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ClinicalTrials.gov public records Last synced Oct 24, 2026, 2:46 AM EDT

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Showing 1033–1056 of 1,148 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 1Phase 2 Interventional

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

NCT05517603
View directory record Official record
Conditions
Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
Interventions
AJ201, Placebo
Drug
Lead sponsor
AnnJi Pharmaceutical Co., Ltd.
Industry
Eligibility
18 Years and older · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
6
States / cities
Orange, California • Palo Alto, California • Jacksonville, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 15, 2024 · Synced Oct 24, 2026, 2:46 AM EDT
Completed No phase listed Observational

Magnetic Resonance Spectroscopy to Evaluate Tourette s Syndrome

NCT00030953
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
14 Years to 65 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2015
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 17, 2019 · Synced Oct 24, 2026, 2:46 AM EDT
Completed Phase 2 Interventional Results available

Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors

NCT00304083
View directory record Official record
Conditions
Neurofibromatosis Type 1, Sarcoma
Interventions
filgrastim, doxorubicin hydrochloride, etoposide, ifosfamide, conventional surgery, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
Sarcoma Alliance for Research through Collaboration
Other
Eligibility
Not listed
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2014
U.S. locations
17
States / cities
Birmingham, Alabama • Santa Monica, California • Chicago, Illinois + 13 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2018 · Synced Oct 24, 2026, 2:46 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 24, 2026, 2:46 AM EDT
Completed No phase listed Observational

Measurement of Frataxin mRNA in Biofluids

NCT06496451
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Spinal tap for CSF collection, Venipuncture for blood sample
Other
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jan 16, 2026 · Synced Oct 24, 2026, 2:46 AM EDT
Completed Phase 2 Interventional Results available

A Phase II Study of the mTOR Inhibitor Sirolimus in Neurofibromatosis Type 1 Related Plexiform Neurofibromas

NCT00634270
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Sirolimus
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
3 Years to 75 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
9
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 6 more
Source: ClinicalTrials.gov public record
Updated Nov 17, 2017 · Synced Oct 24, 2026, 2:46 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III

NCT00439569
View directory record Official record
Conditions
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Years to 11 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
5
States / cities
Stanford, California • St Louis, Missouri • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2010 · Synced Oct 24, 2026, 2:46 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Analysis of Lumbar Spine Stenosis Specimens for Identification of Transthyretin Cardiac Amyloidosis

NCT06034405
View directory record Official record
Conditions
Lumbar Spinal Stenosis, Cardiac Amyloidosis, ATTR Amyloidosis Wild Type, ATTR Gene Mutation, ATTRV122I Amyloidosis, Cardiomyopathy, Hypertrophic
Interventions
Tc99-PYP or Tc99-HDMP Scan
Diagnostic Test
Lead sponsor
Columbia University
Other
Eligibility
50 Years and older
Enrollment
1,663 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2028
U.S. locations
6
States / cities
Beverly Hills, California • Boston, Massachusetts • Rochester, Minnesota + 2 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 24, 2026, 2:46 AM EDT
Completed Not applicable Interventional

Physiological Investigations of Movement Disorders

NCT01019343
View directory record Official record
Conditions
Parkinson's Disease, Tourette's Syndrome, Tic Disorders, Dystonia, Movement Disorders
Interventions
Transcranial Direct Current Stimulation (tDCS), Eye-tracking Device, Pulsed vibrator, Gait Trainer Treadmill (GTT), Standard psychiatric scales SCID-I, YBOCS and SCI-OBS-lifetime and cognitive scales MMSE and MOCA, iMobility, QMAT, Treadmill Test, Arm Intellistretch Device, Transcranial Sonography (TCS), Behavioral tasks, TMS single/paired pulse, MEG, EEG, MRI techniques, Peripheral Nerve Stimulation, EMG, Trancutaneous spinal direct current stimulation (tsDCS)
Procedure · Device · Other + 1 more
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years and older
Enrollment
1,273 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 24, 2026, 2:46 AM EDT
Completed Phase 2 Interventional

Effects of Lithium and Divalproex'on Brain-Derived Neurotrophic Factor in Huntington's Disease

NCT00095355
View directory record Official record
Conditions
Huntington's Disease
Interventions
Lithium, Divalproex
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
35 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 24, 2026, 2:46 AM EDT
Completed No phase listed Observational

Study of the Neurobiology of Tourette Syndrome and Related Disorders

NCT00004325
View directory record Official record
Conditions
Tourette Syndrome, Obsessive Compulsive Disorder
Interventions
Not listed
Lead sponsor
National Center for Research Resources (NCRR)
NIH
Eligibility
12 Years to 45 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1988
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 24, 2026, 2:46 AM EDT
Completed Phase 2 Interventional Results available

Atomoxetine and Huntington's Disease

NCT00368849
View directory record Official record
Conditions
Huntington Disease, Chorea
Interventions
atomoxetine, Matching Placebo
Drug
Lead sponsor
University of Iowa
Other
Eligibility
18 Years to 65 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Dec 20, 2012 · Synced Oct 24, 2026, 2:46 AM EDT
Completed Phase 2 Interventional Results available

