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ClinicalTrials.gov public records Last synced Jul 22, 2026, 8:18 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Recruiting Phase 1 Interventional

A Study to Evaluate the Safety and Tolerability of TOS-358 in Women With HR+ HER2- Breast Cancer

NCT05683418
View directory record Official record
Conditions
HR+/HER2-negative Breast Cancer
Interventions
TOS-358, Fulvestrant, Palbociclib, Ribociclib
Drug
Lead sponsor
Totus Medicines
Industry
Eligibility
18 Years and older · Female only
Enrollment
241 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
9
States / cities
Chicago, Illinois • Boston, Massachusetts • Las Vegas, Nevada + 4 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Jul 22, 2026, 8:18 PM 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 21, 2019 · Synced Jul 22, 2026, 8:18 PM EDT
Terminated Phase 2 Interventional

Effect of Oral Propranolol on mRNA Expresssion in Symptomatic Cavernous Malformation

NCT03474614
View directory record Official record
Conditions
Cerebral Cavernous Malformations
Interventions
Propranolol, DNA and RNA Analysis
Drug · Genetic
Lead sponsor
St. Joseph's Hospital and Medical Center, Phoenix
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Jul 22, 2026, 8:18 PM EDT
Recruiting Not applicable Interventional

Establishing the Preliminary Utility of a Novel Pediatric Manual Mobile Standing Wheelchair

NCT07290556
View directory record Official record
Conditions
Spina Bifida
Interventions
PedMMSWC Wheelchair User Testing, PedMMSWC Simulated Home, School, Community Environment
Device
Lead sponsor
Optimal Mobility, Inc
Industry
Eligibility
8 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Saint Paul, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 23, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Genetics of Neural Tube Defects

NCT01253746
View directory record Official record
Conditions
Neural Tube Defects
Interventions
Not listed
Lead sponsor
John P. Hussman Institute for Human Genomics
Other
Eligibility
Not listed
Enrollment
6,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2009
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Dec 2, 2010 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting Phase 2 Interventional

Safety and Modulation of ABCC9 Pathways by Nicorandil for the Treatment of Hippocampal Sclerosis of Aging

NCT04120766
View directory record Official record
Conditions
Dementia, Mild Cognitive Impairment
Interventions
Nicorandil, Placebo
Drug
Lead sponsor
Gregory Jicha, MD, PhD
Other
Eligibility
70 Years and older
Enrollment
64 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Lexington, Kentucky
Source: ClinicalTrials.gov public record
Updated Dec 14, 2025 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 1 Interventional

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

NCT04095273
View directory record Official record
Conditions
Advanced Solid Tumors
Interventions
Elimusertib (BAY1895344), Pembrolizumab (Keytruda®)
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
8
States / cities
Palo Alto, California • New Haven, Connecticut • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2024 · Synced Jul 22, 2026, 8:18 PM EDT
Withdrawn Phase 2 Interventional

The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia

NCT02608931
View directory record Official record
Conditions
Nausea, Vomiting, Familial Dysautonomia
Interventions
Dronabinol, Placebo
Drug · Other
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years to 60 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 12, 2019 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Innovative Approaches to Gauge Progression of Sturge-Weber Syndrome

NCT01425944
View directory record Official record
Conditions
Sturge-Weber Syndrome
Interventions
Not listed
Lead sponsor
Hugo W. Moser Research Institute at Kennedy Krieger, Inc.
Other
Eligibility
1 Month and older
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2027
U.S. locations
7
States / cities
Baltimore, Maryland • Detroit, Michigan • New York, New York + 4 more
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting Not applicable Interventional

Fetoscopic NEOX Cord 1K® Spina Bifida Repair

NCT04243889
View directory record Official record
Conditions
Spina Bifida, Myelomeningocele, Myeloschisis
Interventions
NEOX Cord 1K
Device
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2029
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Sep 2, 2025 · Synced Jul 22, 2026, 8:18 PM EDT
Not listed Phase 3 Interventional

Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients

NCT04762758
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
(RS)-baclofen, naltrexone hydrochloride and D-sorbitol, Placebo
Drug
Lead sponsor
Pharnext S.C.A.
Other
Eligibility
16 Years to 65 Years
Enrollment
350 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
20
States / cities
Los Angeles, California • Sacramento, California • New Britain, Connecticut + 16 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Jul 22, 2026, 8:18 PM EDT
Terminated Phase 2 Interventional Results available

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease

NCT03124459
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
ACE-083, Placebo
Drug
Lead sponsor
Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA
Industry
Eligibility
18 Years and older
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
16
States / cities
Orange, California • Aurora, Colorado • Gainesville, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2022 · Synced Jul 22, 2026, 8:18 PM 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 – 2026
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Nov 2, 2025 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting Phase 3 Interventional

Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment

NCT02962414
View directory record Official record
Conditions
Tuberous Sclerosis Complex
Interventions
everolimus
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years to 65 Years
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
14
States / cities
Phoenix, Arizona • Los Angeles, California • Oakland, California + 11 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Terminated Phase 1Phase 2 Interventional

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

NCT05394064
View directory record Official record
Conditions
AMN, AMN Gene Mutation, X-ALD
Interventions
SBT101, Imitation Procedure
Genetic · Procedure
Lead sponsor
SwanBio Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 30, 2025 · Synced Jul 22, 2026, 8:18 PM EDT
Completed No phase listed Observational

An Analysis of the Symptomatic Domains Most Relevant to Charcot Marie Tooth Neuropathy (CMT) Patients

