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Conditions: Friedreich's Ataxia
Interventions: VP 20629, Placebo; Drug
Lead sponsor: Shire; Industry
Eligibility: 18 Years to 45 Years
Enrollment: 46 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 5
States / cities: Los Angeles, California • Tampa, Florida • Atlanta, Georgia + 2 more

Conditions: Friedreich Ataxia
Interventions: DT-216, DT-216 matching Placebo; Drug
Lead sponsor: Design Therapeutics, Inc.; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 39 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022
U.S. locations: 1
States / cities: Eatontown, New Jersey

Conditions: Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 10
Interventions: troriluzole, Placebo; Drug
Lead sponsor: Biohaven Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 299 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 21
States / cities: Phoenix, Arizona • Long Beach, California • Los Angeles, California + 16 more

Conditions: Spinal and Bulbar Muscular Atrophy, Kennedy's Disease
Interventions: AJ201, Placebo; Drug
Lead sponsor: AnnJi Pharmaceutical Co., Ltd.; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 6
States / cities: Orange, California • Palo Alto, California • Jacksonville, Florida + 3 more

Conditions: Neurofibromatosis 2, Vestibular Schwannoma
Interventions: Lapatinib; Drug
Lead sponsor: NYU Langone Health; Other
Eligibility: 4 Years to 80 Years
Enrollment: 21 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2012
U.S. locations: 1
States / cities: New York, New York

Conditions: Friedreich Ataxia
Interventions: TAK-831, TAK-831 Placebo; Drug
Lead sponsor: Neurocrine Biosciences; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 67 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2018
U.S. locations: 6
States / cities: Los Angeles, California • Gainesville, Florida • Tampa, Florida + 3 more

Conditions: Friedreich's Ataxia
Interventions: Interferon γ-1b, Placebo; Drug
Lead sponsor: Amgen; Industry
Eligibility: 10 Years to 25 Years
Enrollment: 92 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2016
U.S. locations: 4
States / cities: Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more

Conditions: Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia 3, Spinocerebellar Ataxia Type 6, MSA-C
Interventions: BHV-4157; Drug
Lead sponsor: University of California, Los Angeles; Other
Eligibility: 18 Years to 75 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2024
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Spinal Deformity
Interventions: ATEC IntraOperative Alignment (IOA) Imaging Platform, Standard 36" cassette imaging, EOS radiographs; Diagnostic Test
Lead sponsor: NYU Langone Health; Other
Eligibility: 18 Years and older
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 1
States / cities: New York, New York

Conditions: Spinal Deformity
Interventions: Not listed
Lead sponsor: International Spine Study Group Foundation; Other
Eligibility: 18 Years and older
Enrollment: 1,999 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2026
U.S. locations: 9
States / cities: La Jolla, California • Sacramento, California • San Francisco, California + 5 more

Conditions: Physical Disability
Interventions: EnhanceWellness for Disability (EW-D), Wellness Education; Behavioral
Lead sponsor: University of Washington; Other
Eligibility: 45 Years to 64 Years
Enrollment: 516 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 1
States / cities: Seattle, Washington

Conditions: Scoliosis, Scoliosis Kyphosis
Interventions: Spinal Fusion, No intervention; Procedure · Other
Lead sponsor: University of Kansas Medical Center; Other
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2028
U.S. locations: 1
States / cities: Kansas City, Kansas

Conditions: Adult Spinal Deformity
Interventions: Interventions; Procedure
Lead sponsor: AO Foundation, AO Spine; Other
Eligibility: 18 Years to 80 Years
Enrollment: 77 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 5
States / cities: San Francisco, California • Baltimore, Maryland • St Louis, Missouri + 2 more

Conditions: ER+, HER 2- Breast Cancer, Non-small Cell Lung Cancer (NSCLC), Castration-resistant Prostate Cancer (CRPC), Microsatellite Stable (MSS) Colorectal Carcinoma
Interventions: IDE574, Fulvestrant injection; Drug
Lead sponsor: IDEAYA Biosciences; Industry
Eligibility: 18 Years to 99 Years
Enrollment: 160 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 11
States / cities: Plantation, Florida • East Brunswick, New Jersey • Lake Success, New York + 7 more