A Phase II Study of Single Agent MEK162 in Patients With Advanced Melanoma

NCT01320085
View directory record Official record
Conditions
BRAF or NRAS Mutant Metastatic Melanoma
Interventions
MEK162
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2023
U.S. locations
10
States / cities
Fayetteville, Arkansas • Tampa, Florida • Portland, Oregon + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2024 · Synced Oct 24, 2026, 2:46 AM EDT
Terminated Phase 3 Interventional Results available

A Study of Nivolumab-relatlimab Fixed-dose Combination Versus Regorafenib or TAS-102 in Participants With Later-lines of Metastatic Colorectal Cancer

NCT05328908
View directory record Official record
Conditions
Colorectal Neoplasms
Interventions
Nivolumab-relatlimab FDC, Regorafenib, TAS-102
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years and older
Enrollment
769 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
20
States / cities
Springdale, Arkansas • Los Angeles, California • Norwich, Connecticut + 17 more
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 24, 2026, 2:46 AM EDT
Completed Not applicable Interventional

Resiliency Training for Patients With Neurofibromatosis Via Videoconferencing With Skype

NCT02298270
View directory record Official record
Conditions
Neurofibromatosis, Neurofibromatosis I, Neurofibromatosis 2, Schwannomatosis
Interventions
The Relaxation Response Resiliency Program (3RP) via Skype
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 23, 2016 · Synced Oct 24, 2026, 2:46 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Advancing Understanding of Transportation Options

NCT04141891
View directory record Official record
Conditions
Diabetic Retinopathy, Macular Degeneration, Glaucoma, Retinitis Pigmentosa, Vision Disorders, Acute Coronary Syndrome, Implantable Defibrillator User, Congestive Heart Failure, Hypertrophic Obstructive Cardiomyopathy, Orthostatic Hypotension, Syncope, Presyncope, Narcolepsy, Dementia, Multiple Sclerosis, Parkinson Disease, Brain Injuries, Spinal Cord Injuries, Stroke, Vertigo, Dizziness, Seizures, Substance Use, Insulin Dependent Diabetes Mellitus, Arthritis, Foot--Abnormalities, Chronic Obstructive Pulmonary Disease, Obstructive Sleep Apnea, End Stage Renal Disease, Sleep Apnea, Insomnia, Restless Legs Syndrome
Interventions
Driving Decision Aid, Older Drivers Website
Behavioral
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years and older
Enrollment
529 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Indianapolis, Indiana
Source: ClinicalTrials.gov public record
Updated Feb 21, 2025 · Synced Oct 24, 2026, 2:46 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Feasibility of [11C]Acetate-PET in LAM and TSC

NCT05467397
View directory record Official record
Conditions
Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
Interventions
[11C]acetate
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older · Female only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 21, 2026 · Synced Oct 24, 2026, 2:46 AM EDT
Recruiting Phase 1 Interventional

9-ING-41 Combined With Retifanlimab, Plus Modified FOLFIRINOX for Patients With Advanced Pancreatic Adenocarcinoma (RiLEY)

NCT06896188
View directory record Official record
Conditions
Pancreatic Adenocarcinoma
Interventions
Retifanlimab, Chemotherapy, 9-ING-41
Drug
Lead sponsor
Anwaar Saeed
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 24, 2026, 2:46 AM EDT
Completed No phase listed Observational

Natural History Study of SLC25A46 Mutation-related Mitochondriopathy

NCT04594590
View directory record Official record
Conditions
Neurodegenerative Disease, Hereditary, Mitochondrial Diseases, Optic Atrophy
Interventions
Mutation analysis
Genetic
Lead sponsor
State University of New York at Buffalo
Other
Eligibility
0 Years to 65 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Buffalo, New York
Source: ClinicalTrials.gov public record
Updated Mar 8, 2024 · Synced Oct 24, 2026, 2:46 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Prospective Evaluation of Infants With Spinal Muscular Atrophy:

NCT02831296
View directory record Official record
Conditions
Spinal Muscular Atrophy
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 2, 2020 · Synced Oct 24, 2026, 2:46 AM EDT
Active, not recruiting Phase 3 Interventional

Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

NCT04988386
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin-Related
Interventions
Acoramidis (AG10)
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
389 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
26
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 21 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Oct 24, 2026, 2:46 AM EDT
Active, not recruiting No phase listed Observational

Natural History Study of Patients With Neurofibromatosis Type I

NCT00924196
View directory record Official record
Conditions
Neurofibromatosis Type 1, Malignant Peripheral Nerve Sheath Tumor, Plexiform Neurofibroma, Optic Glioma, Neurofibroma
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
4 Weeks and older
Enrollment
259 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 24, 2026, 2:46 AM EDT
Completed No phase listed Observational

Evaluation and Follow-up of Individuals With Obsessive-Compulsive Disorder and Related Conditions

NCT00004550
View directory record Official record
Conditions
Anorexia Nervosa, Obsessive Compulsive Disorder, Tourette Syndrome, Trichotillomania
Interventions
Not listed
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
18 Years and older
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2008
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 24, 2026, 2:46 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

NCT00439218
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Months to 48 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
5
States / cities
Stanford, California • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 9, 2010 · Synced Oct 24, 2026, 2:46 AM EDT