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
25
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 21 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 24, 2026, 11:23 AM EDT
Completed Not applicable Interventional

Modified Comprehensive Behavioral Intervention for Tics (M_CBIT)

NCT02900144
View directory record Official record
Conditions
Tourette Syndrome, ADHD
Interventions
Modified Comprehensive Behavioral Intervention for Tics, Comprehensive Behavioral Intervention for Tics
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
10 Years to 17 Years
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 16, 2021 · Synced Oct 24, 2026, 11:23 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)

NCT03094832
View directory record Official record
Conditions
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
Interventions
Miransertib
Drug
Lead sponsor
ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Industry
Eligibility
2 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
6
States / cities
Atlanta, Georgia • Chicago, Illinois • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2023 · Synced Oct 24, 2026, 11:23 AM EDT
Active, not recruiting No phase listed Observational

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

NCT04098211
View directory record Official record
Conditions
Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
Interventions
Not listed
Lead sponsor
Children's Hospital of Orange County
Other
Eligibility
4 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 24, 2026, 11:23 AM 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 24, 2026, 11:23 AM EDT
Terminated Phase 3 Interventional Results available

Study of Safety and Efficacy of Brolucizumab 6 mg Dosed Every 4 Weeks Compared to Aflibercept 2 mg Dosed Every 4 Weeks in Patients With Retinal Fluid Despite Frequent Anti-VEGF Injections

NCT03710564
View directory record Official record
Conditions
Age-Related Macular Degeneration
Interventions
Brolucizumab, Aflibercept
Biological
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
50 Years and older
Enrollment
535 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
60
States / cities
Phoenix, Arizona • Beverly Hills, California • Huntington Beach, California + 51 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2023 · Synced Oct 24, 2026, 11:23 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Study of and Genetic Modifiers in Spinocerebellar Ataxias

NCT01060371
View directory record Official record
Conditions
Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 10, RFC1 Gene Mutation, Spinocerebellar Ataxia Type 27b, Healthy Participants
Interventions
Genetic Testing, Blood Collection, Magnetic Resonance Imaging (MRI) Scan, Assessments and Questionnaires, Cerebrospinal Fluid Collection
Genetic · Other
Lead sponsor
Lauren Moore
Other
Eligibility
6 Years and older
Enrollment
1,400 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2030
U.S. locations
16
States / cities
Los Angeles, California • San Francisco, California • Gainesville, Florida + 12 more
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 24, 2026, 11:23 AM EDT
Terminated Not applicable Interventional Results available

Thalamic Deep Brain Stimulation for the Treatment of Refractory Tourette Syndrome

NCT01817517
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Medtronic Activa Deep Brain Stimulation System
Device
Lead sponsor
Johns Hopkins University
Other
Eligibility
15 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2023
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Jan 28, 2026 · Synced Oct 24, 2026, 11:23 AM 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 24, 2026, 11:23 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Natural History of Amyloid Deposition in Adults With Down Syndrome

NCT01303133
View directory record Official record
Conditions
Down Syndrome
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
30 Years and older
Enrollment
81 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2018
U.S. locations
2
States / cities
Pittsburgh, Pennsylvania • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jan 9, 2019 · Synced Oct 24, 2026, 11:23 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 24, 2026, 11:23 AM EDT
Completed Phase 1 Interventional

Safety Study of a Gene Transfer Vector for Children With Late Infantile Neuronal Ceroid Lipofuscinosis

NCT00151216
View directory record Official record
Conditions
Batten Disease, Late Infantile Neuronal Ceroid Lipofuscinosis
Interventions
AAV2CUhCLN2 (3x10^12 particle units)
Biological
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
3 Years to 18 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 24, 2020 · Synced Oct 24, 2026, 11:23 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

NCT03692312
View directory record Official record
Conditions
Congenital Myotonic Dystrophy
Interventions
Tideglusib, Placebo
Drug
Lead sponsor
AMO Pharma Limited
Industry
Eligibility
6 Years to 16 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
9
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2025 · Synced Oct 24, 2026, 11:23 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Stem Cells in NF1 Patients With Tumors of the Central Nervous System

NCT03332030
View directory record Official record
Conditions
Neurofibromatosis Type 1, Tumors of the Central Nervous System
Interventions
Collection of Stem Cells
Diagnostic Test
Lead sponsor
Roger Packer
Other
Eligibility
Not listed
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2025
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Feb 7, 2024 · Synced Oct 24, 2026, 11:23 AM EDT
Completed No phase listed Observational

FuRST 2.0 Cognitive Pre-testing - Round 2

NCT03709173
View directory record Official record
Conditions
Huntington Disease
Interventions
FuRST 2.0
Behavioral
Lead sponsor
CHDI Foundation, Inc.
Other
Eligibility
18 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019
U.S. locations
3
States / cities
Englewood, Colorado • Wichita, Kansas • New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 29, 2019 · Synced Oct 24, 2026, 11:23 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Change in MUNIX in Patients With CMT1A Undergoing a Home Ankle Strengthening Program Versus Standard of Care

NCT03715283
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
Home Ankle Strengthing Program, Standard of Care
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
19 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Dec 20, 2019 · Synced Oct 24, 2026, 11:23 AM EDT
Completed Phase 2 Interventional Results available

Rapalogues for Autism Phenotype in TSC: A Feasibility Study

NCT01929642
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Self-injury, Autism
Interventions
Sirolimus, Everolimus
Drug
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
2 Years to 30 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 29, 2021 · Synced Oct 24, 2026, 11:23 AM EDT
Completed Phase 2 Interventional Results available

Phase 2 Clinical Trial of Crizotinib for Children and Adults With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas

NCT04283669
View directory record Official record
Conditions
Neurofibromatosis 2, Progressive Vestibular Schwannoma (VS)
Interventions
Crizotinib
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
6 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
15
States / cities
Birmingham, Alabama • Los Angeles, California • Washington D.C., District of Columbia + 10 more
Source: ClinicalTrials.gov public record
Updated Mar 13, 2026 · Synced Oct 24, 2026, 11:23 AM EDT
Completed Phase 4 Interventional Results available

Study to Assess the Efficacy and Safety of Raxone in LHON Patients

NCT02774005
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy (LHON)
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
9
States / cities
Phoenix, Arizona • Palo Alto, California • Stanford, California + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2023 · Synced Oct 24, 2026, 11:23 AM EDT
Active, not recruiting No phase listed Observational

Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control

NCT06529146
View directory record Official record
Conditions
Spinocerebellar Ataxias
Interventions
BHV-4157
Drug
Lead sponsor
Biohaven Therapeutics Ltd.
Industry
Eligibility
18 Years to 75 Years
Enrollment
909 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 24, 2026, 11:23 AM EDT