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ClinicalTrials.gov public records Last synced Oct 7, 2026, 4:45 AM EDT

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Showing 73–96 of 911 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Neuromuscular; Disorder, Hereditary Clear all
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 7, 2026, 4:45 AM EDT
Recruiting Phase 3 Interventional

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

NCT06563895
View directory record Official record
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases, Polyneuropathies
Interventions
Acoramidis, Placebo oral tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 75 Years
Enrollment
587 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
40
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 31 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 7, 2026, 4:45 AM EDT
Terminated Phase 1 Interventional

Study of ASN003 in Subjects With Advanced Solid Tumors

NCT02961283
View directory record Official record
Conditions
Neoplasms, Melanoma, Colorectal Neoplasm, Carcinoma, Non-small Cell Lung
Interventions
ASN003 ascending doses, ASN003 MTD
Drug
Lead sponsor
Asana BioSciences
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
5
States / cities
Los Angeles, California • Tampa, Florida • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 9, 2023 · Synced Oct 7, 2026, 4:45 AM EDT
Active, not recruiting Phase 4 Interventional

Pediatric Long-Term Follow-up and Rollover Study

NCT03975829
View directory record Official record
Conditions
Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood, Anaplastic Oligodendroglioma, Glioblastoma, Pilocytic Astrocytoma, Giant Cell Astrocytoma, Pleomorphic Xanthoastrocytoma, Anaplastic Pleomorphic Xanthoastrocytoma, Angiocentric Glioma, Chordoid Glioma of Third Ventricle, Gangliocytoma, Ganglioglioma, Anaplastic Ganglioglioma, Dysplastic Gangliocytoma of Cerebrellum, Desmoplastic Infantile Astrocytoma and Ganglioglioma, Papillary Glioneuronal Tumor, Rosette-forming Glioneuronal Tumor, Central Neurocytoma, Extraventricular Neurocytoma, Cerebellar Liponeurocytoma, Neurofibromatosis Type 1
Interventions
dabrafenib, trametinib
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Year to 99 Years
Enrollment
163 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2026
U.S. locations
11
States / cities
Phoenix, Arizona • Washington D.C., District of Columbia • Miami, Florida + 8 more
Source: ClinicalTrials.gov public record
Updated Aug 27, 2026 · Synced Oct 7, 2026, 4:45 AM EDT
Completed No phase listed Observational

Characteristics of Andersen-Tawil Syndrome

NCT00521794
View directory record Official record
Conditions
Andersen-Tawil Syndrome, Andersen Syndrome
Interventions
Not listed
Lead sponsor
University of Rochester
Other
Eligibility
10 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
4
States / cities
San Francisco, California • Kansas City, Kansas • Rochester, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 16, 2013 · Synced Oct 7, 2026, 4:45 AM EDT
Completed Phase 2 Interventional Results available

SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors

NCT03433183
View directory record Official record
Conditions
Malignant Peripheral Nerve Sheath Tumors, Neurofibromatosis 1
Interventions
Selumetinib, Sirolimus
Drug
Lead sponsor
Sarcoma Alliance for Research through Collaboration
Other
Eligibility
12 Years and older
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
5
States / cities
Washington D.C., District of Columbia • Baltimore, Maryland • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated May 27, 2026 · Synced Oct 7, 2026, 4:45 AM EDT
Completed Phase 2 Interventional Results available

Combination Chemotherapy in Treating Patients With Stage III or Stage IV Malignant Peripheral Nerve Sheath Tumors

NCT00304083
View directory record Official record
Conditions
Neurofibromatosis Type 1, Sarcoma
Interventions
filgrastim, doxorubicin hydrochloride, etoposide, ifosfamide, conventional surgery, radiation therapy
Biological · Drug · Procedure + 1 more
Lead sponsor
Sarcoma Alliance for Research through Collaboration
Other
Eligibility
Not listed
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2014
U.S. locations
17
States / cities
Birmingham, Alabama • Santa Monica, California • Chicago, Illinois + 13 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2018 · Synced Oct 7, 2026, 4:45 AM EDT
Terminated Phase 3 Interventional Results available

