Search clinical trials for: Hereditary study for PH
Total 422225 results
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Peking Union Medical College HospitalRecruitingCryopyrin-Associated Periodic SyndromesChina
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National Cancer Institute (NCI)RecruitingNeurofibromatosis Type 1 | Low Grade Glioma | Visual Pathway GliomaUnited States, Canada, Puerto Rico
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Masonic Cancer Center, University of MinnesotaRecruitingCerebral Adrenoleukodystrophy | Adrenoleukodystrophy | ALD (Adrenoleukodystrophy)United 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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National Cancer Institute (NCI)Children's Oncology GroupRecruitingMalignant Solid Neoplasm | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ependymoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Glioma | Recurrent Malignant Solid Neoplasm | Recurrent... and other conditionsUnited States, Puerto Rico, Australia
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University Hospital TuebingenRecruitingHereditary Spastic Paraplegia | Hereditary, Spastic Paraplegia, Autosomal DominantGermany
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National Cancer Institute (NCI)RecruitingMalignant Glioma | Rhabdoid Tumor | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ependymoma | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Solid Neoplasm | Recurrent... and other conditionsUnited States, Puerto Rico, Australia, Canada
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Massachusetts Eye and Ear InfirmaryMassachusetts General Hospital; United States Department of DefenseRecruitingNeurofibromatosis 2 | Vestibular Schwannoma | Acoustic NeuromaUnited States
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Boston Children's HospitalEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruitingAutism | ASD | Macrocephaly | PTEN | PTEN Hamartoma Tumor SyndromeUnited States
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AstraZenecaRecruitingAdv Solid Malig - H&N SCC, ATM Pro / Def NSCLC, Gastric, Breast and Ovarian CancerSpain, United States, Belgium, United Kingdom, France, Hungary, Canada, Korea, Republic of, Australia
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Massachusetts General HospitalHarvard Medical School (HMS and HSDM)RecruitingKeratoconus | Ectasia | Fuchs' Endothelial Dystrophy | CrosslinkingUnited States
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Johns Hopkins UniversityNational Cancer Institute (NCI); National Institutes of Health (NIH); ChiRhoClin...RecruitingLynch Syndrome | Pancreas Cancer | Gene Mutation | Peutz-Jeghers Syndrome (PJS) | Germline Mutation CarrierUnited 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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CHDI Foundation, Inc.RecruitingHuntington's DiseaseUnited States, Canada, Belgium, Spain, Australia, Germany, France, Netherlands, United Kingdom, New Zealand, Argentina, Austria, Chile, Colombia, Denmark, Ireland, Italy, Poland, Portugal, Switzerland
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National Institute of Neurological Disorders and...RecruitingMuscular Dystrophies | Inherited Neuromuscular Conditions | Inherited Neuropathies | Muscle Myopathies | Hereditary Spastic ParaplegiasUnited States
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Gynecologic Oncology GroupNational Cancer Institute (NCI)Not yet recruitingFallopian Tube Carcinoma | Ovarian Carcinoma | Hereditary Breast and Ovarian Cancer Syndrome | Primary Peritoneal CarcinomaUnited States
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National Eye Institute (NEI)RecruitingRetinal Degeneration | Retinitis Pigmentosa | Retinal Disease | AMDUnited States
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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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Children's Oncology GroupNational Cancer Institute (NCI)RecruitingRenal Cell Carcinoma | Wilms Tumor | Clear Cell Sarcoma of the Kidney | Papillary Renal Cell Carcinoma | Kidney Medullary Carcinoma | Diffuse Hyperplastic Perilobar Nephroblastomatosis | Rhabdoid Tumor of the Kidney | Congenital Mesoblastic Nephroma | Renal Cell Carcinoma Associated With Xp11.2 Translocations... and other conditionsUnited States, Canada, Puerto Rico, Australia, New Zealand, Israel, Switzerland
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National Institute of Neurological Disorders and...Recruiting
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National Human Genome Research Institute (NHGRI)Recruiting
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National Cancer Institute (NCI)CompletedNeurofibromatosis Type 1 | Legius SyndromeUnited States
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Horizon Therapeutics USA, Inc.CompletedThyroid Eye DiseaseUnited States
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National Institute on Deafness and Other Communication...CompletedSensorineural Hearing Loss | Vestibular Disease | Hearing DisorderUnited States, Nigeria
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Horizon Therapeutics USA, Inc.CompletedThyroid Eye Disease | Chronic (Inactive) Thyroid Eye DiseaseUnited States
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M.D. Anderson Cancer CenterNational Cancer Institute (NCI)Active, not recruitingCastleman Disease | Lymphangioleiomyomatosis | Digestive System Carcinoma | Malignant Neoplasm | Recurrent Breast Carcinoma | Recurrent Thyroid Gland Carcinoma | Metastatic Urothelial Carcinoma | Malignant Female Reproductive System Neoplasm | Neurofibromatosis Type 2 | Recurrent Adult Soft Tissue Sarcoma and other conditionsUnited States
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National Cancer Institute (NCI)Active, not recruitingRecurrent Childhood Ependymoma | Advanced Malignant Solid Neoplasm | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Glioma | Recurrent Medulloblastoma | Recurrent Neuroblastoma | Recurrent Non-Hodgkin... and other conditionsUnited States, Puerto Rico
