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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: Painful Peripheral Neuropathy, Diabetic Peripheral Neuropathic Pain (DPN), Chemotherapy Induced Peripheral Neuropathy (CIPN), Idiopathic Peripheral Neuropathy
Interventions: Pregabalin, Duloxetine, Placebo; Drug · Other
Lead sponsor: University of Rochester; Other
Eligibility: 18 Years to 80 Years
Enrollment: 190 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 7
States / cities: Boston, Massachusetts • New York, New York • Rochester, New York + 4 more

Conditions: 16P11.2 Deletion Syndrome, 16p11.2 Duplications, 1Q21.1 Deletion, 1Q21.1 Microduplication Syndrome (Disorder), ACTL6B, ADNP, AHDC1, ANK2, ANKRD11, ARID1B, ASH1L, BCL11A, CHAMP1, CHD2, CHD8, CSNK2A1, CTBP1, CTNNB1 Gene Mutation, CUL3, DDX3X, DNMT3A, DSCAM, DYRK1A, FOXP1, GRIN2A, GRIN2B, HIVEP2-Related Intellectual Disability, HNRNPH2, KATNAL2, KDM5B, KDM6B, KMT2C Gene Mutation, KMT2E, KMT5B, MBD5, MED13L, PACS1, PPP2R5D-Related Intellectual Disability, PTCHD1, REST, SCN2A Encephalopathy, SETBP1 Gene Mutation, SETD5, SMARCA4 Gene Mutation, SMARCC2, STXBP1 Encephalopathy With Epilepsy, SYNGAP1-Related Intellectual Disability, TBR1, ARHGEF9, HNRNPU, PPP3CA, PPP2R1A, SLC6A1, 2p16.3 Deletions, 5q35 Deletions, 5q35 Duplications, 7q11.23 Duplications, 15Q13.3 Deletion Syndrome, 16p11.2 Triplications, 16P12.2 Microdeletion, 16P13.11 Microdeletion Syndrome (Disorder), 17Q12 Microdeletion Syndrome (Disorder), 17Q12 Duplication Syndrome, 17Q21.31 Deletion Syndrome, 17q21.3 Duplications, ACTB, ADSL, AFF2, ALDH5A1, ANK3, ARX, ATRX Gene Mutation, AUTS2 Syndrome, BCKDK, BRSK2, CACNA1C, CAPRIN1, CASK, CASZ1, CHD3, CIC, CNOT3, CREBBP Gene Mutation, CSDE1, CTCF, DEAF1, DHCR7, DLG4, EBF3, EHMT1, EP300 Gene Mutation, GIGYF1, GRIN1, GRIN2D, IQSEC2-Related Syndromic Intellectual Disability, IRF2BPL, KANSL1, KCNB1, KDM3B, NEXMIF, KMT2A, MBOAT7, MEIS2, MYT1L, NAA15, NBEA, NCKAP1, NIPBL, NLGN2, NLGN3, NLGN4X, NR4A2, NRXN1, NRXN2, NSD1 Gene Mutation, PHF21A, PHF3, PHIP, PSMD12, RELN, RERE, RFX3, RIMS1, RORB, SCN1A, SETD2 Gene Mutation, SHANK2, SIN3A, SLC9A6, SON, SOX5, SPAST, SRCAP, TAOK1, TANC2, TCF20, TLK2, TRIO, TRIP12, UPF3B, USP9X, VPS13B, WAC, WDFY3, ZBTB20, ZNF292, ZNF462, 2Q37 Deletion Syndrome, 9q34 Duplications, 15q15 Deletions, 15Q24 Deletion, NR3C2, SYNCRIP, 2q37.3 Deletion, 6q16 Deletion, 15q11.2 BP1-BP2 Deletion, 16p13.3 Deletion, 17Q11.2 Microduplication Syndrome (Disorder), 17p13.3, Xq28 Duplication, CLCN4, CSNK2B, DYNC1H1, EIF3F, GNB1, MED13, MEF2C, RALGAPB, SCN1B, YY1, Xp11.22 Duplication, PACS2, MAOA, MAOB, HNRNPC, HNRNPD, HNRNPK, HNRNPR, HNRNPUL2, 5P Deletion Syndrome, TCF7L2 Gene Mutation, HECW2, PPM1D, RNU4-2, SNAP25, FOXP2, ITSN1
Interventions: Not listed
Lead sponsor: Simons Searchlight; Other
Eligibility: Not listed
Enrollment: 100,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2050
U.S. locations: 2
States / cities: Boston, Massachusetts • Lewisburg, Pennsylvania