Long-Term Analgesic Efficacy And Safety Of Tanezumab Alone Or In Combination With Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) Versus NSAIDs Alone In Patients With Osteoarthritis Of The Knee Or Hip

NCT00809354
View directory record Official record
Conditions
Osteoarthritis, Arthritis
Interventions
NSAID, tanezumab
Drug · Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 99 Years
Enrollment
2,720 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
250
States / cities
Birmingham, Alabama • Huntsville, Alabama • Mobile, Alabama + 161 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2021 · Synced Oct 7, 2026, 4:45 AM EDT
Enrolling by invitation Not applicable Interventional

AI Echocardiographic Screening of Cardiac Amyloidosis

NCT06664866
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
EchoNet-LVH Assessment
Diagnostic Test
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
22 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
4
States / cities
Los Angeles, California • Palo Alto, California • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 7, 2026, 4:45 AM EDT
Withdrawn No phase listed Observational Accepts healthy volunteers

COVID-19 In-vitro Diagnostic Test and Androgen Receptor Gene Expression

NCT04530500
View directory record Official record
Conditions
Covid19, Androgen Receptor Gene Overexpression
Interventions
COVID-19 Androgen Sensitivity Test (CoVAST)
Genetic
Lead sponsor
University of California, Irvine
Other
Eligibility
18 Years to 90 Years · Male only
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2023
U.S. locations
2
States / cities
Irvine, California
Source: ClinicalTrials.gov public record
Updated May 8, 2024 · Synced Oct 7, 2026, 4:45 AM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Electrical Impedance Myography: Natural History Studies inNeuromuscular Disorders and Healthy Volunteers

NCT01900132
View directory record Official record
Conditions
Neuromuscular Disease, Motor Neuron Disease, Inherited Neuromuscular Conditions, Inherited Neuropathies
Interventions
EIM testing, Nerve & muscle ultrasonography, Nerve conduction studies, Electromyography (EMG)
Device
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
2 Years to 110 Years
Enrollment
275 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 7, 2026, 4:45 AM EDT
Active, not recruiting No phase listed Observational

Genetic and Physical Study of Childhood Nerve and Muscle Disorders

NCT01568658
View directory record Official record
Conditions
Muscular Dystrophies, Muscle Myopathies, Hereditary Spastic Paraplegias, Inherited Neuropathies, Inherited Neuromuscular Conditions
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
1 Day to 100 Years
Enrollment
2,586 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 7, 2026, 4:45 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Study of Electrical Impedance Myography (EIM) in ALS

NCT02011204
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Motor Neuron Disease, Charcot-Marie-Tooth Disease, Multiple Sclerosis
Interventions
Electrical impedance myography (EIM)
Device
Lead sponsor
Skulpt, Inc.
Industry
Eligibility
35 Years to 80 Years
Enrollment
106 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2016
U.S. locations
6
States / cities
Phoenix, Arizona • Miami, Florida • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 11, 2016 · Synced Oct 7, 2026, 4:45 AM EDT
Completed Phase 3 Interventional Results available

Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DMD)

NCT01027884
View directory record Official record
Conditions
Muscular Dystrophy, Duchenne, Ambulatory Care
Interventions
Placebo, Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
10 Years to 18 Years · Male only
Enrollment
65 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
9
States / cities
Sacramento, California • Aurora, Colorado • Gainesville, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 19, 2015 · Synced Oct 7, 2026, 4:45 AM EDT
Recruiting No phase listed Observational

Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)

NCT06222203
View directory record Official record
Conditions
Neurofibromatosis 1, Nerve Sheath Neoplasms
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
3 Years to 120 Years
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 2, 2026 · Synced Oct 7, 2026, 4:45 AM EDT
Completed Not applicable Interventional Results available

Cardiac Amyloidosis Discovery Trial

NCT06469372
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
Cardiac amyloidosis deep learning model
Device
Lead sponsor
Pierre Elias
Other
Eligibility
50 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Dec 4, 2025 · Synced Oct 7, 2026, 4:45 AM EDT
Completed No phase listed Observational

