- Conditions
- Tuberous Sclerosis Complex
- Interventions
- Not listed
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 7 Years to 65 Years
- Enrollment
- 450 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2013
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
Search Results
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Showing 1–24
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- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- RTI International
- Other
- Eligibility
- 1 Day to 31 Days
- Enrollment
- 30,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- Systemic Lupus Erythematosus, Lupus Nephritis, Systemic Sclerosis, Idiopathic Inflammatory Myopathy
- Interventions
- KITE-363, Fludarabine, Cyclophosphamide
- Biological · Drug
- Lead sponsor
- Kite, A Gilead Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 52 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2029
- U.S. locations
- 4
- States / cities
- Duarte, California • Stanford, California • Tampa, Florida + 1 more
- Conditions
- Pyelonephritis, Pyelonephritis Acute, Renal Sclerosis
- Interventions
- Quick MRI
- Diagnostic Test
- Lead sponsor
- University of Wisconsin, Madison
- Other
- Eligibility
- 0 Years to 21 Years
- Enrollment
- 100 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2025
- U.S. locations
- 1
- States / cities
- Madison, Wisconsin
- Conditions
- Antineutrophilic Cytoplasmic Antibody (ANCA)- Associated Vasculitis (AAV), Idiopathic Inflammatory Myositis (IIM), Systemic Sclerosis (SSc), Systemic Lupus Erythematosus (SLE), Lupus Nephritis
- Interventions
- FT819, Fludarabine, Cyclophosphamide, Bendamustine
- Drug
- Lead sponsor
- Fate Therapeutics
- Industry
- Eligibility
- 12 Years to 70 Years
- Enrollment
- 244 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2042
- U.S. locations
- 16
- States / cities
- Beverly Hills, California • Fullerton, California • Irvine, California + 13 more
- Conditions
- Proteinuric Kidney Disease, Diabetic Nephropathy, Hypertensive Nephrosclerosis, IgA Nephropathy, Focal Segmental Glomerulosclerosis, Glomerulopathy (Obesity-associated), Glomerulonephritis, Membranous
- Interventions
- Aliskiren, Valsartan
- Drug
- Lead sponsor
- Columbia University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- New York, New York
- Conditions
- Idiopathic Inflammatory Myopathies, Diffuse Cutaneous Systemic Sclerosis, SLE Nephritis, ANCA Associated Vasculitis
- Interventions
- KYV-101, Cyclophosphamide, Fludarabine
- Drug
- Lead sponsor
- David Porter
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2027
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Systemic Lupus Erythematosus, Lupus Nephritis, Idiopathic Inflammatory Myopathies, Diffuse Cutaneous Systemic Sclerosis
- Interventions
- CNTY-101, IL-2, Lymphodepleting Chemotherapy
- Biological · Drug
- Lead sponsor
- Century Therapeutics, Inc.
- Industry
- Eligibility
- 17 Years and older
- Enrollment
- 6 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2028
- U.S. locations
- 6
- States / cities
- Los Angeles, California • Sacramento, California • Chicago, Illinois + 3 more
- Conditions
- Prostate Cancer, Renal Cancer, Brain Cancer, ALS
- Interventions
- Not listed
- Lead sponsor
- Duke University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2017
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Autoimmune Disorders, Systemic Sclerosis, Systemic Lupus Erythematosus, Lupus, Lupus Nephritis
- Interventions
- Tafasitamab, Fludarabine phosphate, Cyclophosphamide, Tafasitamab and NK cells
- Drug
- Lead sponsor
- M.D. Anderson Cancer Center
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 47 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024 – 2030
- U.S. locations
- 2
- States / cities
- Houston, Texas
- Conditions
- Amyotrophic Lateral Sclerosis (ALS), Angina, Stable, Anxiety, Asthma, Atrial Fibrillation, Cancer Brain, Cancer, Breast, Cancer Colon, Cancer, Lung, Cancer, Ovarian, Cancer Prostate, Cancer Skin, Throat Cancer, Lymphoma, Cancer, Thyroid, Cancer, Other, Cerebral Palsy, Chronic Fatigue Syndrome, Cluster Headache, Chronic Obstructive Pulmonary Disease (COPD), Chronic Kidney Diseases, Crohn Disease, Deafness, Depression, Diabetes, Eczema, Epilepsy, Fibroids, Fibromyalgia, Heart Failure, Hypertension, Hyperthyroidism, Hypothyroidism, Irritable Bowel Syndrome (IBS), Infertility, Lyme Disease, Macular Degeneration, Migraine, Multiple Allergies, Multiple Sclerosis, Osteoarthritis, Osteoporosis, Ovarian Cysts, Parkinson Disease, Phantom Limb Pain, Psoriasis, Post Traumatic Stress Disorder (PTSD), Rheumatoid Arthritis, Sjogrens Disease, Spinal Cord Injury, Spinal Stenosis, Stroke, Tension Headache, Tinnitus, Ulcerative Colitis
- Interventions
- Advanced multi-component meditation practice
- Behavioral
- Lead sponsor
- Tobias Moeller-Bertram
- Industry
- Eligibility
- 21 Years and older
- Enrollment
- 3,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Palm Desert, California
- Conditions
- Nonmalignant Neoplasm, Tuberous Sclerosis, Lymphangioleimyomatosis, Kidney Angiomyolipoma
- Interventions
- Rapamycin
- Drug
- Lead sponsor
- Dana-Farber Cancer Institute
- Other
- Eligibility
