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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Completed Phase 2 Interventional Results available

Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)

NCT01289912
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
RAD001, Placebo
Drug
Lead sponsor
Mustafa Sahin
Other
Eligibility
6 Years to 21 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
Boston, Massachusetts • Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Jan 24, 2018 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 2 Interventional

Thalamic Deep Brain Stimulation for Tourette Syndrome

NCT00311909
View directory record Official record
Conditions
Tourette Syndrome
Interventions
Thalamic deep brain stimulation
Device
Lead sponsor
University Hospitals Cleveland Medical Center
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2006
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated May 11, 2022 · Synced Sep 23, 2026, 3:18 PM EDT
Active, not recruiting Phase 2 Interventional

AMX0035 in Adult Patients With Wolfram Syndrome

NCT05676034
View directory record Official record
Conditions
Wolfram Syndrome
Interventions
AMX0035
Drug
Lead sponsor
Amylyx Pharmaceuticals Inc.
Industry
Eligibility
17 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2028
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 1 Interventional

Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia

NCT00078481
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Idebenone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 23, 2026, 3:18 PM EDT
Recruiting Phase 2 Interventional

Mirdametinib in Patients With Advanced NF1-mutant Melanoma

NCT07237100
View directory record Official record
Conditions
Advanced Unresectable Melanoma, Metastatic Melanoma
Interventions
Mirdametinib
Drug
Lead sponsor
Kevin Kim, MD
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Nov 18, 2025 · Synced Sep 23, 2026, 3:18 PM 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 23, 2016 · Synced Sep 23, 2026, 3:18 PM EDT
Not listed Phase 1Phase 2 Interventional

Metformin in Patients With Fragile X

NCT04141163
View directory record Official record
Conditions
Fragile X Syndrome
Interventions
Metformin, Placebo oral tablet
Drug
Lead sponsor
Rowan University
Other
Eligibility
18 Years to 50 Years · Male only
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Stratford, New Jersey
Source: ClinicalTrials.gov public record
Updated Oct 30, 2019 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 3 Interventional Results available

Open-Label Extension Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome

NCT04279314
View directory record Official record
Conditions
Rett Syndrome
Interventions
Trofinetide
Drug
Lead sponsor
ACADIA Pharmaceuticals Inc.
Industry
Eligibility
5 Years to 21 Years · Female only
Enrollment
154 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
21
States / cities
Birmingham, Alabama • Phoenix, Arizona • La Jolla, California + 18 more
Source: ClinicalTrials.gov public record
Updated Apr 10, 2024 · Synced Sep 23, 2026, 3:18 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

NCT02327845
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Progressive Muscular Atrophy, Multisystem Proteinopathy
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
Not listed
Enrollment
708 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2031
U.S. locations
13
States / cities
Palo Alto, California • San Diego, California • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 1Phase 2 Interventional

Effect of Functional Exercise in Patients With Spinal Bulbar Muscular Atrophy

NCT01369901
View directory record Official record
Conditions
Motor Neuron Disease
Interventions
A functional exercise program, A stretching exercise program
Behavioral
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
18 Years to 90 Years · Male only
Enrollment
61 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Dec 16, 2019 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 3 Interventional Results available

Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy

NCT02791230
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
1,733 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
41
States / cities
Birmingham, Alabama • Beverly Hills, California • La Jolla, California + 20 more
Source: ClinicalTrials.gov public record
Updated Feb 2, 2025 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A PET Brain Imaging Study of mGluR5 in Subjects With Neuropsychiatric Conditions

NCT00870974
View directory record Official record
Conditions
Parkinson Disease, Huntington Disease, Autistic Spectrum Disorders, Fragile X Syndrome, Alzheimer Disease, Mild Cognitive Impairment
Interventions
[18F]FPEB
Drug
Lead sponsor
Institute for Neurodegenerative Disorders
Other
Eligibility
18 Years to 85 Years
Enrollment
48 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2016
U.S. locations
1
States / cities
New Haven, Connecticut
Source: ClinicalTrials.gov public record
Updated Jan 5, 2017 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

NCT03860935
View directory record Official record
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases
Interventions
acoramidis, Placebo Oral Tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
632 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
40
States / cities
Los Angeles, California • Santa Monica, California • Aurora, Colorado + 32 more
Source: ClinicalTrials.gov public record
Updated Jun 26, 2024 · Synced Sep 23, 2026, 3:18 PM EDT
Completed No phase listed Observational

Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT

NCT01203085
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
Up to 21 Years
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2025
U.S. locations
9
States / cities
Palo Alto, California • Hartford, Connecticut • Orlando, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2025 · Synced Sep 23, 2026, 3:18 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 8, 2022 · Synced Sep 23, 2026, 3:18 PM EDT
Terminated No phase listed Observational

US Selumetinib Registry

NCT05683678
View directory record Official record
Conditions
Neurofibromatosis Type 1, Plexiform Neurofibromas
Interventions
Not listed
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
2 Years to 18 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
10
States / cities
Phoenix, Arizona • Los Angeles, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Apr 3, 2025 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 2 Interventional

Effectiveness of Behavior Therapy and Psychosocial Therapy for the Treatment of Tourette Syndrome and Chronic Tic Disorder

NCT00231985
View directory record Official record
Conditions
Tourette Syndrome, Tic Disorders
Interventions
Habit reversal therapy, Supportive therapy
Behavioral
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
16 Years and older
Enrollment
122 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2010
U.S. locations
3
States / cities
New Haven, Connecticut • Boston, Massachusetts • San Antonio, Texas
Source: ClinicalTrials.gov public record
Updated Feb 8, 2012 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 2Phase 3 Interventional

A Clinical Study to Evaluate the Efficacy and Safety of MIN-102 (IMP) in Male AMN Patients.

NCT03231878
View directory record Official record
Conditions
Adrenoleukodystrophy
Interventions
MIN-102, Placebos
Drug
Lead sponsor
Minoryx Therapeutics, S.L.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
105 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
3
States / cities
Stanford, California • Baltimore, Maryland • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 17, 2025 · Synced Sep 23, 2026, 3:18 PM EDT
Completed No phase listed Observational

Protein Synthesis in the Brain of Patients With Fragile X Syndrome

NCT00362843
View directory record Official record
Conditions
Fragile X Syndrome, Healthy Volunteers
Interventions
[15-O]water
Drug
Lead sponsor
National Institute of Mental Health (NIMH)
NIH
Eligibility
18 Years to 24 Years · Male only
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2020
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 7, 2024 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 2 Interventional Results available

Combination Study of RMC-4630 and Sotorasib for NSCLC Subjects With KRASG12C Mutation After Failure of Prior Standard Therapies

NCT05054725
View directory record Official record
Conditions
Non-Small Cell Lung Cancer
Interventions
RMC-4630, Sotorasib
Drug
Lead sponsor
Revolution Medicines, Inc.
Industry
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
23
States / cities
Fort Myers, Florida • Plantation, Florida • Saint Augustine, Florida + 20 more
Source: ClinicalTrials.gov public record
Updated Jan 5, 2026 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Not applicable Interventional

Incorporating teleCBIT Into a Hospital-Based Tic Program

NCT04007913
View directory record Official record
Conditions
Tic Disorders, Tourette Syndrome
Interventions
teleCBIT
Behavioral
Lead sponsor
San Jose State University
Other
Eligibility
5 Years to 65 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
2
States / cities
San Jose, California • Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 4, 2019 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 3 Interventional Results available

Tanezumab In Osteoarthritis Of The Knee (2)

NCT00830063
View directory record Official record
Conditions
Arthritis, Osteoarthritis
Interventions
tanezumab 10 mg, tanezumab 5 mg, naproxen, placebo
Biological · Drug · Other
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
832 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
94
States / cities
Anniston, Alabama • Mesa, Arizona • Paradise Valley, Arizona + 72 more
Source: ClinicalTrials.gov public record
Updated May 12, 2021 · Synced Sep 23, 2026, 3:18 PM 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 2, 2025 · Synced Sep 23, 2026, 3:18 PM EDT
Completed Phase 2 Interventional

VoIP Delivered Behavior Therapy for Tourette Syndrome

NCT02247206
View directory record Official record
Conditions
Tourette Syndrome, Chronic Tic Disorder
Interventions
Behavior Therapy for Tics (CBIT)
Behavioral
Lead sponsor
University of Wisconsin, Milwaukee
Other
Eligibility
8 Years to 17 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 22, 2014 · Synced Sep 23, 2026, 3:18 PM EDT