Clinical Trials on Program Development
Total 5498 results
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Stanford UniversityThe Marfan FoundationCompletedChronic Pain | Marfan Syndrome | Vascular Ehlers-Danlos Syndrome | Loeys-Dietz SyndromeUnited States
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Bellicum PharmaceuticalsActive, not recruitingStudy of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell TransplantLymphoma, Non-Hodgkin | Myelodysplastic Syndromes | Acute Lymphoblastic Leukemia | Anemia, Aplastic | Primary Immune Deficiency Disorder | Cytopenia | Osteopetrosis | Leukemia, Acute Myeloid (AML), Child | Hemoglobinopathy in ChildrenUnited States
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Boston Children's HospitalCompletedSmith-Lemli-Opitz SyndromeUnited States
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Forbes Porter, M.D.Eunice Kennedy Shriver National Institute of Child Health and Human Development...Completed
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Medical University of GrazCompletedSkin Cancer | Basal Cell Nevus Syndrome | Xeroderma PigmentosumAustria
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Rolfs Consulting und Verwaltungs-GmbH (RCV)Rhythm Pharmaceuticals, Inc.Active, not recruitingCognitive Impairment | Hyperphagia | Obesity, Childhood | Bardet-Biedl Syndrome | Polydactyly | Retinopathy | Syndactyly | POMC DeficiencyGermany
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IRCCS Policlinico S. DonatoFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, ItalyCompletedDepression | Fatigue | Insomnia | Rare DiseasesItaly
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Universidad Nacional Autonoma de MexicoInstituto Mexicano del Seguro Social; Instituto Nacional de Ciencias Medicas...RecruitingPrimary Hyperparathyroidism | Lynch Syndrome | Tuberous Sclerosis | Gastrointestinal Stromal Tumors | Neurofibromatosis 1 | Medullary Thyroid Cancer | Von Hippel-Lindau Disease | Pheochromocytoma | Neuroendocrine Neoplasm | Paraganglioma | Pituitary Tumor | Multiple Endocrine Neoplasia Type 2 | Multiple Endocrine... and other conditionsMexico
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Kidney Cancer Research BureauRecruitingNon-small Cell Lung Cancer Metastatic | Prostate Cancer Metastatic | Renal Cell Carcinoma Metastatic | Breast Cancer Metastatic | Head & Neck CancerRussia
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Columbia UniversityCompletedSevere Combined Immunodeficiency | Fanconi Anemia | Bone Marrow Failure | OsteopetrosisUnited States
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University of NebraskaUniversity of Colorado, Denver; University of Pittsburgh; Children's Hospital...Completed
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Golden Jubilee National HospitalUniversity of Glasgow; Royal Infirmary of Edinburgh; Aberdeen Royal Infirmary; Network...Active, not recruitingAortic Valve Insufficiency | Marfan Syndrome | Bicuspid Aortic Valve | Aortic Diseases | Aortic Dissection | Aortic Aneurysm, Thoracic | Ehlers-Danlos Syndrome | Aortic Ectasia | Loeys-Dietz SyndromeUnited Kingdom
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University of CalgaryAlberta Health services; University of AlbertaCompletedStroke | Epilepsy | Autoimmune Diseases | Endocrine System Diseases | Diabetes | Hematologic Diseases | Genetic Diseases, Inborn | Traumatic Brain Injury | Autism Spectrum Disorder | Respiratory Disease | Cardiac Disease | Metabolic Disease | Renal Disease | Neuro-Degenerative Disease | Fetal Alcohol Spectrum Disorders | Gastro-Intestinal DisorderCanada
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Andres SchanzerRecruitingMarfan Syndrome | Ehlers-Danlos Syndrome | Aortic Arch Aneurysm | Complex Aortic Aneurysms | Juxtarenal Aneurysms | Loeys-Dietz Syndrome | Thoracoabdominal Aneurysms | Pararenal AneurysmsUnited States
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Christopher DvorakNo longer availableAcute Myeloid Leukemia | Leukocyte Disorders | Acute Lymphoblastic Leukemia | Chronic Myeloid Leukemia | Myelodysplastic Syndrome | Cytopenias | Immune Deficiency | Lymphomas | Bone Marrow Failure | Osteopetrosis | Hemoglobinopathy | Anemia Due to Intrinsic Red Cell AbnormalityUnited States
