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ClinicalTrials.gov public records Last synced Oct 23, 2026, 7:12 AM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 937–960 of 1,148 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Evaluate Bioavailability and Food Effect of Selumetinib (AZD6244) in Healthy Male Participants

NCT03649165
View directory record Official record
Conditions
Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs), Healthy Participants
Interventions
Treatment A, Treatment B, Treatment C, Treatment D, Acetaminophen
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 45 Years · Male only
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Nov 8, 2018 · Synced Oct 23, 2026, 7:12 AM EDT
Completed No phase listed Observational

Official Record of Patients Diagnosed With Lymphangioleiomyomatosis (LAM)

NCT00001869
View directory record Official record
Conditions
Leiomyomatosis
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
Not listed
Enrollment
400 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 23, 2026, 7:12 AM EDT
Active, not recruiting Not applicable Interventional Results available

Tourette Deep Brain Stimulation (DBS) Target Detection & Suppression

NCT05371041
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Medtronic Percept Neurostimulator
Device
Lead sponsor
University of Florida
Other
Eligibility
18 Years to 85 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 25, 2026 · Synced Oct 23, 2026, 7:12 AM EDT
Completed Phase 3 Interventional Results available

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

NCT02319005
View directory record Official record
Conditions
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Familial Transthyretin Cardiac Amyloidosis
Interventions
Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 90 Years
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
41
States / cities
Bakersfield, California • Beverly Hills, California • La Mesa, California + 30 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2018 · Synced Oct 23, 2026, 7:12 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 23, 2026, 7:12 AM EDT
Recruiting Phase 1 Interventional

PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas

NCT06961565
View directory record Official record
Conditions
NF1 Mutation, Neurofibroma Plexiform, Neurofibroma, Plexiform, Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs), Neurofibromatosis Type 1 (NF1)
Interventions
PAS-004 Tablets
Drug
Lead sponsor
Pasithea Therapeutics Corp.
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Birmingham, Alabama
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Oct 23, 2026, 7:12 AM EDT
Completed Phase 1 Interventional

A Safety, Pharmacokinetic and Pharmacodynamic Study of Kevetrin in Patients With Advanced Solid Tumors

NCT01664000
View directory record Official record
Conditions
Solid Tumors
Interventions
thioureidobutyronitrile
Drug
Lead sponsor
Cellceutix Corporation
Industry
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 24, 2016 · Synced Oct 23, 2026, 7:12 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

NCT03692312
View directory record Official record
Conditions
Congenital Myotonic Dystrophy
Interventions
Tideglusib, Placebo
Drug
Lead sponsor
AMO Pharma Limited
Industry
Eligibility
6 Years to 16 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
9
States / cities
Little Rock, Arkansas • Los Angeles, California • Palo Alto, California + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2025 · Synced Oct 23, 2026, 7:12 AM EDT
Completed Phase 1Phase 2 Interventional Results available

L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1

NCT01733407
View directory record Official record
Conditions
Hereditary Sensory and Autonomic Neuropathy Type I
Interventions
L-serine, placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
2
States / cities
Boston, Massachusetts • Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Sep 12, 2018 · Synced Oct 23, 2026, 7:12 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Complex Eye Movements in Parkinson's Disease and Related Movement Disorders

NCT04925622
View directory record Official record
Conditions
Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, Corticobasal Degeneration, Parkinsonian Disorders, Essential Tremor, Vascular Parkinsonism, Multiple System Atrophy, Parkinson Variant, Parkinsonian Syndrome, Huntington Disease
Interventions
Complex eye movement exam
Diagnostic Test
Lead sponsor
Saccadous, Inc.
Other
Eligibility
30 Years and older
Enrollment
90 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated May 4, 2022 · Synced Oct 23, 2026, 7:12 AM EDT
Completed No phase listed Observational

Studies in Patients With Tuberous Sclerosis Complex

NCT03276195
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
Translational Genomics Research Institute
Other
Eligibility
Not listed
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 29, 2023 · Synced Oct 23, 2026, 7:12 AM EDT
Completed Phase 2 Interventional

Creatine Safety and Tolerability in Premanifest HD: PRECREST

NCT00592995
View directory record Official record
Conditions
Huntington Disease
Interventions
Creatine monohydrate, Placebo
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
26 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Charlestown, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 10, 2014 · Synced Oct 23, 2026, 7:12 AM EDT
Withdrawn Early Phase 1 Interventional

D-cycloserine Augmented Treatment for Youth With Tic Disorders

NCT04357951
View directory record Official record
Conditions
Tourette Syndrome
Interventions
d-cycloserine, Placebo pill, Behavior Therapy
Drug · Behavioral
Lead sponsor
Johns Hopkins University
Other
Eligibility
8 Years to 17 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 14, 2023 · Synced Oct 23, 2026, 7:12 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)

NCT03094832
View directory record Official record
Conditions
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
Interventions
Miransertib
Drug
Lead sponsor
ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Industry
Eligibility
2 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
6
States / cities
Atlanta, Georgia • Chicago, Illinois • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2023 · Synced Oct 23, 2026, 7:12 AM EDT
Recruiting Phase 1Phase 2 Interventional

