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ClinicalTrials.gov public records Last synced Oct 21, 2026, 7:38 AM EDT

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Showing 673–696 of 1,147 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Recruiting Not applicable Interventional

Home-based Transcranial Direct Current Stimulation Open Trial for Behavioral and Cognitive Symptoms in Huntington's Disease

NCT05326451
View directory record Official record
Conditions
Huntington Disease
Interventions
active tDCS
Device
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years to 80 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 11, 2026 · Synced Oct 21, 2026, 7:38 AM EDT
Recruiting Phase 1Phase 2 Interventional

First-in-Human Study of Tersolisib (STX-478) as Monotherapy and in Combination With Other Antineoplastic Agents in Participants With Advanced Solid Tumors

NCT05768139
View directory record Official record
Conditions
Breast Cancer, Solid Tumors, Adult
Interventions
STX-478, Fulvestrant, Ribociclib, Palbociclib, Letrozole, Anastrozole, Exemestane, Tamoxifen, Abemaciclib, Imlunestrant, Metformin
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
18 Years and older
Enrollment
880 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
28
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2026 · Synced Oct 21, 2026, 7:38 AM EDT
Completed Phase 3 Interventional Results available

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

NCT03746522
View directory record Official record
Conditions
Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Interventions
Setmelanotide, Placebo
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
6
States / cities
San Diego, California • Springfield, Massachusetts • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2023 · Synced Oct 21, 2026, 7:38 AM EDT
Active, not recruiting Phase 2 Interventional

A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)

NCT04980872
View directory record Official record
Conditions
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Interventions
Miransertib
Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
2 Years to 120 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
5
States / cities
Atlanta, Georgia • Boston, Massachusetts • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 7, 2025 · Synced Oct 21, 2026, 7:38 AM EDT
Recruiting No phase listed Observational

Study of Skin Tumors in Tuberous Sclerosis

NCT00001975
View directory record Official record
Conditions
Tuberous Sclerosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 90 Years
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 21, 2026, 7:38 AM EDT
Completed No phase listed Observational

Using Positron Emission Tomography to Predict Intracranial Tumor Growth in Neurofibromatosis Type II Patients

NCT01222728
View directory record Official record
Conditions
Neoplasms, Nervous System Disease, Vestibular Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 50 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 21, 2026, 7:38 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

Understanding the Natural History Early in the Course or Presentation of Friedreich Ataxia

NCT06560346
View directory record Official record
Conditions
Friedreich Ataxia, Rare Diseases
Interventions
Geneticlly confirmed disease causing FXN mutatuion, Healthy Control
Other
Lead sponsor
Friedreich's Ataxia Research Alliance
Other
Eligibility
4 Years to 21 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2028
U.S. locations
3
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 8, 2025 · Synced Oct 21, 2026, 7:38 AM EDT
Completed Not applicable Interventional

Functional and Structural Imaging and Motor Control in Spinocerebellar Ataxia

NCT02488031
View directory record Official record
Conditions
Spinocerebellar Ataxia
Interventions
Error-reduction, International Cooperative Ataxia Rating Scale, Scale for the Assessment and Rating of Ataxia, Beck Depression Inventory, 2nd Ed, Stroop, Purdue Pegboard, Brief Test of Attention, 6-minute Walk, Hand Grip Dynamometer, Montreal Cognitive Assessment, Physical Performance Function, Biomechanical Assessments of Dysmetria, Neurophysiological assessment of brain activity, Biomechanical gait analysis
Behavioral
Lead sponsor
University of Florida
Other
Eligibility
21 Years to 85 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated May 13, 2019 · Synced Oct 21, 2026, 7:38 AM EDT
Completed Phase 2 Interventional Results available

Bevacizumab in Treating Patients With Recurrent or Progressive Meningiomas

NCT01125046
View directory record Official record
Conditions
Acoustic Schwannoma, Adult Anaplastic Meningioma, Adult Ependymoma, Adult Grade I Meningioma, Adult Grade II Meningioma, Adult Meningeal Hemangiopericytoma, Adult Papillary Meningioma, Neurofibromatosis Type 1, Neurofibromatosis Type 2, Recurrent Adult Brain Tumor
Interventions
bevacizumab
Biological
Lead sponsor
Northwestern University
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2018
U.S. locations
5
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 22, 2021 · Synced Oct 21, 2026, 7:38 AM EDT
Completed No phase listed Observational

Study of Hepatic Function in Patients With Spinal and Bulbar Muscular Atrophy

NCT02124057
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy (SBMA), Motor Neuron Disease
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years and older
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 18, 2022 · Synced Oct 21, 2026, 7:38 AM EDT
Completed Phase 2Phase 3 Interventional Results available

A Study of the Experimental Drug BKM120 With Paclitaxel in Patients With HER2 Negative, Locally Advanced or Metastatic Breast Cancer, With or Without PI3K Activation