Conditions: Stress, Job, PTSD, Hypervigilance, Hyperthyroidism, Migraine, Migraine With Aura, Immunodeficiency, Sickle Cell Trait, Sickle Cell Disease, Anemia, Sickle Cell, Anemia, Fatigue, Anxiety, Depression, Flat Feet, Lordosis, TBI (Traumatic Brain Injury), Rheumatoid Arthritis, Arthritis, Masturbation, Priapism, Erectile Dysfunction, Flight Reaction, Hypertension, Sexual Arousal Disorder, Hemorrhoids, Fibroid
Interventions: Holistic Medicine (HM); Behavioral
Lead sponsor: Goddess Zena I. Jones; Other
Eligibility: 22 Years and older
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Anchorage, Alaska

Conditions: Scoliosis, Kyphosis, Kyphoscoliosis, Spinal Deformity, Spondylosis
Interventions: Not listed
Lead sponsor: Mazor Robotics; Industry
Eligibility: 21 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 1
States / cities: Kansas City, Kansas

Conditions: Spinocerebellar Ataxia Type 3
Interventions: SLS-005, Placebo; Drug
Lead sponsor: Seelos Therapeutics, Inc.; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 23 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2023
U.S. locations: 7
States / cities: Los Angeles, California • Aurora, Colorado • Tampa, Florida + 4 more

Conditions: Spinal Curvatures
Interventions: Surgical intervention, None (Asymptomatic group); Procedure · Other
Lead sponsor: Texas Back Institute; Other
Eligibility: 30 Years and older
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2018
U.S. locations: 1
States / cities: Plano, Texas

Conditions: Spina Bifida, Myelomeningocele, Meningocele, Bladder Exstrophy, Genitourinary Congenital Anomalies, Cloacal Exstrophy
Interventions: Not listed
Lead sponsor: The Methodist Hospital Research Institute; Other
Eligibility: 18 Years and older
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2024
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Friedreich's Ataxia
Interventions: interferon γ-1b; Drug
Lead sponsor: Amgen; Industry
Eligibility: 11 Years to 27 Years
Enrollment: 38 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 4
States / cities: Los Angeles, California • Gainesville, Florida • Iowa City, Iowa + 1 more

Conditions: Gut Microbiome, Gastrointestinal Microbiome, Autism, Autism Spectrum Disorder, Autism Spectrum Disorder High-Functioning, Alzheimer Disease, Alzheimer Dementia (AD), Alzheimer Dementia, Alzheimer Disease (AD), Crohn, Crohn Colitis, Crohn Disease (CD), Crohn Disease Colon, Myalgic Encephalomyelitis, ME/CFS, Myalgic Encephalomyelitis (ME), Myalgic Encephalomyelitis/Chronic Fatigue Syndrome, Psoriasis, Psoriasis Annularis, Psoriasis Chronic, Chronic Urinary Tract Infection, Ulcerative Colitis (Disorder), Ulcerative Colitis (UC), Ulcerative Colitis Acute, Ulcerative Colitis Chronic, Multiple Sclerosis, Multiple Sclerosis (MS) - Relapsing-remitting, Multiple Sclerosis (MS) Primary Progressive, Constipation Chronic Idiopathic, Constipation, Celiac, Celiac Disease, Celiac Sprue, Lyme Arthritis, Lyme Borreliosis, Nervous System, Lyme Disease, Chronic, Cholesterol, Cholesterol Level, High, Cancer, Colon Cancer, Amyotrophic Lateral Sclerosis (ALS), Amyotrophic Lateral Sclerosis, Rheumatoid Arthritis (RA), Rheumatoid Arthritis - Rheumatism, Chronic Fatigue Syndrome (CFS), PARKINSON DISEASE (Disorder), Parkinson Disease, Parkinson Disease (PD), Depression, Major Depressive Disorder (MDD), Depression Disorder, Depression in Adults, Anxiety, Anxiety Disorder Generalized, Obsessive Compulsive Disorder (OCD), Obsessive Compulsive Disorder OCD, Obsessive - Compulsive Disorder, Bipolar, Bipolar 1 Disorder, Bipolar Disorder (BD), Bipolar Disorder I and II, Bipolar and Related Disorders, Migraine, Migraine Disorder, Diabetes (DM), Diabetes, Lupus, Lupus Erythematosus, Epidermolysis Bullosa (EB), Mesothelioma, Mesothelioma Malignant, IBS (Irritable Bowel Syndrome), Irritable Bowel, IBS - Irritable Bowel Syndrome, Irritable Bowel Syndrome (IBS), Eczema, Eczema Atopic Dermatitis, Acne, Myasthaenia Gravis, Gout
Interventions: There is no intervention in this study; Other
Lead sponsor: ProgenaBiome; Other
Eligibility: 12 Years and older
Enrollment: 1,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2030
U.S. locations: 1
States / cities: Ventura, California