A Study to Assess the Effects of ACI-24.060 in Alzheimer's Disease and in Down Syndrome (ABATE Study)

NCT05462106
View directory record Official record
Conditions
Amyloid Plaque, Beta-Amyloid, DSAD, Prodromal Alzheimer's Disease, Alzheimer's Disease
Interventions
Placebo (Study Part 1a), ACI-24.060 at Dose A in Study Part 1a, ACI-24.060 at Dose B in Study Part 1a, ACI-24.060 at Dose C in Study Part 1a, ACI-24.060 with an additional adjuvant at Dose D in Study Part 1b, Placebo (Study Part 2), ACI-24.060 at Dose A in Study Part 2, ACI-24.060 at Dose B in Study Part 2, ACI-24.060 at Dose C in Study Part 2, Placebo (Study Part 1b), ACI-24.060 with an additional adjuvant at Dose E in Study Part 1b
Biological
Lead sponsor
AC Immune SA
Industry
Eligibility
35 Years to 85 Years
Enrollment
304 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
13
States / cities
Phoenix, Arizona • Lady Lake, Florida • Orlando, Florida + 9 more
Source: ClinicalTrials.gov public record
Updated Jul 10, 2026 · Synced Oct 23, 2026, 7:12 AM EDT
Terminated Phase 3 Interventional

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

NCT00654433
View directory record Official record
Conditions
Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions
ALD-101
Biological
Lead sponsor
Aldagen
Industry
Eligibility
Up to 16 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2011
U.S. locations
3
States / cities
Los Angeles, California • New York, New York • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 8, 2014 · Synced Oct 23, 2026, 7:12 AM EDT
Active, not recruiting No phase listed Observational

Longitudinal Assessment of Atypical Tripeptidyl Peptidase 1 Enzyme Deficiency Patients

NCT04098211
View directory record Official record
Conditions
Neuronal Ceroid-Lipofuscinoses, Neuronal Ceroid Lipofuscinosis CLN2, Spinocerebellar Ataxia, Autosomal Recessive 7
Interventions
Not listed
Lead sponsor
Children's Hospital of Orange County
Other
Eligibility
4 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
1
States / cities
Orange, California
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Oct 23, 2026, 7:12 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease

NCT04120493
View directory record Official record
Conditions
Huntington's Disease
Interventions
intra-striatal rAAV5-miHTT, Imitation (sham) surgery
Genetic · Other
Lead sponsor
UniQure Biopharma B.V.
Industry
Eligibility
25 Years to 65 Years
Enrollment
43 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2029
U.S. locations
12
States / cities
Birmingham, Alabama • Tucson, Arizona • San Francisco, California + 9 more
Source: ClinicalTrials.gov public record
Updated Oct 21, 2025 · Synced Oct 23, 2026, 7:12 AM EDT
Completed Not applicable Interventional

Clinical Trial of a Serious Game for Individuals With SCI/D

NCT02341950
View directory record Official record
Conditions
Spinal Cord Injury, Spinal Cord Involvement, Spina Bifida, Transverse Myelitis, Polio and Post-polio Syndrome, Syringomyelia, Spinal Stenosis, Spinal Neoplasms, Spinal Cord Diseases
Interventions
SCI Hard
Behavioral
Lead sponsor
University of Michigan
Other
Eligibility
13 Years to 29 Years
Enrollment
184 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2017
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Feb 22, 2019 · Synced Oct 23, 2026, 7:12 AM EDT
Completed Early Phase 1 Interventional

Methylprednisolone Treatment of Friedreich Ataxia

NCT02424435
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Methylprednisolone
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
5 Years and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 25, 2021 · Synced Oct 23, 2026, 7:12 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient

NCT07223632
View directory record Official record
Conditions
Charcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD)
Interventions
VCA-894A
Drug
Lead sponsor
Vanda Pharmaceuticals
Industry
Eligibility
Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 23, 2026, 7:12 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Sensorimotor and Psychosocial Trajectories in Adolescents With Tic Disorder

NCT06576726
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorder
Interventions
This study involves no intervention.
Other
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
11 Years to 17 Years
Enrollment
351 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2030
U.S. locations
1
States / cities
Nashvile, Tennessee
Source: ClinicalTrials.gov public record
Updated Nov 24, 2025 · Synced Oct 23, 2026, 7:12 AM EDT
Completed Not applicable Interventional Results available

Electronic-health Application To Measure Outcomes REmotely Clinical Trial

NCT02418546
View directory record Official record
Conditions
Neurodegenerative Disease, Weight Loss, Amyotrophic Lateral Sclerosis, Parkinson's Disease, Huntington's Disease, Cachexia
Interventions
In-Person Nutritional Counseling by a Registered Dietitian, Nutritional counseling using an e-Health Application
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years and older
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 20, 2020 · Synced Oct 23, 2026, 7:12 AM EDT
Completed Phase 4 Interventional Results available

Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis

NCT00028262
View directory record Official record
Conditions
Infantile Neronal Ceroid Lipofuscinosis
Interventions
Cystagon
Drug
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
6 Months to 3 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2013
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 27, 2016 · Synced Oct 23, 2026, 7:12 AM EDT