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Janssen Research & Development, LLCActive, not recruitingNeoplasms | Myelodysplastic Syndromes | Non-Hodgkin Lymphoma | Solid Tumor, AdultUnited States, Spain, Israel, Germany, Canada
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Vigil Neuroscience, Inc.Active, not recruitingALSPUnited States, France, Germany, Netherlands, United Kingdom
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid PolyneuropathiesUnited States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom
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Tufts Medical CenterNational Kidney FoundationActive, not recruitingChronic Kidney Disease | Dialysis | Kidney TransplantUnited States
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Alnylam PharmaceuticalsCompletedAcute Hepatic Porphyria | Acute Intermittent Porphyria | Porphyria, Acute Intermittent | Acute Porphyria | Hereditary Coproporphyria (HCP) | Variegate Porphyria (VP) | ALA Dehydratase Deficient Porphyria (ADP)United States, Spain, United Kingdom, Korea, Republic of, Australia, Bulgaria, Canada, Denmark, Finland, France, Germany, Italy, Japan, Mexico, Netherlands, Poland, Sweden, Taiwan
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Sanguine BiosciencesCompletedDepression | Melanoma | Epilepsy | Lymphoma | Multiple Sclerosis | Kidney Cancer | Cervical Cancer | Diabetes Mellitus | Leukemia | Breast Cancer | Chronic Kidney Diseases | Chronic Obstructive Pulmonary Disease | Parkinson's Disease | Systemic Lupus Erythematosus | Multiple Myeloma | Hepatitis B | Insomnia | Colorectal Cancer | HIV/AID... and other conditionsUnited States
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Dompé Farmaceutici S.p.ACompleted
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National Cancer Institute (NCI)Active, not recruitingSoft Tissue Sarcoma | Renal Cell Carcinoma | Hepatocellular Carcinoma | Osteosarcoma | Ewing Sarcoma | Recurrent Renal Cell Carcinoma | Refractory Malignant Solid Neoplasm | Rhabdomyosarcoma | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Malignant Solid Neoplasm | Recurrent Osteosarcoma | Recurrent... and other conditionsUnited States
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St. Jude Children's Research HospitalActive, not recruitingRetinoblastomaUnited States
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Spark Therapeutics, Inc.Children's Hospital of Philadelphia; University of IowaActive, not recruitingLeber Congenital Amaurosis | Inherited Retinal Dystrophy Due to RPE65 MutationsUnited States
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Assistance Publique - Hôpitaux de ParisCompletedColchicine Resistance | Mediterranean FeverFrance
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AmgenActive, not recruitingThyroid Eye DiseaseUnited States, Spain, Germany, Italy, France, United Kingdom
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National Cancer Institute (NCI)Active, not recruitingRecurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Advanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Hepatoblastoma | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Glioma | Recurrent Malignant Solid Neoplasm | Recurrent Neuroblastoma and other conditionsUnited States, Puerto Rico
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Memorial Sloan Kettering Cancer CenterCompletedSarcoma | Small Intestine Cancer | Ovarian Cancer | Lung Cancer | Brain and Central Nervous System Tumors | Metastatic Cancer | Neuroblastoma | Intraocular Melanoma | Melanoma (Skin) | RetinoblastomaUnited States
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Ashley ShoemakerEnrolling by invitationPseudohypoparathyroidism Type 1a | Albright Hereditary Osteodystrophy | PseudohypoparathyroidismUnited States
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National Cancer Institute (NCI)Active, not recruitingAdvanced Malignant Solid Neoplasm | Refractory Malignant Solid Neoplasm | Recurrent Ewing Sarcoma | Recurrent Hepatoblastoma | Recurrent Histiocytic and Dendritic Cell Neoplasm | Recurrent Langerhans Cell Histiocytosis | Recurrent Malignant Germ Cell Tumor | Recurrent Malignant Solid Neoplasm | Recurrent... and other conditionsUnited States, Puerto Rico
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Alnylam PharmaceuticalsActive, not recruitingTransthyretin Amyloidosis | Amyloidosis, HereditaryUnited States, Argentina, Australia, Belgium, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Greece, Italy, Japan, Korea, Republic of, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, United Kingdom
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Novartis PharmaceuticalsActive, not recruitingTuberous Sclerosis ComplexUnited States, Spain, Belgium, Taiwan, Turkey, Australia, Korea, Republic of, Thailand, France, Hungary, United Kingdom, Italy, Japan, Canada, Russian Federation, Poland, Mexico, Colombia
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City of Hope Medical CenterActive, not recruitingUnspecified Childhood Solid Tumor, Protocol Specific | Solid Tumor | Ewing Sarcoma | Unspecified Adult Solid Tumor, Protocol Specific | Recurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Recurrent Neuroblastoma | Recurrent Adult Soft Tissue Sarcoma | Recurrent Adult Brain Tumor | Recurrent... and other conditionsUnited States
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Ionis Pharmaceuticals, Inc.CompletedHereditary Transthyretin-Mediated Amyloid PolyneuropathyUnited States, Spain, Taiwan, Italy, Germany, Argentina, Australia, Brazil, Canada, Cyprus, France, Greece, New Zealand, Portugal, Sweden, Turkey
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Children's Hospital Medical Center, CincinnatiActive, not recruitingNeurofibromatosis 1United States
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Reata, a wholly owned subsidiary of BiogenTerminatedChronic Kidney Diseases | Autosomal Dominant Polycystic Kidney | Alport SyndromeUnited States, France, Australia, Japan, Spain, Puerto Rico
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MeiraGTx UK II LtdSyne Qua Non Limited; EMAS PharmaActive, not recruitingAchromatopsiaUnited Kingdom, United States