Conditions: Adult Spinal Deformity
Interventions: Routinely performed surgical correction of spinal deformity; Procedure
Lead sponsor: AO Foundation, AO Spine; Other
Eligibility: 18 Years to 80 Years
Enrollment: 273 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2015
U.S. locations: 8
States / cities: San Francisco, California • Louisville, Kentucky • Baltimore, Maryland + 5 more

Conditions: Adult Spinal Deformity, Scoliosis, Kyphosis
Interventions: Index or revision spine surgery for complex adult spinal deformity; Procedure
Lead sponsor: International Spine Study Group Foundation; Other
Eligibility: 18 Years and older
Enrollment: 225 participants
Timeline: 2021 – 2034
U.S. locations: 9
States / cities: Phoenix, Arizona • La Jolla, California • San Francisco, California + 6 more

Conditions: Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome, Prenatal Benign Hypophosphatasia, Perinatal Lethal Hypophosphatasia, Odontohypophosphatasia, Adult Hypophosphatasia, Childhood-onset Hypophosphatasia, Infantile Hypophosphatasia, Hypophosphatasia, Kabuki Syndrome, Bohring-Opitz Syndrome, Narcolepsy Without Cataplexy, Narcolepsy-cataplexy, Hypersomnolence Disorder, Idiopathic Hypersomnia Without Long Sleep Time, Idiopathic Hypersomnia With Long Sleep Time, Idiopathic Hypersomnia, Kleine-Levin Syndrome, Kawasaki Disease, Leiomyosarcoma, Leiomyosarcoma of the Corpus Uteri, Leiomyosarcoma of the Cervix Uteri, Leiomyosarcoma of Small Intestine, Acquired Myasthenia Gravis, Addison Disease, Hyperacusis (Hyperacousis), Juvenile Myasthenia Gravis, Transient Neonatal Myasthenia Gravis, Williams Syndrome, Lyme Disease, Myasthenia Gravis, Marinesco Sjogren Syndrome(Marinesco-Sjogren Syndrome), Isolated Klippel-Feil Syndrome, Frasier Syndrome, Denys-Drash Syndrome, Beckwith-Wiedemann Syndrome, Emanuel Syndrome, Isolated Aniridia, Axenfeld-Rieger Syndrome, Aniridia-intellectual Disability Syndrome, Aniridia - Renal Agenesis - Psychomotor Retardation, Aniridia - Ptosis - Intellectual Disability - Familial Obesity, Aniridia - Cerebellar Ataxia - Intellectual Disability, Aniridia - Absent Patella, Aniridia, Peters Anomaly - Cataract, Peters Anomaly, Potocki-Shaffer Syndrome, Silver-Russell Syndrome Due to Maternal Uniparental Disomy of Chromosome 11, Silver-Russell Syndrome Due to Imprinting Defect of 11p15, Silver-Russell Syndrome Due to 11p15 Microduplication, Syndromic Aniridia, WAGR Syndrome, Wolf-Hirschhorn Syndrome, 4p16.3 Microduplication Syndrome, 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome, Autosomal Recessive Stickler Syndrome, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Stickler Syndrome, Mucolipidosis Type 4, X-linked Spinocerebellar Ataxia Type 4, X-linked Spinocerebellar Ataxia Type 3, X-linked Intellectual Disability - Ataxia - Apraxia, X-linked Progressive Cerebellar Ataxia, X-linked Non Progressive Cerebellar Ataxia, X-linked Cerebellar Ataxia, Vitamin B12 Deficiency Ataxia, Toxic Exposure Ataxia, Unclassified Autosomal Dominant Spinocerebellar Ataxia, Thyroid Antibody Ataxia, Sporadic Adult-onset Ataxia of Unknown Etiology, Spinocerebellar Ataxia With Oculomotor Anomaly, Spinocerebellar Ataxia With Epilepsy, Spinocerebellar Ataxia With Axonal Neuropathy Type 2, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 5, Spinocerebellar Ataxia Type 4, Spinocerebellar Ataxia Type 37, Spinocerebellar Ataxia Type 36, Spinocerebellar Ataxia Type 35, Spinocerebellar Ataxia Type 34, Spinocerebellar Ataxia Type 32, Spinocerebellar Ataxia Type 31, Spinocerebellar Ataxia Type 30, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 29, Spinocerebellar Ataxia Type 28, Spinocerebellar Ataxia Type 27, Spinocerebellar Ataxia Type 26, Spinocerebellar Ataxia Type 25, Spinocerebellar Ataxia Type 23, Spinocerebellar Ataxia Type 22, Spinocerebellar Ataxia Type 21, Spinocerebellar Ataxia Type 20, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 19/22, Spinocerebellar Ataxia Type 18, Spinocerebellar Ataxia Type 17, Spinocerebellar Ataxia Type 16, Spinocerebellar Ataxia Type 15/16, Spinocerebellar Ataxia Type 14, Spinocerebellar Ataxia Type 13, Spinocerebellar Ataxia Type 12, Spinocerebellar Ataxia Type 11, Spinocerebellar Ataxia Type 10, Spinocerebellar