- 3 Years to 65 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2010
- U.S. locations
- 7
- States / cities
- Loma Linda, California • Hartford, Connecticut • Boston, Massachusetts + 4 more
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
- Interventions
- Not listed
- Lead sponsor
- Sanguine Biosciences
- Industry
- Eligibility
- 18 Years to 100 Years
- Enrollment
- 17,667 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2024
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Systemic Lupus Erythematosus, Lupus Nephritis, Systemic Sclerosis
- Interventions
- SYNCAR-001, STK-009
- Drug
- Lead sponsor
- Synthekine
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2041
- U.S. locations
- 6
- States / cities
- Scottsdale, Arizona • Hackensack, New Jersey • Manhasset, New York + 3 more
- Conditions
- Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
- Interventions
- Everolimus (RAD001), Everolimus Placebo
- Drug
- Lead sponsor
- Novartis Pharmaceuticals
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 118 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2015
- U.S. locations
- 6
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Boston, Massachusetts + 3 more
- Conditions
- Focal Segmental Glomerulosclerosis, Steroid Resistant Nephrotic Syndrome
- Interventions
- D-Galactose
- Drug
- Lead sponsor
- Children's National Research Institute
- Other
- Eligibility
- 2 Years to 21 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2009 – 2013
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Refractory Autoimmune Diseases
- Interventions
- BMS-986515, Fludarabine, Cyclophosphamide, Tocilizumab
- Genetic · Drug
- Lead sponsor
- Juno Therapeutics, Inc., a Bristol-Myers Squibb Company
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 125 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 2
- States / cities
- Boston, Massachusetts • Durham, North Carolina
- Conditions
- Refractory Lupus Nephritis, Myasthaenia Gravis, Stiff Person Syndrome, Rheumatoid Arthritis (RA), Multiple Sclerosis, Dermatomyositis, Systemic Sclerosis (SSc)
- Interventions
- KYV-101
- Drug
- Lead sponsor
- Kyverna Therapeutics
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 70 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2041
- U.S. locations
- 1
- States / cities
- Denver, Colorado
- Conditions
- Lupus Nephritis, Systemic Lupus Erythematosus, Systemic Sclerosis (SSc), Diffuse Cutaneous Systemic Sclerosis
- Interventions
- zamtocabtagene autoleucel, Cyclophosphamide, Fludarabine
- Biological · Drug
- Lead sponsor
- Miltenyi Biomedicine GmbH
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 48 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2028
- U.S. locations
- 1
- States / cities
- Milwaukee, Wisconsin
- Conditions
- Amyotrophic Lateral Sclerosis, Renal Insufficiency
- Interventions
- Dexpramipexole (dose 1), Dexpramipexole (dose 2)
- Drug
- Lead sponsor
- Knopp Biosciences
- Industry
- Eligibility
- 18 Years to 75 Years
- Enrollment
- 36 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2012
- U.S. locations
- 2
- States / cities
- Orlando, Florida • Brooklyn Center, Minnesota
- Conditions
- Refractory Lupus Nephritis, Refractory Systemic Sclerosis
- Interventions
- TAK-007, Chemotherapy Agents
- Biological · Drug
- Lead sponsor
- Takeda
- Industry
- Eligibility
- 18 Years to 75 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2030
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Rheumatoid Arthritis (RA), Systemic Lupus Erthematosus (SLE), Dermatomyositis (DM), Systemic Sclerosis (SSc)
- Interventions
- LMY-922
- Biological
- Lead sponsor
- Luminary Therapeutics
- Industry
- Eligibility
- 16 Years to 75 Years
- Enrollment
- 94 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease, Amyloidosis, Ankylosing Spondylitis, Arthritis, Alopecia Areata, Asthma, Atopic Dermatitis, Autism, Autoimmune Hepatitis, Behcet's Disease, Beta-Thalassemia, Cancer, Celiac Disease, Kidney Diseases, COPD, Crohn Disease, Cystic Fibrosis, Diabetes, Dravet Syndrome, DMD, Fibromyalgia, Graves Disease, Thyroid Diseases, Hepatitis, Hidradenitis Suppurativa, ITP, Leukemia, ALS, Lupus or SLE, Lymphoma, Multiple Sclerosis, Myasthenia Gravis, Heart Diseases, Parkinson Disease, Pemphigus Vulgaris, Cirrhosis, Psoriasis, Schizophrenia, Scleroderma, Sickle Cell Disease, Stroke, Ulcerative Colitis, Vasculitis, Vitiligo
- Interventions
- Specimen sample
- Diagnostic Test
- Lead sponsor
- Sanguine Biosciences
- Industry
- Eligibility
- 18 Years to 85 Years
- Enrollment
- 20,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Waltham, Massachusetts
- Conditions
- Metastatic Cancer, Congestive Heart Failure, Chronic Obstructive Pulmonary Disease, Parkinson Disease, Interstitial Lung Disease, Amyotrophic Lateral Sclerosis, End Stage Liver Disease, End Stage Renal Disease, Diabetes Complications
- Interventions
- Respecting Choices First Steps, Five Wishes Form
- Behavioral
- Lead sponsor
- Duke University
- Other
- Eligibility
- 65 Years and older
- Enrollment
- 790 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 4
- States / cities
- Birmingham, Alabama • Atlanta, Georgia • Columbia, South Carolina + 1 more