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ClinicalTrials.gov public records Last synced Oct 17, 2026, 3:42 AM EDT

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Showing 361–384 of 1,147 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neurodegenerative Disease, Hereditary Clear all
Terminated Phase 2 Interventional Results available

AZD2171 in Treating Patients With Neurofibromatosis Type 1 and Plexiform Neurofibroma and/or Neurofibroma Near the Spine

NCT00326872
View directory record Official record
Conditions
Neurofibromatosis Type 1, Plexiform Neurofibroma, Spinal Cord Neurofibroma
Interventions
Cediranib Maleate
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2016
U.S. locations
9
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 5 more
Source: ClinicalTrials.gov public record
Updated Aug 18, 2017 · Synced Oct 17, 2026, 3:42 AM EDT
Recruiting Phase 1 Interventional

A Study to Investigate the Safety, Pharmacokinetics, and Preliminary Efficacy of IDE574 Therapy in Adult Participants With Advanced Solid Tumors

NCT07540572
View directory record Official record
Conditions
ER+, HER 2- Breast Cancer, Non-small Cell Lung Cancer (NSCLC), Castration-resistant Prostate Cancer (CRPC), Microsatellite Stable (MSS) Colorectal Carcinoma
Interventions
IDE574, Fulvestrant injection
Drug
Lead sponsor
IDEAYA Biosciences
Industry
Eligibility
18 Years to 99 Years
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
11
States / cities
Plantation, Florida • East Brunswick, New Jersey • Lake Success, New York + 7 more
Source: ClinicalTrials.gov public record
Updated Jun 16, 2026 · Synced Oct 17, 2026, 3:42 AM EDT
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 17, 2026, 3:42 AM EDT
Recruiting Not applicable Interventional

Exercise Training in Transthyretin Cardiac Amyloidosis

NCT05797857
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin Cardiac Amyloidosis
Interventions
Exercise Training
Other
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
60 Years to 90 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2025 · Synced Oct 17, 2026, 3:42 AM EDT
Enrolling by invitation Phase 2 Interventional

Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

NCT06148311
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
Matching placebo, Dexmedetomidine
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
16 Years to 80 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Oct 17, 2026, 3:42 AM EDT
Recruiting Not applicable Interventional

Strengthening Tourette Treatment OPtions Using TMS to Improve CBIT, a Double-blind, Randomized, Controlled Study

NCT05705999
View directory record Official record
Conditions
Tourette Syndrome
Interventions
transcranial magnetic stimulation
Device
Lead sponsor
West Virginia University
Other
Eligibility
18 Years to 100 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Morgantown, West Virginia
Source: ClinicalTrials.gov public record
Updated Jul 18, 2025 · Synced Oct 17, 2026, 3:42 AM EDT
Completed Phase 1 Interventional

Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas

NCT06132165
View directory record Official record
Conditions
Neurofibromatosis 1
Interventions
Deoxycholic Acid, Polidocanol, 1064nm Nd:YAG laser, 755nm Alexandrite Laser
Drug · Device
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 80 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Nov 21, 2025 · Synced Oct 17, 2026, 3:42 AM EDT
Completed Phase 1Phase 2 Interventional

Study of PX-866 and Docetaxel in Solid Tumors

NCT01204099
View directory record Official record
Conditions
Non Small Cell Lung Cancer (NSCLC), Squamous Cell Carcinoma of the Head and Neck (SCCHN)
Interventions
Docetaxel, PX-866
Drug
Lead sponsor
Cascadian Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
223 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
29
States / cities
Birmingham, Alabama • Escondido, California • Pleasant Hill, California + 23 more
Source: ClinicalTrials.gov public record
Updated May 16, 2018 · Synced Oct 17, 2026, 3:42 AM EDT
Completed No phase listed Observational

Early Biomarkers of Autism in Infants With Tuberous Sclerosis Complex (TSC)

NCT01780441
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
3 Months to 12 Months
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
5
States / cities
Birmingham, Alabama • Los Angeles, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 4, 2021 · Synced Oct 17, 2026, 3:42 AM EDT
Withdrawn Not applicable Interventional

Multi-site Transcranial Magnetic Stimulation Therapy of the Supplementary Motor Area in Children With Tourette Syndrome

NCT03642951
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Active Treatment, Sham Treatment
Device
Lead sponsor
Children's Mercy Hospital Kansas City
Other
Eligibility
8 Years to 20 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Kansas City, Missouri
Source: ClinicalTrials.gov public record
Updated Feb 15, 2022 · Synced Oct 17, 2026, 3:42 AM EDT
No Longer Available No phase listed Expanded access

Compassionate Use Arm - ABI541 ABI for 10 NF2 Patients

NCT02589912
View directory record Official record
Conditions
Neurofibromatosis Type 2, Severe Profound Sensorineural Hearing Loss
Interventions
Nucleus Profile ABI541 Auditory Brainstem Implant
Device
Lead sponsor
Massachusetts Eye and Ear Infirmary
Other
Eligibility
12 Years to 80 Years
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 6, 2019 · Synced Oct 17, 2026, 3:42 AM EDT
Completed Phase 1 Interventional