Conditions: Laryngeal Diseases, Chronic Laryngeal Neuropathy
Interventions: Amitriptyline, Placebo; Drug · Other
Lead sponsor: Boston Medical Center; Other
Eligibility: 18 Years to 64 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 1
States / cities: Boston, Massachusetts

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, Charcot Marie Tooth Disease (CMT)
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

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: Charcot-Marie-Tooth Disease
Interventions: NMD670, Placebo; Drug
Lead sponsor: NMD Pharma A/S; Industry
Eligibility: 18 Years to 70 Years
Enrollment: 81 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2025
U.S. locations: 8
States / cities: Kansas City, Kansas • Boston, Massachusetts • Columbia, Missouri + 5 more

Conditions: Pancreatic Cancer, ATM, BRCA, Hereditary Cancer
Interventions: Not listed
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years and older
Enrollment: 12,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 1994 – 2032
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Myelofibrosis
Interventions: Ruxolitinib, 9-ING-41; Drug
Lead sponsor: Actuate Therapeutics Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 17 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2024
U.S. locations: 9
States / cities: Los Angeles, California • Augusta, Georgia • Rochester, Minnesota + 5 more

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

Conditions: Breast Cancer
Interventions: Alpelisib, Fulvestrant, Metformin XR, Dapagliflozin + metformin XR, Dapagliflozin; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years and older
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 1
States / cities: St Louis, Missouri

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

Conditions: Advanced Solid Tumors
Interventions: PX-866; Drug
Lead sponsor: Cascadian Therapeutics Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 2
States / cities: Aurora, Colorado • Houston, Texas

Conditions: Anatomic Stage I Breast Cancer AJCC v8, Anatomic Stage IA Breast Cancer AJCC v8, Anatomic Stage IB Breast Cancer AJCC v8, Anatomic Stage II Breast Cancer AJCC v8, Anatomic Stage IIA Breast Cancer AJCC v8, Anatomic Stage IIB Breast Cancer AJCC v8, Anatomic Stage III Breast Cancer AJCC v8, Anatomic Stage IIIA Breast Cancer AJCC v8, Anatomic Stage IIIB Breast Cancer AJCC v8, Anatomic Stage IIIC Breast Cancer AJCC v8, Grade 1 Peripheral Motor Neuropathy, CTCAE, Grade 1 Peripheral Sensory Neuropathy, CTCAE, Grade 2 Peripheral Motor Neuropathy, CTCAE, Grade 2 Peripheral Sensory Neuropathy, CTCAE, Prognostic Stage I Breast Cancer AJCC v8, Prognostic Stage IA Breast Cancer AJCC v8, Prognostic Stage IB Breast Cancer AJCC v8, Prognostic Stage II Breast Cancer AJCC v8, Prognostic Stage IIA Breast Cancer AJCC v8, Prognostic Stage IIB Breast Cancer AJCC v8, Prognostic Stage III Breast Cancer AJCC v8, Prognostic Stage IIIA Breast Cancer AJCC v8, Prognostic Stage IIIB Breast Cancer AJCC v8, Prognostic Stage IIIC Breast Cancer AJCC v8
Interventions: Acupuncture Therapy, Best Practice, Quality-of-Life Assessment, Questionnaire Administration; Procedure · Other
Lead sponsor: Wake Forest University Health Sciences; Other
Eligibility: 18 Years and older · Female only
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 1
States / cities: Winston-Salem, North Carolina

