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Conditions: Muscle Spasticity, Hypertonia, Muscle, Cerebral Palsy, Multiple Sclerosis, Stroke, Hereditary Spastic Paraplegia, Dystonia, Spinal Cord Injuries
Interventions: MTR-601; Drug
Lead sponsor: Motric Bio; Industry
Eligibility: 18 Years to 45 Years
Enrollment: 89 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2024
U.S. locations: 1
States / cities: San Antonio, Texas

Conditions: Cardiac Amyloidosis, Heart Diseases, TTR Cardiac Amyloidosis
Interventions: Beta blocker; Drug
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 65 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: New York, New York

Conditions: Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome, Primary Lateral Sclerosis, Spinal Muscular Atrophy, Charcot Marie Tooth Disease (CMT), Fascioscapulohumeral Muscular Dystrophy, Inclusion Body Myositis, Mitochondrial Myopathy, Nemaline Myopathy, Centronuclear Myopathy, Postpolio Syndrome, Pompe Disease (Late-onset), Chronic Inflammatory Demyelinating Polyneuropathy, Hereditary Spastic Paraplegia, Postural Orthostatic Tachycardia Syndrome (POTS), Progressive Muscular Atrophy
Interventions: Whole-body Electrical Muscle Stimulation Exercise; Device
Lead sponsor: University of Missouri-Columbia; Other
Eligibility: 18 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2031
U.S. locations: 1
States / cities: Columbia, Missouri

Conditions: Charcot Marie Tooth Disease
Interventions: Not listed
Lead sponsor: Michael Shy; Other
Eligibility: Not listed
Enrollment: 5,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2010 – 2026
U.S. locations: 18
States / cities: Los Angeles, California • Palo Alto, California • Aurora, Colorado + 13 more

Conditions: Neonatal Brachial Plexus Palsy
Interventions: Tetra-grip; Device
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 3 Years to 7 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 1
States / cities: Dallas, Texas

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

Conditions: Leber's Hereditary Optic Neuropathy
Interventions: EPI-743; Drug
Lead sponsor: Edison Pharmaceuticals Inc; Industry
Eligibility: 8 Years to 65 Years
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Autoimmune Hemolytic Anemia
Interventions: Parsaclisib; Drug
Lead sponsor: Incyte Corporation; Industry
Eligibility: 18 Years and older
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2024
U.S. locations: 6
States / cities: Washington D.C., District of Columbia • Minneapolis, Minnesota • St Louis, Missouri + 3 more

Conditions: Peripheral Neuropathy, Chronic Inflammatory Demyelinating Polyradiculoneuropathy, Vasculitic Neuropathy (Disorder), Amyloidosis, Hereditary Neuropathy, Sarcoid Neuropathy
Interventions: Biopsy + Nerve Repair, Biopsy Only; Device · Procedure
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years to 75 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2027
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Amyloid Cardiomyopathy, Transthyretin-Related
Interventions: Acoramidis (AG10); Drug
Lead sponsor: Eidos Therapeutics, a BridgeBio company; Industry
Eligibility: 18 Years to 90 Years
Enrollment: 389 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 26
States / cities: Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 21 more

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

Conditions: AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month to 100 Years
Enrollment: 999 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2099
U.S. locations: 1
States / cities: Bethesda, Maryland

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

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

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

Conditions: Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions: Not listed
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 30 Years to 80 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions: AI Toolkit for ATTR-CM Diagnosis; Diagnostic Test
Lead sponsor: Yale University; Other
Eligibility: 50 Years to 95 Years
Enrollment: 1,500,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 11
States / cities: San Francisco, California • New Haven, Connecticut • Detroit, Michigan + 8 more

Conditions: Charcot Marie Tooth Disease
Interventions: Placebo, Mexiletine; Other · Drug
Lead sponsor: University of Rochester; Other
Eligibility: 18 Years and older
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 1
States / cities: Rochester, New York

Conditions: Giant Axonal Neuropathy (GAN)
Interventions: scAAV9/JeT-GAN; Genetic
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 10 Years to 25 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2032
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
Interventions: Not listed
Lead sponsor: Alnylam Pharmaceuticals; Industry
Eligibility: Not listed
Enrollment: 1,500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2033
U.S. locations: 13
States / cities: La Jolla, California • Los Angeles, California • Jacksonville, Florida + 10 more

Conditions: Friedreich Ataxia
Interventions: Idebenone; Drug
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Not listed
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2006
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
Interventions: Patisiran; Drug
Lead sponsor: Alnylam Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 67 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 27
States / cities: Los Angeles, California • San Diego, California • Aurora, Colorado + 22 more

Conditions: Advanced Solid Malignancy, Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, Tumour Response, Advanced or Metastatic Breast Cancer, Ovarian Cancer, Cervical Cancer, Endometrial Cancer, PIK3CA, AKT1, PTEN, ER Positive, HER2 Positive
Interventions: AZD5363; Drug
Lead sponsor: AstraZeneca; Industry
Eligibility: 18 Years to 130 Years
Enrollment: 285 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2024
U.S. locations: 12
States / cities: Los Angeles, California • Stanford, California • West Hollywood, California + 8 more

Conditions: Familial Amyloid Polyneuropathy, Familial Amyloidosis
Interventions: diflunisal, placebo; Drug · Other
Lead sponsor: Boston University; Other
Eligibility: 18 Years to 75 Years
Enrollment: 130 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2012
U.S. locations: 3
States / cities: Boston, Massachusetts • Rochester, Minnesota • New York, New York

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Single and Multiple Ascending Dose Study of MTR-601 in Healthy Individuals

N-of-1 for Beta-Blockers in Cardiac Amyloidosis

Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease

Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

Does Wearing Tetra-Grip Improve Arm Function in Children Diagnosed With Neonatal Brachial Plexus Palsy?

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

Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]

A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

Clinical Trial to Assess the Safety of a Novel Scaffold Biomaterial

Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

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

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

L-Serine Supplementation in Hereditary Sensory Neuropathy Type 1

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

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

Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study

An Observational Study Using Artificial Intelligence (AI) Algorithms on Electrocardiography (ECG), Point-of-care Ultrasound (POCUS), and Transthoracic Echocardiophy (TTE) to Estimate the Under-diagnosis of Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Across a Diverse Range of US Health Systems.

Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease

Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

Safety Study of Idebenone to Treat Friedreich's Ataxia

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Safety, Tolerability & Potential Anti-cancer Activity of Increasing Doses of AZD5363 in Different Treatment Schedules

The Effect of Diflunisal on Familial Amyloidosis