Ataxia Type 1 With Axonal Neuropathy, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia - Unknown, Spinocerebellar Ataxia - Dysmorphism, Non Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature, Spasticity-ataxia-gait Anomalies Syndrome, Spastic Ataxia With Congenital Miosis, Spastic Ataxia - Corneal Dystrophy, Spastic Ataxia, Rare Hereditary Ataxia, Rare Ataxia, Recessive Mitochondrial Ataxia Syndrome, Progressive Epilepsy and/or Ataxia With Myoclonus as a Major Feature, Posterior Column Ataxia - Retinitis Pigmentosa, Post-Stroke Ataxia, Post-Head Injury Ataxia, Post Vaccination Ataxia, Polyneuropathy - Hearing Loss - Ataxia - Retinitis Pigmentosa - Cataract, Muscular Atrophy - Ataxia - Retinitis Pigmentosa - Diabetes Mellitus, Non-hereditary Degenerative Ataxia, Paroxysmal Dystonic Choreathetosis With Episodic Ataxia and Spasticity, Olivopontocerebellar Atrophy - Deafness, NARP Syndrome, Myoclonus - Cerebellar Ataxia - Deafness, Multiple System Atrophy, Parkinsonian Type, Multiple System Atrophy, Cerebellar Type, Multiple System Atrophy, Maternally-inherited Leigh Syndrome, Machado-Joseph Disease Type 3, Machado-Joseph Disease Type 2, Machado-Joseph Disease Type 1, Leigh Syndrome, Late-onset Ataxia With Dementia, Infection or Post Infection Ataxia, GAD Ataxia, Hereditary Episodic Ataxia, Gliadin/Gluten Ataxia, Friedreich Ataxia, Fragile X-associated Tremor/Ataxia Syndrome, Familial Paroxysmal Ataxia, Exposure to Medications Ataxia, Episodic Ataxia With Slurred Speech, Episodic Ataxia Unknown Type, Episodic Ataxia Type 7, Episodic Ataxia Type 6, Episodic Ataxia Type 5, Episodic Ataxia Type 4, Episodic Ataxia Type 3, Episodic Ataxia Type 1, Epilepsy and/or Ataxia With Myoclonus as Major Feature, Early-onset Spastic Ataxia-neuropathy Syndrome, Early-onset Progressive Neurodegeneration - Blindness - Ataxia - Spasticity, Early-onset Cerebellar Ataxia With Retained Tendon Reflexes, Early-onset Ataxia With Dementia, Childhood-onset Autosomal Recessive Slowly Progressive Spinocerebellar Ataxia, Dilated Cardiomyopathy With Ataxia, Cataract - Ataxia - Deafness, Cerebellar Ataxia, Cayman Type, Cerebellar Ataxia With Peripheral Neuropathy, Cerebellar Ataxia - Hypogonadism, Cerebellar Ataxia - Ectodermal Dysplasia, Cerebellar Ataxia - Areflexia - Pes Cavus - Optic Atrophy - Sensorineural Hearing Loss, Brain Tumor Ataxia, Brachydactyly - Nystagmus - Cerebellar Ataxia, Benign Paroxysmal Tonic Upgaze of Childhood With Ataxia, Autosomal Recessive Syndromic Cerebellar Ataxia, Autosomal Recessive Spastic Ataxia With Leukoencephalopathy, Autosomal Recessive Spastic Ataxia of Charlevoix-Saguenay, Autosomal Recessive Spastic Ataxia - Optic Atrophy - Dysarthria, Autosomal Recessive Spastic Ataxia, Autosomal Recessive Metabolic Cerebellar Ataxia, Autosomal Dominant Spinocerebellar Ataxia Due to Repeat Expansions That do Not Encode Polyglutamine, Autosomal Recessive Ataxia, Beauce Type, Autosomal Recessive Ataxia Due to Ubiquinone Deficiency, Autosomal Recessive Ataxia Due to PEX10 Deficiency, Autosomal Recessive Degenerative and Progressive Cerebellar Ataxia, Autosomal Recessive Congenital Cerebellar Ataxia Due to MGLUR1 Deficiency, Autosomal Recessive Congenital Cerebellar Ataxia Due to GRID2 Deficiency, Autosomal Recessive Congenital Cerebellar Ataxia, Autosomal Recessive Cerebellar Ataxia-pyramidal Signs-nystagmus-oculomotor Apraxia Syndrome, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to WWOX Deficiency, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to TUD Deficiency, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome Due to KIAA0226 Deficiency, Autosomal Recessive Cerebellar Ataxia-epilepsy-intellectual Disability Syndrome, Autosomal Recessive Cerebellar Ataxia With Late-onset Spasticity, Autosomal Recessive Cerebellar Ataxia Due to STUB1 Deficiency, Autosomal Recessive Cerebellar Ataxia Due to a DNA Repair Defect, Autosomal Recessive Cerebellar Ataxia - Saccadic Intrusion, Autosomal Recessive Cerebellar Ataxia - Psychomotor Retardation, Autosomal Recessive Cerebellar Ataxia - Blindness - Deafness, Autosomal Recessive Cerebellar Ataxia, Autosomal Dominant Spinocerebellar Ataxia Due to a Polyglutamine Anomaly, Autosomal Dominant Spinocerebellar