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ClinicalTrials.gov public records Last synced Oct 17, 2026, 7:36 PM EDT

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Showing 481–504 of 1,147 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Recruiting Not applicable Interventional

Brain Outcomes With Lifestyle Change in Down Syndrome

NCT05985486
View directory record Official record
Conditions
Down Syndrome, Alzheimer Disease, Obesity
Interventions
Diet, Health Education
Behavioral
Lead sponsor
University of Kansas Medical Center
Other
Eligibility
18 Years to 64 Years
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Kansas City, Kansas
Source: ClinicalTrials.gov public record
Updated Mar 11, 2026 · Synced Oct 17, 2026, 7:36 PM EDT
Completed Phase 3 Interventional Results available

Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year

NCT03293524
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
GS010, Placebo
Genetic · Drug
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
7
States / cities
Pasadena, California • Aurora, Colorado • Atlanta, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 16, 2026 · Synced Oct 17, 2026, 7:36 PM 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 17, 2026, 7:36 PM 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 17, 2026, 7:36 PM EDT
Active, not recruiting Phase 2 Interventional

9-ING-41 in Patients With Advanced Cancers

NCT03678883
View directory record Official record
Conditions
Cancer, Pancreatic Cancer, Sarcoma, Renal Cancer, Refractory Cancer, Refractory Neoplasm, Refractory Non-Hodgkin Lymphoma, Pancreatic Adenocarcinoma, Resistant Cancer, Neoplasm Metastasis, Neoplasm of Bone, Neoplasm, Breast, Neoplasm of Lung, Neoplasms,Colorectal, Neoplasms Pancreatic, Malignant Glioma, Malignancies, Malignancies Multiple, Bone Metastases, Bone Neoplasm, Bone Cancer, Pancreas Cancer, Pancreatic Neoplasms, Breast Neoplasms, Acute T Cell Leukemia Lymphoma
Interventions
9-ING-41, Gemcitabine - 21 day cycle, Doxorubicin., Lomustine, Carboplatin., Nab paclitaxel., Paclitaxel., Gemcitabine - 28 day cycle, Irinotecan
Drug
Lead sponsor
Actuate Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
40
States / cities
Phoenix, Arizona • Tucson, Arizona • Orange, California + 34 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2026 · Synced Oct 17, 2026, 7:36 PM EDT
Completed Phase 4 Interventional

A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex

NCT05864846
View directory record Official record
Conditions
Tuberous Sclerosis Complex Associated Neuropsychiatric Disease
Interventions
Cannabidiol Oral Solution [Epidiolex]
Drug
Lead sponsor
Jazz Pharmaceuticals
Industry
Eligibility
1 Year to 65 Years
Enrollment
79 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
12
States / cities
Aurora, Colorado • New Haven, Connecticut • Gainesville, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 27, 2026 · Synced Oct 17, 2026, 7:36 PM 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 17, 2026, 7:36 PM EDT
Recruiting Phase 1 Interventional

PrP-targeting siRNA Safety & Mechanism Study

NCT07444580
View directory record Official record
Conditions
Prion Disease
Interventions
PrP-siRNA
Drug
Lead sponsor
Broad Institute of MIT and Harvard
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
5
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 7:36 PM EDT
Recruiting Phase 2 Interventional Accepts healthy volunteers

Cholinergic Integrity in Down Syndrome in Association With Aging, Alzheimer's Disease Pathology, and Cognition

NCT05231798
View directory record Official record
Conditions
Down Syndrome, Down Syndrome, Partial Trisomy 21, Alzheimer Disease
Interventions
[18F]-FEOBV Radiotracer
Drug
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years to 55 Years
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 22, 2026 · Synced Oct 17, 2026, 7:36 PM EDT
Terminated Phase 1 Interventional Results available

Safety, Tolerability and Pharmacokinetics of Cobimetinib in Combination With Pictilisib in Patients With Locally Advanced or Metastatic Solid Tumors

NCT00996892
View directory record Official record
Conditions
Solid Tumors
Interventions
Cobimetinib, Pictilisib
Drug
Lead sponsor
Genentech, Inc.
Industry
Eligibility
18 Years and older
Enrollment
178 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
6
States / cities
Baltimore, Maryland • Boston, Massachusetts • Detroit, Michigan + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 30, 2016 · Synced Oct 17, 2026, 7:36 PM EDT
Completed Early Phase 1 Interventional

Rosuvastatin (Crestor) in Friedreich Ataxia

NCT02705547
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Rosuvastatin
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 65 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 25, 2021 · Synced Oct 17, 2026, 7:36 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases