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St. Jude Children's Research HospitalRecruitingLiposarcoma | Angiosarcoma | Myxofibrosarcoma | Angiomatoid Fibrous Histiocytoma | Myoepithelioma | Atypical Fibroxanthoma | Adipocytic Neoplasm | Fibrosarcoma NOS | Osteosarcoma, ExtraskeletalUnited States
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Children's Hospital of PhiladelphiaIlera HealthcareTerminatedEpilepsy | Multiple Sclerosis | Glaucoma | Cancer | Parkinson Disease | HIV/AIDS | ALS | Autism Spectrum Disorder | Sickle Cell Disease | Spasticity, Muscle | Huntington Disease | Opioid Use | Neuropathy | Terminal Illness | Inflammatory Bowel Disease (IBD) | Intractable Pain | Ptsd | Dyskinetic SyndromeUnited States
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Universitaire Ziekenhuizen KU LeuvenKU LeuvenActive, not recruitingObesity | Healthy Volunteers | Prader-Willi Syndrome | Muscle Hypotonia | Hypotonia-Cystinuria Syndrome | Dwarfism, Growth Hormone DeficiencyBelgium
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Zimmer BiometCompletedTumor | Neuroma | Mandibular Fractures | Treacher Collins Syndrome | Facial Fracture | Fibrous Dysplasia | Hemifacial Microsomia | Miller Syndrome | Osteoma of Mandibular Condyle | Cleft Face | Nager SyndromeArgentina
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Boston Children's HospitalLymphatic Malformation InstituteRecruitingLymphangiomatosis | Kaposiform Lymphangiomatosis | Lymphatic Malformation | Generalized Lymphatic Anomaly (GLA) | Central Conducting Lymphatic Anomaly | CLOVES Syndrome | Gorham-Stout Disease ("Disappearing Bone Disease") | Blue Rubber Bleb Nevus Syndrome | Kaposiform Hemangioendothelioma/Tufted Angioma and other conditionsUnited States
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Bellicum PharmaceuticalsTerminatedLymphoma, Non-Hodgkin | Acute Lymphoblastic Leukemia | Primary Immunodeficiency | Anemia, Sickle Cell | Hemoglobinopathies | Fanconi Anemia | Anemia, Aplastic | Myelodysplastic Syndrome | Thalassemia | Cytopenia | Diamond Blackfan Anemia | Osteopetrosis | Leukemia, Acute Myeloid (AML), ChildUnited Kingdom, Italy
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UK Kidney AssociationRecruitingVasculitis | AL Amyloidosis | Tuberous Sclerosis | Fabry Disease | Cystinuria | Focal Segmental Glomerulosclerosis | IgA Nephropathy | Bartter Syndrome | Pure Red Cell Aplasia | Membranous Nephropathy | Atypical Hemolytic Uremic Syndrome | Autosomal Dominant Polycystic Kidney Disease | Cystinosis | Nephronophthisis | BK Nephropathy and other conditionsUnited Kingdom
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St. Jude Children's Research HospitalRecruitingPancreatic Cancer | Hodgkin Lymphoma | Lynch Syndrome | Tuberous Sclerosis | Fanconi Anemia | AML | Non Hodgkin Lymphoma | Familial Adenomatous Polyposis | Acute Leukemia | Nevoid Basal Cell Carcinoma Syndrome | Neurofibromatosis Type 1 | Neuroblastoma | Retinoblastoma | MDS | Rhabdomyosarcoma | Von Hippel-Lindau Disease | Adrenocortical Carcinoma and other conditionsUnited States
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Odense University HospitalOdense Patient Data Explorative NetworkRecruitingToe Joint Deformity | Clubfoot | Flatfoot | Trigger Finger | Syndactyly | Hand Deformities, Congenital | Tarsal Coalition | Congenital Talipes Equinovarus | Valgus Foot Deformity | Club Foot | Polydactyly Toe | Thumb Hypoplasia | Polydactyly; Fingers | Osteomyelitis of Hindfoot | Osteomyelitis of Midfoot | Congenital Talipes... and other conditionsDenmark
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University of Colorado, DenverRecruitingHearing Loss | Smith-Lemli-Opitz Syndrome | Cone-Rod DystrophyUnited States
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Incyte CorporationTerminatedNasopharyngeal Cancer | Esophageal Cancer | Ovarian Cancer | Hepatocellular Carcinoma (HCC) | Cholangiocarcinoma | Solid TumorsUnited States, Belgium
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Cure CMDRecruitingEmery-Dreifuss Muscular Dystrophy | Congenital Myasthenic Syndrome | Limb-Girdle Muscular Dystrophy | Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency | Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) and other conditionsUnited States
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MD Stem CellsEnrolling by invitationAlzheimer Disease | Traumatic Brain Injury | Autism Spectrum Disorder | Lewy Body Disease | Vascular Dementia | Autism | Dementia, Mixed | Alzheimer Dementia | Dementia, Multi-Infarct | Cadasil | Parkinson-Dementia Syndrome | Chronic Traumatic Encephalopathy | Autistic Behavior | Lewy Body Dementia With Behavioral... and other conditionsUnited States, United Arab Emirates
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Children's Hospital of PhiladelphiaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)RecruitingNephronophthisis | Joubert Syndrome | Bardet Biedl Syndrome | Autosomal Recessive Polycystic Kidney Disease | Congenital Hepatic Fibrosis | Hepato/Renal Fibrocystic Disease | Meckel-Gruber Syndrome | Caroli Syndrome | Oro-Facial-Digital Syndrome Type I | Glomerulocystic Kidney DiseaseUnited States
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McGill University Health Centre/Research Institute...RecruitingPeroxisome Biogenesis Disorder | Zellweger Spectrum Disorder | RCDP - Rhizomelic Chondrodysplasia Punctata | D-Bifunctional Protein Deficiency | Alpha-Methylacyl-CoA Racemase Deficiency | Peroxisomal Acyl-CoA Oxidase Deficiency | Peroxisomal Acyl-CoA Oxidase 2 Deficiency | ATP Binding Cassette Subfamily... and other conditionsCanada
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National Human Genome Research Institute (NHGRI)RecruitingMetabolic Disease | Purine-Pyrimidine Metabolism | AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2 | UNG, OMIM *191525, Hyper-IgM Syndrome 5 | NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency | UMPS, OMIM *613891, Orotic Aciduria | DHODH, OMIM *126064,... and other conditionsUnited States
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Hospital do CoracaoUniversidade Federal do Rio de Janeiro; Instituto Nacional de Cardiologia de...RecruitingCardiomyopathy, Dilated | Marfan Syndrome | Familial Hypercholesterolemia | Cardiomyopathy, Hypertrophic | Sudden Cardiac Death | Brugada Syndrome | Long QT Syndrome | Arrhythmogenic Right Ventricular Dysplasia | Catecholaminergic Polymorphic Ventricular Tachycardia | Loeys-Dietz Syndrome | Ehlers-Danlos... and other conditionsBrazil
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Dana-Farber Cancer InstituteRecruitingLung Cancer | Cirrhosis | Barrett Esophagus | Hematologic Malignancy | Non-Alcoholic Fatty Liver Disease | Cancer Risk | Vulvar Intraepithelial Neoplasia | Childhood Cancer Survivors | Lung; Node | Non Alcoholic Steatohepatitis | Osteochondroma | Cancer Predisposition Syndrome | Hereditary Cancer Prediction | Adult... and other conditionsUnited States
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Children's Hospital Los AngelesCompletedGranuloma | Anemia | Thrombocytopenia | Neutropenia | Sickle Cell Disease | Thalassemia | Niemann-Pick Disease | Wiskott-Aldrich Syndrome | Osteopetrosis | Fucosidosis | Chediak Higashi Syndrome | Hurler DiseaseUnited States
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University of PittsburghRecruitingLysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditionsUnited States
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University of AarhusCompletedHeart Failure | Stroke | Epilepsy | Pain | Multiple Sclerosis | Hypertension | Diabetes Mellitus | Cancer | Chronic Kidney Diseases | Schizophrenia | Parkinson Disease | Migraine | Inflammatory Bowel Diseases | Anemia | HIV/AIDS | Atrial Fibrillation | Dyslipidemias | Allergy | Osteoporosis | Ischemic Heart Disease | Hearing Disorders | Substance... and other conditions
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RTI InternationalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationPrimary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditionsUnited States
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Children's National Research InstituteEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationCongenital Diaphragmatic Hernia | Severe Hemophilia A | Noonan Syndrome | Short Bowel Syndrome | Trisomy 13 Syndrome | Cockayne Syndrome | CHARGE Syndrome | Beta-Propeller Protein-Associated Neurodegeneration | Early Infantile Epileptic Encephalopathy | Asparagine Synthetase Deficiency | FOXG1 Syndrome | KBG... and other conditionsUnited States
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Masonic Cancer Center, University of MinnesotaActive, not recruitingHurler Syndrome | Sphingolipidoses | Peroxisomal Disorders | Metachromatic Leukodystrophy | Alpha-Mannosidosis | Hunter Syndrome | Mucopolysaccharidosis Disorders | Maroteaux Lamy Syndrome | Sly Syndrome | Fucosidosis | Aspartylglucosaminuria | Glycoprotein Metabolic Disorders | Recessive Leukodystrophies | Globoid... and other conditionsUnited States
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Hospital Italiano de Buenos AiresRecruitingDemyelinating Diseases | Congenital Adrenal Hyperplasia | Inflammatory Bowel Diseases | Amyloidosis | Pulmonary Hypertension | Prader-Willi Syndrome | Immunoglobulin G4-Related Disease | Wilson Disease | Rare Diseases | Sarcoidosis | Hypertrophic Cardiomyopathy | Cushing Syndrome | Von Hippel-Lindau Disease | Pheo... and other conditionsArgentina
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Daniel BenjaminEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsCompletedHeart Failure | Epilepsy | Pain | Pneumonia | Sepsis | Inflammation | Hypertension | Chronic Kidney Diseases | Hemophilia | Schizophrenia | Central Nervous System Infections | Nosocomial Pneumonia | Insomnia | Anxiety | Bipolar Disorder | Neutropenia | Staphylococcal Infections | Bradycardia | Seizures | Urinary Tract Infections | Pulmonary... and other conditionsUnited States, Canada, United Kingdom, Israel, Singapore
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Paul SzabolcsRecruitingCongenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary AnemiasUnited States
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Paul SzabolcsRecruitingCongenital Bone Marrow Failure Syndromes | Inflammatory Conditions | Primary Immunodeficiency (PID) | Inherited Metabolic Disorders (IMD) | Hereditary Anemias | Systemic Juvenile Idiopathic Arthritis (sJIA) | Juvenile Rheumatoid Arthritis (JRA)United States
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ProgenaBiomeSuspendedDepression | Multiple Sclerosis | Diabetes | Cancer | Parkinson Disease | Constipation | Migraine | Amyotrophic Lateral Sclerosis | Psoriasis | Anxiety | Alzheimer Disease | Bipolar and Related Disorders | Celiac Disease | Eczema | Autism Spectrum Disorder | Mesothelioma | Colon Cancer | Amyotrophic Lateral Sclerosis (ALS) | Gout | Lyme Arthritis and other conditionsUnited States
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Centre Hospitalier Universitaire de LiegeSanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaboratorsCompletedCongenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditionsBelgium
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Children's Hospital of PhiladelphiaEli Lilly and Company; University of Pennsylvania; Takeda; National Institute of... and other collaboratorsRecruitingMucopolysaccharidoses | Leukoencephalopathies | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2... and other conditionsUnited States
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Children's Hospital of PhiladelphiaIllumina, Inc.CompletedMucopolysaccharidoses | Leukodystrophy | Adrenoleukodystrophy | Adrenomyeloneuropathy | X-linked Adrenoleukodystrophy | Gangliosidoses | Metachromatic Leukodystrophy | Krabbe Disease | Refsum Disease | Cadasil | Sjogren-Larsson Syndrome | Allan-Herndon-Dudley Syndrome | White Matter Disease | GM2 Gangliosidosis | Zellweger... and other conditionsUnited States
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Sanford HealthNational Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaboratorsRecruitingMitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Moyamoya Disease | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar... and other conditionsUnited States, Australia