Ataxia Due to a Point Mutation, Autosomal Dominant Spinocerebellar Ataxia Due to a Channelopathy, Autosomal Dominant Spastic Ataxia Type 1, Autosomal Dominant Spastic Ataxia, Autosomal Dominant Optic Atrophy, Ataxia-telangiectasia Variant, Ataxia-telangiectasia, Autosomal Dominant Cerebellar Ataxia, Deafness and Narcolepsy, Autosomal Dominant Cerebellar Ataxia Type 4, Autosomal Dominant Cerebellar Ataxia Type 3, Autosomal Dominant Cerebellar Ataxia Type 2, Autosomal Dominant Cerebellar Ataxia Type 1, Autosomal Dominant Cerebellar Ataxia, Ataxia-telangiectasia-like Disorder, Ataxia With Vitamin E Deficiency, Ataxia With Dementia, Ataxia - Oculomotor Apraxia Type 1, Ataxia - Other, Ataxia - Genetic Diagnosis - Unknown, Acquired Ataxia, Adult-onset Autosomal Recessive Cerebellar Ataxia, Alcohol Related Ataxia, Multiple Endocrine Neoplasia, Multiple Endocrine Neoplasia Type II, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Multiple Endocrine Neoplasia, Type IV, Multiple Endocrine Neoplasia, Type 3, Multiple Endocrine Neoplasia (MEN) Syndrome, Multiple Endocrine Neoplasia Type 2B, Multiple Endocrine Neoplasia Type 2A, Atypical Hemolytic Uremic Syndrome, Atypical HUS, Wiedemann-Steiner Syndrome, Breast Implant-Associated Anaplastic Large Cell Lymphoma, Autoimmune/Inflammatory Syndrome Induced by Adjuvants (ASIA), Hemophagocytic Lymphohistiocytosis, Behcet's Disease, Alagille Syndrome, Inclusion Body Myopathy With Early-onset Paget Disease and Frontotemporal Dementia (IBMPFD), Lowe Syndrome, Pitt Hopkins Syndrome, 1p36 Deletion Syndrome, Jansen Type Metaphyseal Chondrodysplasia, Cockayne Syndrome, Chronic Recurrent Multifocal Osteomyelitis, CRMO, Malan Syndrome, Hereditary Sensory and Autonomic Neuropathy Type Ie, VCP Disease, Hypnic Jerking, Sleep Myoclonus, Mollaret Meningitis, Recurrent Viral Meningitis, CRB1, Leber Congenital Amaurosis, Retinitis Pigmentosa, Rare Retinal Disorder, KCNMA1-Channelopathy, Primary Biliary Cirrhosis, ZMYND11, Transient Global Amnesia, Glycogen Storage Disease, Alstrom Syndrome, White Sutton Syndrome, DNM1, EIEE31, Myhre Syndrome, Recurrent Respiratory Papillomatosis, Laryngeal Papillomatosis, Tracheal Papillomatosis, Refsum Disease, Nicolaides Baraitser Syndrome, Leukodystrophy, Tango2, Cauda Equina Syndrome, Rare Gastrointestinal Disorders, Achalasia-Addisonian Syndrome, Achalasia Cardia, Achalasia Icrocephaly Syndrome, Anal Fistula, Congenital Sucrase-Isomaltase Deficiency, Eosinophilic Gastroenteritis, Idiopathic Gastroparesis, Hirschsprung Disease, Rare Inflammatory Bowel Disease, Intestinal Pseudo-Obstruction, Scleroderma, Short Bowel Syndrome, Sacral Agenesis, Sacral Agenesis Syndrome, Caudal Regression, Scheuermann Disease, SMC1A Truncated Mutations (Causing Loss of Gene Function), Cystinosis, Juvenile Nephropathic Cystinosis, Nephropathic Cystinosis, Kennedy Disease, Spinal Bulbar Muscular Atrophy, Warburg Micro Syndrome, Mucolipidoses, Mitochondrial Diseases, Mitochondrial Aminoacyl-tRNA Synthetases, Mt-aaRS Disorders, Hypertrophic Olivary Degeneration, Non-Ketotic Hyperglycinemia, Fish Odor Syndrome, Halitosis, Isolated Congenital Asplenia, Lambert Eaton (LEMS), Biliary Atresia, STAG1 Gene Mutation, Coffin Lowry Syndrome, Borjeson-Forssman-Lehman Syndrome, Blau Syndrome, Arginase 1 Deficiency, HSPB8 Myopathy, Beta-Mannosidosis, TBX4 Syndrome, DHDDS Gene Mutations, MAND-MBD5-Associated Neurodevelopmental Disorder, Constitutional Mismatch Repair Deficiency (CMMRD), SPATA5 Disorder, SPATA5L1 Related Disorder, Acrodysostosis, Multi-systematic Smooth Muscle Dysfunction Syndrome, CRELD1 (Cysteine Rich With EGF Like Domains 1), GNB1 Syndrome, Pyruvate Dehydrogenase Complex Deficiency Disease, Beta Mannosidosis, Kbg Syndrome, Labrune Syndrome, Metachromatic Leukodystrophy (MLD), Moyamoya Disease, OPHN1 Syndrome, Oculopharyngeal Muscular Dystrophy (OPMD), TUBB3 Mutation, WOREE (WWOX-related Epileptic Encephalopathy, SCAR12, Skraban-Deardorff Syndrome, Hereditary Myopathy With Early Respiratory Failure
Interventions: Not listed
Lead sponsor: Sanford Health; Other
Eligibility: Not listed
Enrollment: 20,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2100
U.S. locations: 1
States / cities: Sioux Falls, South Dakota

