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ClinicalTrials.gov public records Last synced Oct 17, 2026, 2:06 PM EDT

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Showing 457–480 of 1,147 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 1Phase 2 Interventional Results available

Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

NCT03140969
View directory record Official record
Conditions
Leber's Congenital Amaurosis
Interventions
QR-110
Drug
Lead sponsor
Laboratoires Thea
Industry
Eligibility
6 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 15, 2024 · Synced Oct 17, 2026, 2:06 PM EDT
Completed Phase 1Phase 2 Interventional

A Phase I/II Study of BGT226 in Adult Patients With Advanced Solid Malignancies Including Patients With Advanced Breast Cancer

NCT00600275
View directory record Official record
Conditions
Solid Tumors, Breast Cancer, Cowden Syndrome
Interventions
BGT226
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
57 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2007
U.S. locations
4
States / cities
Boston, Massachusetts • Las Vegas, Nevada • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Dec 17, 2020 · Synced Oct 17, 2026, 2:06 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)

NCT01031901
View directory record Official record
Conditions
Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
Interventions
Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
13 Years and older
Enrollment
52 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2011
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Feb 27, 2012 · Synced Oct 17, 2026, 2:06 PM EDT
Withdrawn Not applicable Interventional

Treating Sleep/Wake Cycle Disturbances in Basal Ganglia Disorders With Ramelteon

NCT00907595
View directory record Official record
Conditions
Huntington's Disease, Parkinson's Disease, Dementia With Lewy Bodies, Sleep Disorders, Circadian Dysregulation
Interventions
Ramelteon, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
20 Years to 90 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 6, 2012 · Synced Oct 17, 2026, 2:06 PM EDT
Completed Phase 1 Interventional

Sorafenib to Treat Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform Neurofibromas

NCT00727233
View directory record Official record
Conditions
Neurofibromatosis Type I, Plexiform Neurofibroma
Interventions
Nexavar (BAY 43-9006) (Sorafenib), Toxicity, Pharmacokinetics, Pharmacodynamics, Radiographic Evaluation, QOL assessment, Neuropsychological, Bony Toxicity
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 18 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
2
States / cities
Birmingham, Alabama • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 12, 2019 · Synced Oct 17, 2026, 2:06 PM 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 17, 2026, 2:06 PM EDT
Active, not recruiting Phase 2 Interventional Results available

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

NCT03962543
View directory record Official record
Conditions
Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
Interventions
Mirdametinib (PD-0325901) oral capsule or dispersible tablet
Drug
Lead sponsor
SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Industry
Eligibility
2 Years and older
Enrollment
114 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2028
U.S. locations
43
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 37 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2026 · Synced Oct 17, 2026, 2:06 PM EDT
Completed No phase listed Observational

Genetic Evaluation of NF1 and Scoliosis Patients

NCT01776125
View directory record Official record
Conditions
Neurofibromatosis 1, Scoliosis
Interventions
Cheek swab
Other
Lead sponsor
University of Minnesota
Other
Eligibility
6 Years to 65 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2015
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Nov 1, 2019 · Synced Oct 17, 2026, 2:06 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 17, 2026, 2:06 PM EDT
Recruiting Phase 2Phase 3 Interventional Accepts healthy volunteers

A Study of a Potential Disease Modifying Treatment in Individuals at Risk for or With a Type of Early Onset AD Caused by a Genetic Mutation

NCT06647498
View directory record Official record
Conditions
Alzheimers Disease, Dementia, Alzheimers Disease, Familial
Interventions
Remternetug, Matching Placebo (Remternetug)
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
280 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2034
U.S. locations
13
States / cities
Birmingham, Alabama • La Jolla, California • Los Angeles, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Oct 17, 2026, 2:06 PM EDT
Completed Phase 3 Interventional Results available

Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation

NCT02652767
View directory record Official record
Conditions
Optic, Atrophy, Hereditary, Leber
Interventions
GS010, Sham Intravitreal Injection
Biological · Device
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
39 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
3
States / cities
Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 17, 2026, 2:06 PM EDT
Recruiting No phase listed Observational

Trial Readiness and Endpoint Assessment in Pediatric Myotonic Dystrophy Extension