Conditions: Epilepsy, Tuberous Sclerosis Complex
Interventions: Everolimus; Drug
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 2 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2016
U.S. locations: 2
States / cities: Cincinnati, Ohio • Houston, Texas

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 – 2027
U.S. locations: 1
States / cities: Madison, Wisconsin

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

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

Conditions: Ovarian Cancer, Breast Cancer, Solid Tumor, Prostate Cancer, Endometrial Cancer
Interventions: CYH33; Drug
Lead sponsor: Haihe Biopharma Co., Ltd.; Industry
Eligibility: 18 Years and older
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 3
States / cities: New Haven, Connecticut • Dallas, Texas • Houston, Texas

Conditions: Chemotherapy-induced Peripheral Neuropathy
Interventions: Electroacupuncture, NeuroMetrix, Rydel-Seiffer tuning fork; Device
Lead sponsor: H. Lee Moffitt Cancer Center and Research Institute; Other
Eligibility: 18 Years and older
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: Tampa, Florida

Conditions: Diabetes Mellitus, Diabetic Neuropathy
Interventions: Anodyne Therapy System; Device
Lead sponsor: Scott and White Hospital & Clinic; Other
Eligibility: 25 Years and older
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 1
States / cities: Temple, Texas

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: Postherpetic Neuralgia, Diabetic Neuropathy, Complex Regional Pain Syndrome, Carpal Tunnel Syndrome, HIV Neuropathy, Idiopathic Sensory Neuropathy, Peripheral Neuropathy
Interventions: Placebo Capsules + Placebo Patch, Placebo capsules + Lidoderm®, Gabapentin + Placebo, Gabapentin + Lidoderm®, Gabapentin 300 mg capsules 1800 mg/day + placebo patch, Gabapentin 1800 mg/day + Lidoderm patch; Drug
Lead sponsor: Endo USA Inc., a Keenova Therapeutics Company; Industry
Eligibility: 18 Years and older
Enrollment: 62 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2003
U.S. locations: 7
States / cities: Birmingham, Alabama • Hueytown, Alabama • Phoenix, Arizona + 4 more

Conditions: Diabetic Neuropathy
Interventions: Ruboxistaurin; Drug
Lead sponsor: Chromaderm, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2005
U.S. locations: 1
States / cities: Norfolk, Virginia

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

SPENDD: Quantitative Sensory Testing and Analgesic Response for Painful Peripheral Neuropathy.

Online Study of People Who Have Genetic Changes and Features of Autism: Simons Searchlight

Trial of the Treatment of Chronic Laryngitis With Amitryptiline

Natural History Study for Charcot Marie Tooth Disease

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

Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease

Pancreatic Cancer Registry: For Any Person With a Personal or Family History

Actuate 1901: 9-ING-41 in Myelofibrosis

Study of Electrical Impedance Myography (EIM) in ALS

Study of Safety and Efficacy of Dapagliflozin + Metformin XR Versus Metformin XR in Participants With HR+, HER2-, Advanced Breast Cancer While on Treatment With Alpelisib and Fulvestrant

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

Phase I Trial of Oral PX-866

Acupuncture in Reducing Chemotherapy-Induced Peripheral Neuropathy in Participants With Stage I-III Breast Cancer

Everolimus (RAD001) Therapy for Epilepsy in Patients With Tuberous Sclerosis Complex (TSC)

Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient

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

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

Study of CYH33 in Combination With Olaparib an Oral PARP Inhibitor in Patients With Advanced Solid Tumors.

Feasibility Study for Electroacupuncture for Chemotherapy- Induced Peripheral Neuropathy (CIPN)

Anodyne Therapy in Diabetic Sensory Neuropathy

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

A Study Of Lidocaine Patch 5% Alone, Gabapentin Alone, And Lidocaine Patch 5% And Gabapentin In Combination For The Relief Of Pain In Patients With Diverse Peripheral Neuropathic Pain Conditions

The Effect of Ruboxistaurin on Small Fiber Function