Pirfenidone in Treating Young Patients With Neurofibromatosis Type 1 and Plexiform Neurofibromas

NCT00053937
View directory record Official record
Conditions
Neurofibromatosis Type 1, Precancerous Condition
Interventions
pirfenidone
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 21 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2002
U.S. locations
15
States / cities
Birmingham, Alabama • Washington D.C., District of Columbia • Chicago, Illinois + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 30, 2015 · Synced Oct 17, 2026, 3:42 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

University of Central Florida CereBank

NCT06876857
View directory record Official record
Conditions
Biospecimen Donation, Brain Injury, Brain Diseases
Interventions
Not listed
Lead sponsor
University of Central Florida
Other
Eligibility
18 Years and older · Female only
Enrollment
50,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2075
U.S. locations
1
States / cities
Orlando, Florida
Source: ClinicalTrials.gov public record
Updated Mar 14, 2025 · Synced Oct 17, 2026, 3:42 AM EDT
Completed Phase 1 Interventional

Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia

NCT04519567
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
CTI-1601, Placebo
Biological
Lead sponsor
Larimar Therapeutics, Inc.
Industry
Eligibility
18 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Eatontown, New Jersey
Source: ClinicalTrials.gov public record
Updated Jun 30, 2021 · Synced Oct 17, 2026, 3:42 AM EDT
Recruiting No phase listed Observational

Molecular and Genetic Studies of Congenital Myopathies

NCT00272883
View directory record Official record
Conditions
Central Core Disease, Centronuclear Myopathy, Congenital Fiber Type Disproportion, Multiminicore Disease, Myotubular Myopathy, Nemaline Myopathy, Rigid Spine Muscular Dystrophy, Undefined Congenital Myopathy
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Not listed
Enrollment
4,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2050
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 25, 2026 · Synced Oct 17, 2026, 3:42 AM EDT
Active, not recruiting Phase 2 Interventional

MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas

NCT02407405
View directory record Official record
Conditions
Neurofibromatosis 1 (NF1), Plexiform Neurofibromas (PN)
Interventions
Selumetinib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 15, 2026 · Synced Oct 17, 2026, 3:42 AM EDT
Completed Not applicable Interventional

Anger Control Training for Youth With Tourette Syndrome

NCT00486551
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorder, Oppositional Defiant Disorder, ADHD
Interventions
Anger control training
Behavioral
Lead sponsor
Yale University
Other
Eligibility
11 Years to 16 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2006
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Feb 11, 2022 · Synced Oct 17, 2026, 3:42 AM EDT
Completed No phase listed Observational

Frameshift Peptides of Children With NF1

NCT04212351
View directory record Official record
Conditions
Neurofibromatosis Type 1
Interventions
Frameshift Array blood sample test
Genetic
Lead sponsor
Children's National Research Institute
Other
Eligibility
1 Day to 30 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
3
States / cities
Washington D.C., District of Columbia • Atlanta, Georgia • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Aug 31, 2022 · Synced Oct 17, 2026, 3:42 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I

NCT00439218
View directory record Official record
Conditions
Spinal Muscular Atrophy Type I
Interventions
sodium phenylbutyrate
Drug
Lead sponsor
Westat
Other
Eligibility
2 Months to 48 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
5
States / cities
Stanford, California • Boston, Massachusetts • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 9, 2010 · Synced Oct 17, 2026, 3:42 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2

NCT01193088
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Type Ia (Disorder), HMSN
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
Not listed
Enrollment
1,050 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2030
U.S. locations
18
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 14 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 3:42 AM EDT
Recruiting No phase listed Observational

Natural History Study for DNA Repair Disorders

NCT05484570
View directory record Official record
Conditions
DNA Repair Disorder, Cockayne Syndrome, Xeroderma Pigmentosum, Trichothiodystrophy
Interventions
Interval History, Physical Examination, ECAB Assessment, Gait Assessment, Specimen Sample Collection
Other
Lead sponsor
University of Minnesota
Other
Eligibility
6 Months and older
Enrollment
40 participants
Timeline
2022 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 17, 2026, 3:42 AM EDT
Active, not recruiting Phase 2 Interventional

Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

NCT06128356
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
Dexmedetomidine Sublingual
Drug
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years to 80 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 4, 2026 · Synced Oct 17, 2026, 3:42 AM EDT
Active, not recruiting Phase 3 Interventional Accepts healthy volunteers

Molecular Imaging of Myocardial Fibrosis in Cardiac Amyloidosis

NCT05619302
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
[68Ga]CBP8 PET/MRI
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2023 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Oct 17, 2026, 3:42 AM EDT
Completed Phase 2 Interventional Results available

Safety, Tolerability, Pharmacokinetic, and Efficacy Study of AZD5213 in Adolescents With Tourette's Disorder

NCT01904773
View directory record Official record
Conditions
Tourette Syndrome
Interventions
AZD5213 and placebo
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
12 Years to 17 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2015
U.S. locations
7
States / cities
Orange, California • St. Petersburg, Florida • Summit, New Jersey + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2016 · Synced Oct 17, 2026, 3:42 AM EDT