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Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia

Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

Troriluzole in Adult Participants With Spinocerebellar Ataxia

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Of AJ201 In Patients

Lapatinib Study for Children and Adults With Neurofibromatosis Type 2 (NF2) and NF2-Related Tumors

Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia

Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia

Open Pilot Trial of BHV-4157

Intraoperative Alignment in Adult Spinal Deformity Surgery

Prospective, Multi-Center Adult Spinal Deformity Outcomes Database Registry

A Community Wellness Program for Adults Living With Long-term Physical Disability

Effect of Adult Spinal Deformity Surgery on Functional Reach

Neurologic Complications in Spinal Deformity Surgery - Extension

A Study to Investigate the Safety, Pharmacokinetics, and Preliminary Efficacy of IDE574 Therapy in Adult Participants With Advanced Solid Tumors

The Sequestration of Holistic Stress Management Techniques for Adults

ADDRESS - Adult Deformity Robotic vs. Freehand Surgery to Correct Spinal Deformity

STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia

Gait and Balance in Thoracolumbar Spinal Deformity

Prospective Cohort of Transitional Urology Patients

Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia

A Non-Interventional Pilot Study to Explore the Role of Gut Flora in Disease

Neurologic Complications in Spinal Deformity Surgery

Prospective, Multicenter, Case-Control Analysis of the VersaTie Posterior Fixation System to Prevent Proximal Junctional Failure in Long Posterior Spinal Fusion Constructs for Adult Patients

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford