- ICH GCP
- US Clinical Trials Registry
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Clinical Trials on Disability in New Zealand
Total 146 results
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ACADIA Pharmaceuticals Inc.CompletedRett SyndromeUnited States
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Eisai Inc.PfizerTerminated
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Tetra Discovery PartnersRecruitingFragile X SyndromeUnited States
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Tetra Discovery PartnersEnrolling by invitationFragile X SyndromeUnited States
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Jazz PharmaceuticalsTerminatedRett Syndrome | RTTUnited States, Spain, Italy, United Kingdom
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedMyelodysplastic Syndromes | Leukemia | Myelodysplastic/Myeloproliferative NeoplasmsUnited States, Canada, Puerto Rico, Australia, Netherlands, New Zealand, Switzerland
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Jazz PharmaceuticalsGW PharmaceuticalsTerminatedRett Syndrome | RTTUnited States, Canada, Italy, Spain, United Kingdom, Australia
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingAcute Myeloid Leukemia | Down Syndrome | Myelodysplastic Syndrome | Myeloproliferative Neoplasm | Myeloid Leukemia Associated With Down SyndromeUnited States, Canada, Saudi Arabia, Puerto Rico, Australia, New Zealand
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Institute for Neurodegenerative DisordersCompletedParkinson Disease | Alzheimer Disease | Mild Cognitive Impairment | Fragile X Syndrome | Huntington Disease | Autistic Spectrum DisordersUnited States
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Novartis PharmaceuticalsCompletedFragile X SyndromeUnited States, Belgium, Australia, Israel, Switzerland, Germany, France, Canada, Denmark, Indonesia, Italy, Netherlands, Spain, Sweden, Turkey, United Kingdom
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Carelon ResearchNational Heart, Lung, and Blood Institute (NHLBI); National Institutes of Health...CompletedDown Syndrome | Congenital Heart DiseaseUnited States, Canada
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Neuren Pharmaceuticals LimitedCompleted
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Eisai Inc.PfizerCompleted
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National Hospital Organization Nagoya Medical CenterRecruitingAcute Lymphoblastic Leukemia | Down Syndrome | Childhood CancerTaiwan, Thailand, Hong Kong, Singapore, Malaysia, Japan
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National Cancer Institute (NCI)RecruitingDown Syndrome | B Acute Lymphoblastic Leukemia | B Lymphoblastic LymphomaUnited States, Canada, Puerto Rico, Australia, New Zealand
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Hoffmann-La RocheCompleted
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ACADIA Pharmaceuticals Inc.RecruitingHyperphagia in Prader-Willi SyndromeUnited States, Canada, United Kingdom
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University of MinnesotaNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedMucopolysaccharidosis Type I | Mucopolysaccharidosis Type II | Mucopolysaccharidosis Type VI | Mucopolysaccharidosis Type IV | Mucopolysaccharidosis Type VIIUnited States, Canada
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University of Southern CaliforniaNational Institute on Aging (NIA); Alzheimer's Therapeutic Research Institute; Alzheimer's Clinical Trials ConsortiumRecruitingDementia | Alzheimer Disease | Down SyndromeUnited States, Spain, United Kingdom
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Verinata Health, Inc.CompletedPregnancy | Down Syndrome | Edwards Syndrome | Patau Syndrome | Turners SyndromeUnited States
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Seaside Therapeutics, Inc.CompletedFragile X SyndromeUnited States
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Seaside Therapeutics, Inc.TerminatedFragile X SyndromeUnited States
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Seaside Therapeutics, Inc.Completed
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Millendo Therapeutics SASTerminatedHyperphagia | Prader-Willi SyndromeUnited States, Belgium, Spain, France, Italy, United Kingdom, Australia, Netherlands
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International Rett Syndrome FoundationBaylor College of Medicine; Children's Hospital of Philadelphia; University of... and other collaboratorsRecruitingNervous System Diseases | Neurologic Manifestations | Neurobehavioral Manifestations | Genetic Diseases, X-Linked | Intellectual Disability | Neurodevelopmental Disorders | Neurologic Disorder | Rett Syndrome | Genetic Disease | Rett Syndrome, AtypicalUnited States
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Natera, Inc.Children's Hospital of Philadelphia; Montefiore Medical Center; University of... and other collaboratorsCompletedPrader-Willi Syndrome | DiGeorge Syndrome | 22q11 Deletion Syndrome | Angelman Syndrome | Trisomy 21 | Trisomy 18 | Trisomy 13 | Monosomy X | Sex Chromosome Abnormalities | 1p36 Deletion Syndrome | Cri-du-Chat SyndromeUnited States, Australia, Ireland, Spain, Sweden, United Kingdom
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Sangamo TherapeuticsActive, not recruitingHemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis IIUnited States
