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Conditions: Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication &#X7C; Blood or Tissue &#X7C; Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions: Not listed
Lead sponsor: Children's Hospital of Philadelphia; Other
Eligibility: Not listed
Enrollment: 12,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2030
U.S. locations: 23
States / cities: Los Angeles, California • Orange, California • Palo Alto, California + 18 more

Conditions: Corneal Disease, Neuropathy, Dry Eye Disease, Neurotrophic Keratitis
Interventions: Quantitative Sensory Test; Device
Lead sponsor: Tufts Medical Center; Other
Eligibility: 18 Years and older
Enrollment: 108 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Alzheimer's Disease
Interventions: CERE-110: Adeno-Associated Virus injection Surgery, Placebo Surgery; Drug · Procedure
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 55 Years to 80 Years
Enrollment: 49 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2015
U.S. locations: 10
States / cities: Birmingham, Alabama • Los Angeles, California • San Diego, California + 7 more

Conditions: Breast Carcinoma
Interventions: Medical Device Usage and Evaluation, Questionnaire Administration; Device · Other
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years and older
Enrollment: 94 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2023
U.S. locations: 1
States / cities: Jacksonville, Florida

Conditions: Cryptogenic Sensory Polyneuropathy
Interventions: Nortriptyline, Duloxetine, Pregabalin, Mexiletine; Drug
Lead sponsor: University of Kansas Medical Center; Other
Eligibility: 30 Years and older
Enrollment: 402 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 45
States / cities: Phoenix, Arizona • Los Angeles, California • San Francisco, California + 37 more

Conditions: Breast Cancer, Solid Tumors, Adult
Interventions: STX-478, Fulvestrant, Ribociclib, Palbociclib, Letrozole, Anastrozole, Exemestane, Tamoxifen, Abemaciclib, Imlunestrant, Metformin; Drug
Lead sponsor: Eli Lilly and Company; Industry
Eligibility: 18 Years and older
Enrollment: 880 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2030
U.S. locations: 28
States / cities: Los Angeles, California • San Francisco, California • Aurora, Colorado + 22 more

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

Conditions: Metastatic Cancer
Interventions: Palliative radiotherapy; Radiation
Lead sponsor: Memorial Sloan Kettering Cancer Center; Other
Eligibility: 18 Years and older
Enrollment: 45 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 7
States / cities: Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 4 more

Conditions: Amyotrophic Lateral Sclerosis, Hereditary Spastic Paraplegia, Primary Lateral Sclerosis, Progressive Muscular Atrophy, Frontotemporal Dementia
Interventions: Not listed
Lead sponsor: University of Miami; Other
Eligibility: 7 Years and older
Enrollment: 217 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2031
U.S. locations: 3
States / cities: Miami, Florida • Kansas City, Kansas • Philadelphia, Pennsylvania

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: 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: Entrapment Neuropathy
Interventions: Cryo-Touch III Device; Device
Lead sponsor: Pacira Pharmaceuticals, Inc; Industry
Eligibility: 18 Years and older
Enrollment: 7 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 2
States / cities: Redwood City, California • Overland Park, Kansas

Conditions: Breast Cancer
Interventions: Gedatolisib, Palbociclib, Letrozole, Fulvestrant; Drug
Lead sponsor: Celcuity Inc; Industry
Eligibility: 18 Years and older · Female only
Enrollment: 141 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2022
U.S. locations: 41
States / cities: Birmingham, Alabama • Corona, California • Fountain Valley, California + 17 more

Conditions: Familial Dysautonomia
Interventions: Phosphatidylserine; Dietary Supplement
Lead sponsor: NYU Langone Health; Other
Eligibility: 12 Years and older
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2019
U.S. locations: 1
States / cities: New York, New York

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

Conditions: Hereditary Spastic Paraplegia, SPG47, SPG50, SPG51, SPG52, AP4-related Hereditary Spastic Paraplegia, Early Onset Hereditary Spastic Paraplegia, SPG4, SPG3A, SPG15, SPG11
Interventions: Not listed
Lead sponsor: Boston Children's Hospital; Other
Eligibility: Up to 30 Years
Enrollment: 700 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2030
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Incurable Metastatic Colorectal Carcinoma, Incurable Progressive, Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck
Interventions: PX-866 (SCCHN), Cetuximab (SCCHN), PX-866 (CRC), Cetuximab (CRC); Drug
Lead sponsor: Cascadian Therapeutics Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 178 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2014
U.S. locations: 31
States / cities: Birmingham, Alabama • Escondido, California • Monterey, California + 24 more

Conditions: Advanced Solid Tumours
Interventions: Ceralasertib; Drug
Lead sponsor: AstraZeneca; Industry
Eligibility: 18 Years to 130 Years
Enrollment: 54 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2025
U.S. locations: 12
States / cities: Duarte, California • Los Angeles, California • San Francisco, California + 9 more

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

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: Familial Dysautonomia
Interventions: Dexmedetomidine Sublingual; Drug
Lead sponsor: NYU Langone Health; Other
Eligibility: 18 Years to 80 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2028
U.S. locations: 1
States / cities: New York, New York

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: Melanoma
Interventions: Vemurafenib; Drug
Lead sponsor: Philip Friedlander; Other
Eligibility: 18 Years and older
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 1
States / cities: New York, New York

Conditions: Hyperglycemia Drug Induced
Interventions: REMD-477; Biological
Lead sponsor: Duke University; Other
Eligibility: 18 Years and older
Enrollment: 1 participant
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: Durham, North Carolina

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The Myelin Disorders Biorepository Project

QST for Corneal Nerve Function

Randomized, Controlled Study Evaluating CERE-110 in Subjects With Mild to Moderate Alzheimer's Disease

Cryotherapy for the Prevention of Taxane-Induced Sensory Neuropathy of the Hands and Feet in Breast Cancer Patients

Patient Assisted Intervention for Neuropathy: Comparison of Treatment in Real Life Situations

First-in-Human Study of Tersolisib (STX-478) as Monotherapy and in Combination With Other Antineoplastic Agents in Participants With Advanced Solid Tumors

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

A Study of Reduced-dose Radiation in People With Metastatic Tumors With a Genetic Change

Phenotype, Genotype and Biomarkers 2

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

Actuate 1901: 9-ING-41 in Myelofibrosis

Study Evaluating Treatment of Forefoot Pain Related to Nerve Entrapment Using the Cryo-Touch III Device

A Study To Assess The Tolerability And Clinical Activity Of Gedatolisib In Combination With Palbociclib/Letrozole Or Palbociclib/Fulvestrant In Women With Metastatic Breast Cancer

The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia

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

Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia

Phase 1 and 2 Study of PX-866 and Cetuximab

A Study Investigating DNA-damage Response Agents in Molecularly Altered Advanced Cancer

Natural History Study for Charcot Marie Tooth Disease

Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease

Pilot Study of Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia

Study of Electrical Impedance Myography (EIM) in ALS

The Effects of Treatment With Vemurafenib on the Immune System in Advanced Melanoma

Glucagon Receptor Inhibition to Enable Breast Cancer Patients to Benefit From PI3K Inhibitor Therapy (REMD-477)