NCT02429947
View directory record Official record
Conditions
Charcot Marie Tooth Disease (CMT), Hereditary Sensory and Motor Neuropathy, Nerve Compression Syndromes, Tooth Diseases, Congenital Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older
Enrollment
411 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Tampa, Florida
Source: ClinicalTrials.gov public record
Updated Jun 13, 2017 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study to Determine the Relative Bioavailability of Capsule and Tablet Formulations of GDC-0941 and the Effect of Food and Proton Pump Inhibition on GDC-0941 Tablet Pharmacokinetics in Healthy Volunteers

NCT00999128
View directory record Official record
Conditions
Healthy Volunteers
Interventions
GDC-0941, rabeprazole
Drug
Lead sponsor
Genentech, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
50 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2010
U.S. locations
1
States / cities
South San Francisco, California
Source: ClinicalTrials.gov public record
Updated Feb 8, 2017 · Synced Jul 22, 2026, 8:18 PM EDT
Completed No phase listed Observational

The Impact of Community-based Wellness Programs on The Triple Aim

NCT02938130
View directory record Official record
Conditions
Spina Bifida, Cerebral Palsy, Spinal Cord Injury, Traumatic Brain Injury
Interventions
Wellness Programs
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
7 Years and older
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 11, 2025 · Synced Jul 22, 2026, 8:18 PM EDT
Not listed Phase 2 Interventional

Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)

NCT05103358
View directory record Official record
Conditions
Tumor, Tumor, Solid, Metastasis, Metastatic Cancer, Cancer, Cancer Metastatic, Tumors, Neoplasms, Neoplasm Metastasis, Solid Tumor, Advanced Solid Tumor, Advanced Cancer, Malignant Solid Tumor, Malignant Solid Neoplasm, Malignant Neoplasm, Malignant Tumor, TSC, TSC1, TSC2, Metastatic Solid Tumor, Metastatic Neoplasm
Interventions
nab-sirolimus
Drug
Lead sponsor
Aadi Bioscience, Inc.
Industry
Eligibility
12 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
159
States / cities
Birmingham, Alabama • Mobile, Alabama • Goodyear, Arizona + 137 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2024 · Synced Jul 22, 2026, 8:18 PM EDT
Recruiting No phase listed Observational

Natural History Study for Charcot Marie Tooth Disease

NCT05902351
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease Type 2, Charcot-Marie-Tooth Disease, Type 2C, Charcot-Marie-Tooth Disease Type 2A2B, Charcot-Marie-Tooth Disease Type 2B2, Charcot-Marie-Tooth Disease Type 2A1, Charcot-Marie-Tooth Disease Type 4B1, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease Type 2B1, Charcot-Marie-Tooth Disease Type 2U (Diagnosis), Charcot-Marie-Tooth Disease Type 4A, Charcot-Marie-Tooth Disease, Type 4A, Axonal Form, Charcot-Marie-Tooth Disease Type 2A2A, Charcot-Marie-Tooth Disease Type 2S (Disorder), Charcot-Marie-Tooth Disease and Deafness, Charcot-Marie-Tooth Disease Type 4B2, Charcot-Marie-Tooth Disease Type 4H, Charcot-Marie-Tooth Disease Type 1F, Charcot-Marie-Tooth Disease Type 4C, Charcot-Marie-Tooth Disease Type 4E, Charcot-Marie-Tooth Disease Type 1D, Charcot-Marie-Tooth Disease Type 2Q (Diagnosis), Charcot-Marie-Tooth Disease Type 2A2, Charcot-Marie-Tooth Disease Type 2N (Diagnosis), Charcot-Marie-Tooth Disease Type 2B5, Charcot-Marie-Tooth Disease Type 2D, Charcot-Marie-Tooth Disease Type 4D, Charcot-Marie-Tooth Disease Type 2K, Charcot-Marie-Tooth Disease Type 2L (Diagnosis), Charcot-Marie-Tooth Disease Type 2T, Charcot-Marie-Tooth Disease Type 2I, Charcot-Marie-Tooth Disease Type 2J, Charcot-Marie-Tooth Disease Type 2E, Charcot-Marie-Tooth Disease Type 2G, Charcot-Marie-Tooth Disease Type 1C, Charcot-Marie-Tooth Disease Type 2R, Charcot-Marie-Tooth Disease Type 2O (Diagnosis), Charcot-Marie-Tooth Disease Type 2M, Charcot-Marie-Tooth Disease Type 2P, Charcot-Marie-Tooth Disease Type 2Y, Charcot-Marie-Tooth Disease Type 4F (Diagnosis), Charcot-Marie-Tooth Disease Type 4B3, Charcot-Marie-Tooth Disease Type 2H, HNPP, X-Linked Charcot-Marie-Tooth Disease
Interventions
Not listed
Lead sponsor
Hereditary Neuropathy Foundation
Government
Eligibility
Not listed
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2029
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 30, 2024 · Synced Jul 22, 2026, 8:18 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

NCT01931644
View directory record Official record
Conditions
All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions
Not listed
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 100 Years
Enrollment
17,667 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 17, 2024 · Synced Jul 22, 2026, 8:18 PM 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 28, 2023 · Synced Jul 22, 2026, 8:18 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Observational Study of Neuromuscular Function in CMT Type 1&2 and Healthy Controls

NCT04980807
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
Not listed
Lead sponsor
NMD Pharma A/S
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 6, 2022 · Synced Jul 22, 2026, 8:18 PM EDT
Recruiting No phase listed Observational

Study of Selected X-Linked Disorders: Aicardi Syndrome

NCT00697411
View directory record Official record
Conditions
Aicardi Syndrome, Brain Disorders
Interventions
Syndrome cause identification
Other
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Not listed
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2030
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 4, 2026 · Synced Jul 22, 2026, 8:18 PM EDT