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TakedaAvailableHunter SyndromeUnited States, Australia, Mexico, Spain, United Kingdom
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TakedaTakeda Development Center Americas, Inc.RecruitingHunter Syndrome | Mucopolysaccharidosis (MPS)United States
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Natera, Inc.Houston Perinatal Associates; Lyndhurst Clinical Research; Dr. Carpenter Maternal... and other collaboratorsCompletedTrisomy 21 | Trisomy 18 | Trisomy 13 | Sex Chromosome AbnormalitiesUnited States, India, Malaysia
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Natera, Inc.Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsTerminatedAneuploidy | Trisomy 21 | Trisomy 18 | Trisomy 13United States, Ireland, Canada, Italy, Korea, Republic of, Spain
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Hoffmann-La RocheCompletedFragile X SyndromeFrance, Spain, United States, Peru, Canada, United Kingdom, Chile, Argentina, Mexico, Sweden
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Sequenom, Inc.PRA Health SciencesCompletedPregnancy | Down Syndrome | Aneuploidy | Edwards SyndromeUnited States, France
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National Cancer Institute (NCI)SuspendedDown Syndrome | Recurrent B Acute Lymphoblastic LeukemiaUnited States, Canada, Puerto Rico, Australia
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University of ChicagoNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaboratorsCompletedKrabbe Disease | Mucopolysaccharidosis Type II (MPS II) | Mucopolysaccharidosis Type I (MPS I) | Mucopolysaccharidosis Type III (MPS III) | Mucopolysaccharidosis Type VI (MPS VI)United States
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LLS PedAL Initiative, LLCNational Cancer Institute (NCI); Children's Oncology GroupRecruitingAcute Myeloid Leukemia | Juvenile Myelomonocytic Leukemia | Acute Lymphoblastic Leukemia | Myelodysplastic Syndrome | Myeloid Leukemia Associated With Down Syndrome | Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic Syndrome Post Cytotoxic TherapyUnited States, Canada, Australia, Puerto Rico, New Zealand
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Children's Oncology GroupNational Cancer Institute (NCI)Active, not recruitingAcute Lymphoblastic Leukemia | Down Syndrome | Philadelphia Chromosome Positive | Childhood B Acute Lymphoblastic Leukemia With t(9;22)(q34.1;q11.2); BCR-ABL1 | Childhood B Acute Lymphoblastic Leukemia | Adult B Lymphoblastic Lymphoma | Ann Arbor Stage I B Lymphoblastic Lymphoma | Ann Arbor Stage... and other conditionsUnited States, Canada, Puerto Rico, Australia, New Zealand, Switzerland, Ireland
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Denali Therapeutics Inc.RecruitingMucopolysaccharidosis IIUnited States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Colombia, Brazil
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Progenity, Inc.CompletedDown Syndrome | Aneuploidy | DiGeorge Syndrome | Turner Syndrome | Klinefelter Syndrome | Chromosome Deletion | Edwards Syndrome | Patau SyndromeUnited States
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Children's Oncology GroupNational Cancer Institute (NCI)RecruitingDown Syndrome | Myeloid Leukemia Associated With Down Syndrome | B Acute Lymphoblastic Leukemia Associated With Down SyndromeUnited States
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Albert Einstein College of MedicineEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsEnrolling by invitationMucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditionsUnited States
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National Cancer Institute (NCI)Childrens Oncology Group (COG); LLS PedAL LLCRecruitingDown Syndrome | Recurrent Acute Myeloid Leukemia | Recurrent Myelodysplastic Syndrome | Refractory Acute Myeloid Leukemia | Refractory Myelodysplastic Syndrome | Recurrent Mixed Phenotype Acute Leukemia | Refractory Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic...United States
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ShireCompletedHunter Syndrome | Mucopolysaccharidosis II (MPS II)United States, France, United Kingdom, Germany, Sweden, Spain, Brazil, Canada, Italy, Romania
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New York Medical CollegeCompletedMucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditionsUnited States
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Children's Oncology GroupNational Cancer Institute (NCI)CompletedSecondary Acute Myeloid Leukemia | Childhood Acute Erythroleukemia (M6) | Childhood Acute Megakaryocytic Leukemia (M7) | Childhood Acute Monoblastic Leukemia (M5a) | Childhood Acute Monocytic Leukemia (M5b) | Childhood Acute Myeloblastic Leukemia With Maturation (M2) | Childhood Acute Myeloblastic... and other conditionsUnited States, Canada, Australia, Puerto Rico
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Duke UniversityEunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaboratorsRecruitingHeart Failure | Pain | Pneumonia | Hypertension | Edema | Hemophilia | Insomnia | Pulmonary Arterial Hypertension | Coagulation Disorder | Asthma in Children | Down Syndrome | Attention Deficit Hyperactivity Disorder | Bronchopulmonary Dysplasia | Hypokalemia | Hyperphosphatemia | Primary Hyperaldosteronism | Adrenal Insufficiency and other conditionsUnited States, Canada