NCT06747884
View directory record Official record
Conditions
Congenital Myotonic Dystrophy, Childhood Myotonic Dystrophy, Myotonic Dystrophy
Interventions
Not listed
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
3 Years to 17 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
3
States / cities
Little Rock, Arkansas • Kansas City, Kansas • Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Aug 11, 2026 · Synced Oct 17, 2026, 2:06 PM EDT
Completed Not applicable Interventional

TicHelper: A Computerized Comprehensive Behavioral Intervention for Tics (CBIT)

NCT02413216
View directory record Official record
Conditions
Tourette's Disorder, Persistent (Chronic) Motor or Vocal Tic Disorder
Interventions
TicHelper, Internet Based Resources
Behavioral · Other
Lead sponsor
University of Utah
Other
Eligibility
8 Years to 18 Years
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
2
States / cities
Salt Lake City, Utah • Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Dec 18, 2017 · Synced Oct 17, 2026, 2:06 PM EDT
Completed Not applicable Interventional

Body Weight Support Harness System in Spinal Muscular Atrophy

NCT05715749
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I, Spinal Muscular Atrophy Type II
Interventions
In-home body weight support harness system
Other
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
Not listed
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 22, 2024 · Synced Oct 17, 2026, 2:06 PM 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 17, 2026, 2:06 PM EDT
Completed No phase listed Observational

Natural History Study of FDXR Mutation-related Mitochondriopathy

NCT04580979
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
33 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 17, 2026, 2:06 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Charcot-Marie-Tooth Disease (CMT) Biological Sample Collection for IPSC Generation and Biobanking

NCT06203093
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Healthy
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
5 Years and older
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Oct 17, 2026, 2:06 PM EDT
Terminated Phase 1 Interventional

Glucagon Receptor Inhibition to Enable Breast Cancer Patients to Benefit From PI3K Inhibitor Therapy (REMD-477)

NCT04330625
View directory record Official record
Conditions
Hyperglycemia Drug Induced
Interventions
REMD-477
Biological
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 8, 2022 · Synced Oct 17, 2026, 2:06 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Neuroimaging of Dystonia

NCT02046447
View directory record Official record
Conditions
Primary Cervical Dystonia, DYT 1 Dystonia
Interventions
Primary Cervical Dystonia (Trihexyphenidyl), Controls Primary Cervical Dystonia (Trihexyphenidyl), DYT 1 Dystonia (Healthy Control), DYT 1 Dystonia
Drug · Other
Lead sponsor
University of Florida
Other
Eligibility
7 Years to 70 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Dec 22, 2017 · Synced Oct 17, 2026, 2:06 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 17, 2026, 2:06 PM EDT
Active, not recruiting No phase listed Observational

ESIS in Pediatric DRE

NCT05469373
View directory record Official record
Conditions
Epilepsy
Interventions
Not listed
Lead sponsor
Sarah Katie Ihnen
Other
Eligibility
1 Year to 30 Years
Enrollment
86 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 1, 2026 · Synced Oct 17, 2026, 2:06 PM EDT
Completed No phase listed Observational

Natural History and Functional Status Study of Patients With Lafora Disease

NCT03876522
View directory record Official record
Conditions
Lafora Disease
Interventions
Not listed
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
5 Years and older
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
2
States / cities
Los Angeles, California • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Sep 28, 2022 · Synced Oct 17, 2026, 2:06 PM EDT
Active, not recruiting Phase 2 Interventional

Efficacy and Safety of Gemlapodect (NOE-105) in Adults and Adolescents With Tourette Syndrome

NCT06315751
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Gemlapodect, Placebo
Drug
Lead sponsor
Noema Pharma AG
Industry
Eligibility
18 Years and older
Enrollment
164 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
31
States / cities
Sun City, Arizona • Bellflower, California • Glendale, California + 28 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2026 · Synced Oct 17, 2026, 2:06 PM EDT
Completed Early Phase 1 Interventional

Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1

NCT00657202
View directory record Official record
Conditions
Neurofibromatosis Type 1, Cutaneous Neurofibromas
Interventions
Ranibizumab
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2013
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 7, 2016 · Synced Oct 17, 2026, 2:06 PM EDT