NCT01572727
View directory record Official record
Conditions
Breast Cancer
Interventions
Paclitaxel, BKM120 matching placebo, BKM120
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older · Female only
Enrollment
416 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
21
States / cities
Chandler, Arizona • Phoenix, Arizona • Fayetteville, Arkansas + 17 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2017 · Synced Oct 21, 2026, 7:38 AM EDT
Completed Phase 3 Interventional Results available

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients (PLEO-CMT)

NCT02579759
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease Type 1A
Interventions
PXT3003 dose 1, PXT3003 dose 2, placebo
Drug
Lead sponsor
Pharnext S.C.A.
Other
Eligibility
16 Years to 65 Years
Enrollment
323 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
11
States / cities
Los Angeles, California • New Britain, Connecticut • Gainesville, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Feb 27, 2020 · Synced Oct 21, 2026, 7:38 AM EDT
Completed No phase listed Observational

Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1

NCT04941027
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Not listed
Lead sponsor
Stanford University
Other
Eligibility
40 Years and older
Enrollment
1,046 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
2
States / cities
Baltimore, California • Redwood City, California
Source: ClinicalTrials.gov public record
Updated Dec 2, 2025 · Synced Oct 21, 2026, 7:38 AM EDT
Completed Phase 1 Interventional

Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)

NCT00337636
View directory record Official record
Conditions
Neuronal Ceroid Lipofuscinosis
Interventions
Surgery to implant human CNS stem cells (HuCNS-SC), Medication to suppress the immune system
Procedure · Drug
Lead sponsor
StemCells, Inc.
Industry
Eligibility
18 Months to 12 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Jan 15, 2015 · Synced Oct 21, 2026, 7:38 AM EDT
Completed Phase 1Phase 2 Interventional

A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

NCT02312011
View directory record Official record
Conditions
Myotonic Dystrophy Type 1
Interventions
IONIS-DMPKRx, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
20 Years to 55 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
8
States / cities
Stanford, California • Gainesville, Florida • Kansas City, Kansas + 5 more
Source: ClinicalTrials.gov public record
Updated Dec 6, 2022 · Synced Oct 21, 2026, 7:38 AM EDT
Completed Phase 3 Interventional Results available

PRidopidine's Outcome On Function in Huntington Disease, PROOF- HD

NCT04556656
View directory record Official record
Conditions
Huntington Disease
Interventions
Pridopidine, Placebo
Drug
Lead sponsor
Prilenia
Industry
Eligibility
25 Years and older
Enrollment
499 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
26
States / cities
Davis, California • San Diego, California • Englewood, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated Mar 12, 2025 · Synced Oct 21, 2026, 7:38 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)

NCT02544022
View directory record Official record
Conditions
Neurofibromatosis 1, Plexiform Neurofibromas
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
5 Years and older
Enrollment
476 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2027
U.S. locations
6
States / cities
Washington D.C., District of Columbia • Chicago, Illinois • Baltimore, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 21, 2026, 7:38 AM EDT
Recruiting Not applicable Interventional

Exercise Training in Transthyretin Cardiac Amyloidosis

NCT05797857
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin Cardiac Amyloidosis
Interventions
Exercise Training
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
60 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2025 · Synced Oct 21, 2026, 7:38 AM 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, 7:38 AM EDT
Withdrawn Phase 2 Interventional

Study of CAD-1883 for Spinocerebellar Ataxia

NCT04301284
View directory record Official record
Conditions
Spinocerebellar Ataxias, 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, Spinocerebellar Ataxia Type 17, ARCA1 - Autosomal Recessive Cerebellar Ataxia Type 1
Interventions
CAD-1883, Placebos
Drug
Lead sponsor
Cadent Therapeutics
Industry
Eligibility
18 Years to 75 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
6
States / cities
Long Beach, California • Los Angeles, California • Aurora, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 8, 2021 · Synced Oct 21, 2026, 7:38 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations

NCT05293626
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy (LHON)
Interventions
NR082 Injection, Injection needle
Drug · Device
Lead sponsor
Neurophth Therapeutics Inc
Other
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
3
States / cities
Palo Alto, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 4, 2024 · Synced Oct 21, 2026, 7:38 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 21, 2026, 7:38 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

28-Day Study of SAGE-718 on Functioning Capacity in Participants With Huntington's Disease

NCT05358821
View directory record Official record
Conditions
Huntington Disease
Interventions
SAGE-718, Placebo
Drug
Lead sponsor
Supernus Pharmaceuticals, Inc.
Industry
Eligibility
25 Years to 65 Years
Enrollment
69 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2024
U.S. locations
13
States / cities
Birmingham, Alabama • Los Alamitos, California • Englewood, Colorado + 10 more
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Oct 21, 2026, 7:38 AM EDT
Completed Phase 1 Interventional

A Safety, Pharmacokinetic and Pharmacodynamic Study of Kevetrin in Patients With Advanced Solid Tumors

NCT01664000
View directory record Official record
Conditions
Solid Tumors
Interventions
thioureidobutyronitrile
Drug
Lead sponsor
Cellceutix Corporation
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 24, 2016 · Synced Oct 21, 2026, 7:38 AM EDT