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ClinicalTrials.gov public records Last synced Oct 21, 2026, 7:29 PM EDT

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Showing 769–792 of 1,148 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Not applicable Interventional Results available

Combined CBIT and rTMS to Improve Tourette's Syndrome

NCT04795908
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Transcranial Magnetic Stimulation, Comprehensive behavioral intervention
Device · Behavioral
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 85 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Apr 30, 2024 · Synced Oct 21, 2026, 7:29 PM EDT
Completed Not applicable Interventional

TSC Remote Assessment and Intervention

NCT04698538
View directory record Official record
Conditions
TSC
Interventions
JASPER
Behavioral
Lead sponsor
University of California, Los Angeles
Other
Eligibility
12 Months to 36 Months
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Oct 30, 2024 · Synced Oct 21, 2026, 7:29 PM EDT
Active, not recruiting Phase 2 Interventional Results available

Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia

NCT03190915
View directory record Official record
Conditions
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Interventions
Bone Marrow Aspiration and Biopsy, Trametinib
Procedure · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month to 21 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
57
States / cities
Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 48 more
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Oct 21, 2026, 7:29 PM EDT
Completed Phase 2 Interventional Results available

Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults

NCT02101736
View directory record Official record
Conditions
NF1, Neurofibromatosis, Plexiform Neurofibromas
Interventions
Cabozantinib
Drug
Lead sponsor
University of Alabama at Birmingham
Other
Eligibility
3 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2022
U.S. locations
12
States / cities
Los Angeles, California • Washington D.C., District of Columbia • Chicago, Illinois + 8 more
Source: ClinicalTrials.gov public record
Updated May 24, 2023 · Synced Oct 21, 2026, 7:29 PM EDT
Completed Not applicable Interventional

Efficacy of Computerized Cognitive Training and Stimulant Medication in Neurofibromatosis Type 1

NCT02944032
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
CogmedRM, Mobymax
Other
Lead sponsor
Kristina Hardy
Other
Eligibility
8 Years to 16 Years
Enrollment
103 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 3, 2022 · Synced Oct 21, 2026, 7:29 PM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow-up Study for Patients From AVXS-101-CL-101

NCT03421977
View directory record Official record
Conditions
Spinal Muscular Atrophy 1
Interventions
Onasemnogene Abeparvovec-xioi
Biological
Lead sponsor
Novartis Gene Therapies
Industry
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Sep 22, 2026 · Synced Oct 21, 2026, 7:29 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

NCT01931644
View directory record Official record
Conditions
All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions
Not listed
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 100 Years
Enrollment
17,667 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Oct 21, 2026, 7:29 PM EDT
Completed Phase 2 Interventional Results available

NFX-179 Topical Gel Treatment in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)

NCT04435665
View directory record Official record
Conditions
Neurofibromatosis 1, Cutaneous Neurofibroma
Interventions
NFX-179 Gel, Vehicle Gel
Drug
Lead sponsor
NFlection Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
6
States / cities
Fremont, California • New Brighton, Minnesota • Rochester, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Aug 9, 2022 · Synced Oct 21, 2026, 7:29 PM EDT
Recruiting No phase listed Observational

Longitudinal Impact of Stressors in Adults With Tourette Syndrome

NCT04851678
View directory record Official record
Conditions
Tourette Syndrome
Interventions
None - observational study
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
140 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 21, 2026, 7:29 PM 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, 7:29 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

A Biospecimen and Clinical Data Study on Patients for Drug & Biomarker Discovery

NCT01592552
View directory record Official record
Conditions
Alzheimer's Disease, Multiple Sclerosis, Parkinson's Disease, Huntington's Disease, Amyotrophic Lateral Sclerosis (ALS)
Interventions
Not listed
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2015
U.S. locations
1
States / cities
Sherman Oaks, California
Source: ClinicalTrials.gov public record
Updated Nov 22, 2023 · Synced Oct 21, 2026, 7:29 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Pancreatic Cancer Registry: For Any Person With a Personal or Family History

NCT02886247
View directory record Official record
Conditions
Pancreatic Cancer, ATM, BRCA, Hereditary Cancer
Interventions
Not listed
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
12,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1994 – 2032
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Aug 5, 2026 · Synced Oct 21, 2026, 7:29 PM EDT
Terminated Phase 2 Interventional Results available

Exploratory Study of Danicamtiv in Patients With Primary Dilated Cardiomyopathy (DCM) Due to Genetic Variants or Other Causalities

NCT04572893
View directory record Official record
Conditions
Primary Familial Dilated Cardiomyopathy
Interventions
danicamtiv
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years to 80 Years
Enrollment
41 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
12
States / cities
La Jolla, California • Washington D.C., District of Columbia • Tampa, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated May 21, 2025 · Synced Oct 21, 2026, 7:29 PM EDT
Recruiting Not applicable Interventional

Home-based TDCS (Transcranial Direct Current Stimulation) for Cognitive and Behavioral Symptoms in Huntington's Disease

NCT06843252
View directory record Official record
Conditions
Huntington Disease
Interventions
Transcranial direct current stimulation (tDCS), Sham tDCS
Device
Lead sponsor
The University of Texas Health Science Center at San Antonio
Other
Eligibility
18 Years to 85 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Feb 11, 2026 · Synced Oct 21, 2026, 7:29 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Wearable Technology to Assess Gait Function in SMA and DMD

NCT04193085
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Duchenne Muscular Dystrophy
Interventions
Observational
Other
Lead sponsor
Columbia University
Other
Eligibility
5 Years and older
Enrollment
39 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 23, 2024 · Synced Oct 21, 2026, 7:29 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 21, 2026, 7:29 PM EDT
Completed No phase listed Observational

Apathy in Tourette Syndrome and Changes in Quality of Life

NCT02765178
View directory record Official record
Conditions
Tourette Syndrome, Apathy
Interventions
Children's Motivation Scale, Yale Global Tic Severity Scale (YGTSS), Studies Depression Scale for Children (CES-DC), Gilles de la Tourette Syndrome Quality Of Life scale, Demographic Data
Other
Lead sponsor
University of Florida
Other
Eligibility
12 Years to 16 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 6, 2017 · Synced Oct 21, 2026, 7:29 PM 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 21, 2026, 7:29 PM EDT
Recruiting Not applicable Interventional

A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)

NCT06507748
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
AlgometRx Nociometer(R)
Device
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Year to 120 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
2
States / cities
Washington D.C., District of Columbia • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 21, 2026, 7:29 PM EDT
Active, not recruiting Phase 2 Interventional

Trial of Atezolizumab, Bevacizumab, and Tiragolumab in Patients With Microsatellite Stable, Metastatic Colorectal Cancer

NCT06784947
View directory record Official record
Conditions
Microsatellite Stable (MSS) Colorectal Cancer (CRC), Metastatic Colorectal Cancer (CRC), Colorectal Cancer Stage IV
Interventions
Pre-Treatment Biopsy, On-Treatment Biopsy, Tiragolumab, Atezolizumab, Bevacizumab
Procedure · Drug
Lead sponsor
University of Colorado, Denver
Other
Eligibility
18 Years and older
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
1
States / cities
Aurora, Colorado
Source: ClinicalTrials.gov public record
Updated Oct 2, 2025 · Synced Oct 21, 2026, 7:29 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 21, 2026, 7:29 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN

NCT05309668
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Selumetinib granule formulation, Selumetinib capsule formulation
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
1 Year to 6 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2028
U.S. locations
4
States / cities
Akron, Ohio • Philadelphia, Pennsylvania • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 21, 2026, 7:29 PM EDT
Completed Phase 1 Interventional

Effects of Music Therapy on Huntington's Disease

NCT00178360
View directory record Official record
Conditions
Huntington's Disease
Interventions
Music Therapy
Behavioral
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2010
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Aug 22, 2011 · Synced Oct 21, 2026, 7:29 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

NCT05481879
View directory record Official record
Conditions
Myotonic Dystrophy Type 1 (DM1)
Interventions
DYNE-101, Placebo
Drug
Lead sponsor
Dyne Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
127 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
8
States / cities
Stanford, California • Gainesville, Florida • Indianapolis, Indiana + 5 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 21, 2026, 7:29 PM EDT