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ClinicalTrials.gov public records Last synced Oct 19, 2026, 9:33 AM EDT

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Showing 577–600 of 1,147 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 2 Interventional Results available

An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy

NCT03921528
View directory record Official record
Conditions
Spinal Muscular Atrophy, Spinal Muscular Atrophy Type 3, Spinal Muscular Atrophy Type 2, SMA, Neuromuscular Diseases, Muscular Atrophy, Atrophy, Muscular Atrophy, Spinal, Neuromuscular Manifestations
Interventions
SRK-015
Biological
Lead sponsor
Scholar Rock, Inc.
Industry
Eligibility
2 Years to 21 Years
Enrollment
58 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
11
States / cities
Phoenix, Arizona • Palo Alto, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 2, 2024 · Synced Oct 19, 2026, 9:33 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 19, 2026, 9:33 AM EDT
Terminated Phase 3 Interventional Results available

A Study to Evaluate the Safety and Tolerability of SAGE-718 in Participants With Huntington's Disease

NCT05655520
View directory record Official record
Conditions
Huntington's Disease
Interventions
SAGE-718
Drug
Lead sponsor
Supernus Pharmaceuticals, Inc.
Industry
Eligibility
25 Years to 65 Years
Enrollment
153 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
29
States / cities
Birmingham, Alabama • Little Rock, Arkansas • La Jolla, California + 25 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Oct 19, 2026, 9:33 AM EDT
Not yet recruiting Phase 2 Interventional

GV101 for Friedreich's Ataxia (FA) Treatment

NCT07870590
View directory record Official record
Conditions
Friedreich's Ataxia
Interventions
GV101, Placebo
Drug
Lead sponsor
Graviton Bioscience Corporation
Industry
Eligibility
16 Years to 40 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 15, 2026 · Synced Oct 19, 2026, 9:33 AM EDT
Completed Phase 2 Interventional Results available

Interferon Gamma-1b in Friedreich Ataxia (FRDA)

NCT01965327
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Interferon Gamma-1b
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
5 Years to 17 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 13, 2021 · Synced Oct 19, 2026, 9:33 AM EDT
Completed Phase 1 Interventional

DPCP to Treat Cutaneous Neurofibromas Associated With NF1

NCT05438290
View directory record Official record
Conditions
Cutaneous Neurofibroma
Interventions
DPCP
Drug
Lead sponsor
Nicholas Gulati
Other
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated May 6, 2024 · Synced Oct 19, 2026, 9:33 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 19, 2026, 9:33 AM EDT
No Longer Available No phase listed Expanded access

3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)

NCT02012933
View directory record Official record
Conditions
Lambert-Eaton Myasthenic Syndrome (LEMS), Congenital Myasthenia (CM)
Interventions
3,4-diaminopyridine
Drug
Lead sponsor
Oregon Health and Science University
Other
Eligibility
2 Years and older
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Dec 11, 2019 · Synced Oct 19, 2026, 9:33 AM EDT
Enrolling by invitation No phase listed Observational

F 18 T807 Tau PET Imaging in Dominantly Inherited Alzheimer's Network (DIAN Project)

NCT02414178
View directory record Official record
Conditions
Alzheimer Disease
Interventions
F 18 T807
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
18 Years and older
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Oct 19, 2026, 9:33 AM EDT
Completed Phase 1Phase 2 Interventional Results available

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

NCT02485899
View directory record Official record
Conditions
Jansky-Bielschowsky Disease, Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, CLN2 Disease, CLN2 Disorder
Interventions
BMN 190, Intracerebroventricular (ICV) access device
Biological · Device
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 16 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2020
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Aug 24, 2022 · Synced Oct 19, 2026, 9:33 AM EDT
Recruiting No phase listed Observational

NYSCF Scientific Discovery Biobank

NCT06203106
View directory record Official record
Conditions
ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Batten Disease, Corticobasal Degeneration, Dementia, Frontotemporal Dementia, Huntington Disease, Lewy Body Disease, Multiple Sclerosis, Multiple System Atrophy, Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, INAD, Diabetes, Diabetes Mellitus, Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Macular Degeneration, Ovarian Cancer, Cervical Cancer, Uterine Cancer, Vaginal Cancer, Vulvar Cancer, PTSD, Post Traumatic Stress Disorder
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
30 Days and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2045
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 3, 2025 · Synced Oct 19, 2026, 9:33 AM 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 19, 2026, 9:33 AM EDT
Terminated Phase 1 Interventional

Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease

NCT01552434
View directory record Official record
Conditions
Advanced Malignant Neoplasm, Castleman Disease, Digestive System Carcinoma, Erdheim-Chester Disease, Lip and Oral Cavity Carcinoma, Lymphangioleiomyomatosis, Malignant Endocrine Neoplasm, Malignant Female Reproductive System Neoplasm, Malignant Male Reproductive System Neoplasm, Malignant Neoplasm, Malignant Respiratory Tract Neoplasm, Malignant Thoracic Neoplasm, Malignant Urinary System Neoplasm, Mesothelial Neoplasm, Metastatic Malignant Neoplasm, Metastatic Urothelial Carcinoma, Neurofibromatosis Type 2, Recurrent Adult Soft Tissue Sarcoma, Recurrent Breast Carcinoma, Recurrent Childhood Soft Tissue Sarcoma, Recurrent Digestive System Carcinoma, Recurrent Female Reproductive System Carcinoma, Recurrent Male Reproductive System Carcinoma, Recurrent Malignant Neoplasm, Recurrent Pharyngeal Carcinoma, Recurrent Thyroid Gland Carcinoma, Refractory Malignant Neoplasm, Soft Tissue Neoplasm, Stage III Breast Cancer AJCC v7, Stage III Pharyngeal Cancer, Stage IIIA Breast Cancer AJCC v7, Stage IIIB Breast Cancer AJCC v7, Stage IIIC Breast Cancer AJCC v7, Stage IV Breast Cancer AJCC v6 and v7, Stage IV Pharyngeal Cancer, Stage IVA Pharyngeal Cancer, Stage IVB Pharyngeal Cancer, Stage IVC Pharyngeal Cancer, Thyroid Gland Neoplasm
Interventions
Bevacizumab, Cetuximab, Laboratory Biomarker Analysis, Pharmacological Study, Temsirolimus, Valproic Acid
Biological · Other · Drug
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
Not listed
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2026
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 15, 2026 · Synced Oct 19, 2026, 9:33 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1

NCT06844214
View directory record Official record
Conditions
Myotonic Dystrophy
Interventions
SAR446268
Biological
Lead sponsor
Sanofi
Industry
Eligibility
10 Years to 55 Years
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
5
States / cities
Gainesville, Florida • Tampa, Florida • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 21, 2026 · Synced Oct 19, 2026, 9:33 AM EDT
Recruiting Not applicable Interventional

Neuromodulation to Enhance Motor Function in HSP

NCT07417943
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
transcutaneous spinal cord stimulation
Device
Lead sponsor
Rahul Sachdeva
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Oct 19, 2026, 9:33 AM EDT
Withdrawn Not applicable Interventional Accepts healthy volunteers

Telemedicine Clinical Trial for Cognitive Behavioral Therapy in Familial Dysautonomia

NCT03911063
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
CBT sessions, Talking Sessions
Behavioral
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years to 80 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 28, 2020 · Synced Oct 19, 2026, 9:33 AM EDT
Active, not recruiting No phase listed Observational

Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer

NCT04273243
View directory record Official record
Conditions
CLN6, Batten Disease
Interventions
AT-GTX-501
Genetic
Lead sponsor
Emily de los Reyes
Other
Eligibility
12 Months and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2027
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 2, 2025 · Synced Oct 19, 2026, 9:33 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease

NCT06826612
View directory record Official record
Conditions
Huntington Disease
Interventions
SPK-10001, Placebo Surgery Control
Genetic · Other
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
25 Years to 65 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2035
U.S. locations
5
States / cities
Boston, Massachusetts • Cincinnati, Ohio • Columbus, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated May 5, 2026 · Synced Oct 19, 2026, 9:33 AM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Wearable Sensors for Quantitative Assessment of Motor Impairment in Huntington's Disease Huntington's Disease

NCT03599076
View directory record Official record
Conditions
Huntington Disease
Interventions
HD Wear
Other
Lead sponsor
BioSensics
Industry
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2019
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Jan 24, 2019 · Synced Oct 19, 2026, 9:33 AM EDT
Completed No phase listed Observational

Development of the Virtual Unified Huntington's Disease Rating Scale

NCT05748288
View directory record Official record
Conditions
Huntington Disease
Interventions
Not listed
Lead sponsor
Huntington Study Group
Network
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
17
States / cities
Birmingham, Alabama • Little Rock, Arkansas • La Jolla, California + 13 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2023 · Synced Oct 19, 2026, 9:33 AM EDT
Completed No phase listed Observational

Myotubular Myopathy Genetic Testing Study

NCT01817946
View directory record Official record
Conditions
Myotubular Myopathy
Interventions
Genetic Testing
Other
Lead sponsor
Cure CMD
Other
Eligibility
30 Days and older
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Mar 7, 2018 · Synced Oct 19, 2026, 9:33 AM EDT
Recruiting Phase 2Phase 3 Interventional

Open-label, Long-term Safety, Efficacy, and Pharmacokinetics Study of Vibegron in Pediatric Subjects 2 Years to < 18 Years of Age With NDO and on CIC

NCT05491525
View directory record Official record
Conditions
Neurogenic Detrusor Overactivity
Interventions
Vibegron
Drug
Lead sponsor
Urovant Sciences GmbH
Industry
Eligibility
2 Years to 17 Years
Enrollment
71 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2030
U.S. locations
11
States / cities
Little Rock, Arkansas • Orange, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 19, 2026, 9:33 AM EDT
Terminated No phase listed Observational Accepts healthy volunteers

Investigation of Neurofeedback With Real-Time fMRI in Healthy Volunteers and Patients With Hyperkinetic Movement Disorders

NCT00885040
View directory record Official record
Conditions
Movement Disorder, Tourette Syndrome, Healthy Volunteer
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 60 Years
Enrollment
59 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2019
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jun 24, 2019 · Synced Oct 19, 2026, 9:33 AM EDT
Recruiting No phase listed Observational

Tuberous Sclerosis Complex and Lymphangioleiomyomatosis Pregnancy Registry (TSC-LAM Registry)

NCT06160310
View directory record Official record
Conditions
Tuberous Sclerosis Complex, Lymphangioleiomyomatosis
Interventions
Not listed
Lead sponsor
David M. Ritter
Other
Eligibility
Female only
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 23, 2026 · Synced Oct 19, 2026, 9:33 AM EDT