Peak Cough Flow and Cough Clearance in Patients With Muscular Dystrophy

NCT02034305
View directory record Official record
Conditions
Duchenne Muscular Dystrophy
Interventions
Not listed
Lead sponsor
University of Pittsburgh
Other
Eligibility
6 Years to 21 Years · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 5, 2024 · Synced Oct 7, 2026, 4:45 AM EDT
Enrolling by invitation Phase 1 Interventional

Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)

NCT06300502
View directory record Official record
Conditions
Neurofibromas, Cutaneous, Neurofibromatosis 1
Interventions
Kybella
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 85 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced Oct 7, 2026, 4:45 AM EDT
Completed Phase 2 Interventional Results available

(-)- Epicatechin Becker Muscular Dystrophy

NCT03236662
View directory record Official record
Conditions
Becker Muscular Dystrophy
Interventions
(-)-Epicatechin
Drug
Lead sponsor
Craig McDonald, MD
Other
Eligibility
18 Years to 70 Years · Male only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
1
States / cities
Sacramento, California
Source: ClinicalTrials.gov public record
Updated Nov 24, 2021 · Synced Oct 7, 2026, 4:45 AM EDT
Recruiting Phase 1 Interventional

9-ING-41 Combined With Retifanlimab, Plus Modified FOLFIRINOX for Patients With Advanced Pancreatic Adenocarcinoma (RiLEY)

NCT06896188
View directory record Official record
Conditions
Pancreatic Adenocarcinoma
Interventions
Retifanlimab, Chemotherapy, 9-ING-41
Drug
Lead sponsor
Anwaar Saeed
Other
Eligibility
18 Years and older
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 24, 2026 · Synced Oct 7, 2026, 4:45 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Magnetic Stimulation of the Human Nervous System

NCT00001780
View directory record Official record
Conditions
Demyelinating Disease, Healthy, Lysosomal Storage Disease, Motor Neuron Disease, Movement Disorder
Interventions
Not listed
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
450 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
1998 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 7, 2026, 4:45 AM EDT
Completed Phase 2Phase 3 Interventional Results available

A Study of the Experimental Drug BKM120 With Paclitaxel in Patients With HER2 Negative, Locally Advanced or Metastatic Breast Cancer, With or Without PI3K Activation

NCT01572727
View directory record Official record
Conditions
Breast Cancer
Interventions
Paclitaxel, BKM120 matching placebo, BKM120
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older · Female only
Enrollment
416 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2015
U.S. locations
21
States / cities
Chandler, Arizona • Phoenix, Arizona • Fayetteville, Arkansas + 17 more
Source: ClinicalTrials.gov public record
Updated Mar 9, 2017 · Synced Oct 7, 2026, 4:45 AM EDT
Recruiting Phase 1 Interventional

Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1

NCT07102394
View directory record Official record
Conditions
NF1, Neurofibromatosis, Cutaneous Neurofibroma, Neurofibroma, Von Recklinghausen Disease, Neurofibromas
Interventions
Imlygic
Drug
Lead sponsor
Johns Hopkins University
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Baltimore, Maryland
Source: ClinicalTrials.gov public record
Updated May 8, 2026 · Synced Oct 7, 2026, 4:45 AM EDT
Terminated Phase 3 Interventional

Efficacy Evaluation of Enobosarm Monotherapy in Treatment of AR+/ER+/HER2- Metastatic Breast Cancer

NCT04869943
View directory record Official record
Conditions
Metastatic Breast Cancer
Interventions
Enobosarm, Exemestane
Drug
Lead sponsor
Veru Inc.
Industry
Eligibility
18 Years to 100 Years
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
34
States / cities
Chandler, Arizona • Gilbert, Arizona • Glendale, California + 29 more
Source: ClinicalTrials.gov public record
Updated Jan 26, 2024 · Synced Oct 7, 2026, 4:45 AM EDT