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Showing 1–24 of 29 matching trials from the live ClinicalTrials.gov search.
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Active, not recruiting No phase listed Observational Accepts healthy volunteers

Early Check: Expanded Screening in Newborns

NCT03655223
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
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
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
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
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
Completed Phase 2 Interventional Results available

RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS

NCT00126672
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
Enrolling by invitation No phase listed Observational Accepts healthy volunteers

Quest to Analyze One Thousand Humans Meditating

NCT06583395
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
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
Suspended No phase listed Observational Accepts healthy volunteers

Genomic Resources for Enhancing Available Therapies (GREAT1.0) Study

NCT04306939
Conditions
Chronic Pancreatitis, Inflammatory Bowel Diseases, Hepatitis, NASH - Nonalcoholic Steatohepatitis, Acute Pancreatitis, Rheumatoid Arthritis, Diabetes Mellitus, Dyslipidemias, Multiple Sclerosis, Irritable Bowel Syndrome, Chronic Pain, Chronic Disease, Chronic Kidney Diseases, Crohn Disease, Celiac Disease, Biliary Cirrhosis, Bile Acid Synthesis Defect, Gastritis, Cholecystitis, Cholelithiases, IPMN, Cyst Pancreas, Cystic Fibrosis, Pancreatic Exocrine Insufficiency, Diarrhea Chronic, Constipation - Functional, Constipation Chronic Idiopathic
Interventions
venipuncture, Questionnaires, Additional Sample Collections
Other · Behavioral
Lead sponsor
University of Pittsburgh
Other
Eligibility
12 Years and older
Enrollment
120,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2026
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
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
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
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
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
Conditions
Multiple Organ Failure, End Stage Cardiac Failure, End Stage Chronic Obstructive Airways Disease, Chronic Kidney Disease Stage 5, Hepatic Encephalopathy, Sepsis, Dementia, Multiple Sclerosis, Parkinson's Disease, In-Hospital Cardiac Arrest, Solid Organ Cancer
Interventions
Early order of palliative care consultation
Other
Lead sponsor
Wayne State University
Other
Eligibility
65 Years and older
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2016
U.S. locations
2
States / cities
Detroit, Michigan
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
Systemic Lupus Erythematosus (With and Without Nephritis), Idiopathic Inflammatory Myopathy, Systemic Sclerosis
Interventions
ALLO-329, Cyclophosphamide, Fludarabine
Genetic · Drug
Lead sponsor
Allogene Therapeutics
Industry
Eligibility
18 Years to 69 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
13
States / cities
Phoenix, Arizona • Loma Linda, California • Aurora, Colorado + 10 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
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
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
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
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
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
Conditions
Lupus Nephritis, Autoimmune Diseases, Systemic Sclerosis (SSc), Systemic Lupus Erythematosus (SLE), ANCA-Associated Vasculitis (AAV), Idiopathic Inflammatory Myopathies, Stiff Person Syndrome
Interventions
ADI-001, Fludarabine, Cyclophosphamide
Drug
Lead sponsor
Adicet Therapeutics
Industry
Eligibility
18 Years to 80 Years
Enrollment
180 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
2
States / cities
Redwood City, California • Buffalo, New York