NCT02532244
View directory record Official record
Conditions
Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Spinal Muscular Atrophy With Respiratory Distress 1, Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Neuromuscular Disease, Peroneal Muscular Atrophy
Interventions
sample collection
Other
Lead sponsor
Nemours Children's Clinic
Other
Eligibility
1 Month and older
Enrollment
230 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2024
U.S. locations
3
States / cities
Wilmington, Delaware • Jacksonville, Florida • Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Dec 19, 2024 · Synced Oct 17, 2026, 7:36 PM EDT
Completed Phase 2 Interventional

EPI-743 in Friedreich's Ataxia Point Mutations

NCT01962363
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
EPI-743
Drug
Lead sponsor
University of South Florida
Other
Eligibility
18 Years to 65 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 29, 2016 · Synced Oct 17, 2026, 7:36 PM EDT
Completed Phase 3 Interventional Results available

RESCUE and REVERSE Long-term Follow-up

NCT03406104
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
GS010, Sham Intravitreal Injection
Genetic · Other
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
3
States / cities
Pasadena, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 17, 2026, 7:36 PM EDT
Completed No phase listed Observational Results available

Neurology Measures in FA Children

NCT03418740
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Years to 18 Years
Enrollment
108 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
3
States / cities
Los Angeles, California • Gainesville, Florida • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 16, 2025 · Synced Oct 17, 2026, 7:36 PM EDT
Terminated Early Phase 1 Interventional Results available

Exploratory Evaluation of AR-42 Histone Deacetylase Inhibitor in the Treatment of Vestibular Schwannoma and Meningioma

NCT02282917
View directory record Official record
Conditions
Vestibular Schwannoma, Meningioma, Acoustic Neuroma, Neurofibromatosis Type 2
Interventions
AR-42
Drug
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
18 Years and older
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2021
U.S. locations
4
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 5, 2025 · Synced Oct 17, 2026, 7:36 PM EDT
Completed Not applicable Interventional Results available

Spinal Cord Stimulation in Spinal Muscular Atrophy

NCT05430113
View directory record Official record
Conditions
Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 4
Interventions
Spinal Cord Stimulator (octopolar Medtronic Vectris Leads)
Device
Lead sponsor
Marco Capogrosso
Other
Eligibility
16 Years to 64 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 16, 2026 · Synced Oct 17, 2026, 7:36 PM EDT
Recruiting No phase listed Observational

Familial Investigations of Childhood Cancer Predisposition

NCT03050268
View directory record Official record
Conditions
Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 17, 2026 · Synced Oct 17, 2026, 7:36 PM EDT
Active, not recruiting Not applicable Interventional

Modification of Threat Interpretation Bias to Reduce Anxiety in Neurodegenerative Movement Disorders (Aim 3)

NCT07213648
View directory record Official record
Conditions
Huntington Disease, Parkinson Disease
Interventions
MindTrails-Movement, Waitlist Control
Behavioral · Other
Lead sponsor
University of Virginia
Other
Eligibility
21 Years and older
Enrollment
136 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Charlottesville, Virginia
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Oct 17, 2026, 7:36 PM 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 17, 2026, 7:36 PM EDT
Terminated No phase listed Observational

Natural History Study of Batten Disease

NCT04644549
View directory record Official record
Conditions
Batten Disease, Neuronal Ceroid Lipofuscinosis CLN6, Neuronal Ceroid Lipofuscinosis CLN3
Interventions
Not listed
Lead sponsor
Emily de los Reyes
Other
Eligibility
Not listed
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2022
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jul 30, 2025 · Synced Oct 17, 2026, 7:36 PM EDT
Completed No phase listed Observational

Assessment of Children With Tic Onset in the Past 6 Months

NCT01177774
View directory record Official record
Conditions
Tourette Syndrome, Tourette's Disorder, Chronic Motor or Vocal Tic Disorder, Transient Tic Disorder, Tic Disorders, Provisional Tic Disorder
Interventions
Not listed
Lead sponsor
Washington University School of Medicine
Other
Eligibility
5 Years to 10 Years
Enrollment
99 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2023
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Nov 27, 2023 · Synced Oct 17, 2026, 7:36 PM EDT
Completed Phase 2 Interventional Results available

Selumetinib in Treating Patients With Neurofibromatosis Type 1 and Cutaneous Neurofibroma

NCT02839720
View directory record Official record
Conditions
Cutaneous Neurofibroma, Neurofibromatosis Type 1, Optic Nerve Glioma
Interventions
Laboratory Biomarker Analysis, Selumetinib Sulfate
Other · Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
2
States / cities
Birmingham, Alabama • Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 6, 2023 · Synced Oct 17, 2026, 7:36 PM EDT
Suspended Phase 1 Interventional

A Phase 1a/1b Study to Evaluate the Safety of EZN-4176, in Adult Patients With Castration-Resistant Prostate Cancer

NCT01337518
View directory record Official record
Conditions
Prostatic Neoplasm
Interventions
EZN-4176
Drug
Lead sponsor
Enzon Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 18, 2012 · Synced Oct 17, 2026, 7:36 PM EDT