- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on Genetic Diseases, X-Linked in United States
Total 7967 results
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Ascendis Pharma Endocrinology Division A/SActive, not recruitingTurner SyndromeUnited States
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Memorial Sloan Kettering Cancer CenterRecruitingSolid Tumor | Rhabdoid Tumor | Wilms Tumor | Nephroblastoma | Malignant Peripheral Nerve Sheath Tumors | MPNST | XPO1 Gene MutationUnited States
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Sarcoma Alliance for Research through CollaborationNational Cancer Institute (NCI)CompletedSarcoma | Neurofibromatosis Type 1United States
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Novartis PharmaceuticalsCompletedTuberous Sclerosis | Subependymal Giant Cell AstrocytomaUnited States, Belgium, Canada, Germany, Italy, Russian Federation, Netherlands, Poland, Australia, United Kingdom
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Alnylam PharmaceuticalsCompletedAmyloid Neuropathies | Amyloid Neuropathies, Familial | Amyloidosis, Hereditary | Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC) | Amyloidosis, Hereditary, Transthyretin-Related | Familial Transthyretin Cardiac AmyloidosisUnited States, France, Italy, Spain, Canada, Germany, Belgium, Sweden, United Kingdom
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Cardiff UniversityUniversity of CanterburyRecruitingCleft Lip and Palate | Parenting | Neurofibromatoses | Skin Condition | Alopecia | Craniofacial | Limb DifferenceUnited Kingdom
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Emalex Biosciences Inc.RecruitingTourette DisorderUnited States, Canada, Spain, Poland, Italy, Bulgaria, Denmark, France, Germany, Hungary, Serbia, Romania
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BioMarin PharmaceuticalICON plcCompletedMucopolysaccharidosis IV Type A | Morquio A Syndrome | MPS IVAUnited States, United Kingdom, Australia, Taiwan, Belgium, Malaysia, Austria, Canada, Portugal, France, Ireland, Czechia, Denmark, Italy, Netherlands, Poland, Puerto Rico
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ShireCompletedHereditary AngioedemaUnited States, Australia, Israel, Canada, Hungary, Romania, Russian Federation, South Africa, Ukraine
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Fred Hutchinson Cancer CenterNational Cancer Institute (NCI); National Heart, Lung, and Blood Institute...CompletedAdult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities | Adult Acute Myeloid Leukemia With Del(5q) | Adult Acute Myeloid Leukemia With Inv(16)(p13;q22) | Adult Acute Myeloid Leukemia With t(16;16)(p13;q22) | Adult Acute Myeloid Leukemia With t(8;21)(q22;q22) | Fanconi Anemia | Adult Acute... and other conditionsUnited States
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Cairo UniversityCompletedAzoospermia Anogenital Distance AMHEgypt
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University of LeicesterUnknownKidney CancerFrance, Netherlands, Sweden, Norway, Denmark, Portugal, Belgium, Croatia, Czech Republic, Greece, Italy, Poland, Slovenia, Spain, United Arab Emirates
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Novartis PharmaceuticalsCompletedMyelogenous Leukemia, ChronicSouth Africa, Italy, Belgium, Japan, Taiwan, Turkey, Canada, Germany, Spain, Egypt, United States, Singapore, Austria, France, Netherlands, Sweden, United Kingdom, Korea, Republic of, Hong Kong, Thailand, Russian Federation, Brazil, Hungar... and more
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Recursion Pharmaceuticals Inc.RecruitingNeurofibromatosis Type 2United States
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Gilead SciencesApproved for marketingCystic Fibrosis | Pseudomonas Aeruginosa Airway InfectionUnited States, Puerto Rico
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BioCryst PharmaceuticalsCompletedHereditary Angioedema (HAE)Austria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Romania, Switzerland, United Kingdom
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ShireJerini AGCompletedHereditary AngioedemaSpain, Argentina, Austria, Denmark, France, Germany, Israel, Italy, Switzerland, United Kingdom
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Pharvaris Netherlands B.V.RecruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States, Puerto Rico
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Mandos LLCTerminatedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Turkey, Switzerland, Germany, New Zealand, Singapore, France
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia AFrance, United States, Denmark, Netherlands, Sweden, Spain, Germany, Switzerland, Taiwan, Japan, United Kingdom, Croatia, Italy, Malaysia, Brazil, Australia, Hungary, Israel, Turkey, Puerto Rico, Norway, Korea, Republic of, Bulgaria, Russian...
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BioCryst PharmaceuticalsCompletedHereditary Angioedema | Prophylaxis | HAEAustria, Denmark, France, Germany, Hungary, Israel, Italy, North Macedonia, Poland, Switzerland, United Kingdom, United States, Australia, Hong Kong, Korea, Republic of, New Zealand, Serbia, Slovakia, South Africa, Spain
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European Huntington's Disease NetworkCompletedHuntington Disease | Huntington's DiseaseFinland, United Kingdom, Austria, Belgium, Czechia, Denmark, France, Germany, Italy, Netherlands, Norway, Poland, Portugal, Russian Federation, Spain, Sweden, Switzerland
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BioCryst PharmaceuticalsCompletedHereditary Angioedema (HAE)Canada, United Kingdom, Germany, Spain, Australia, Hungary, Italy, Austria, North Macedonia, Denmark, Switzerland
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Mandos LLCCompletedNiemann-Pick Disease, Type CUnited States, Australia, United Kingdom, Germany, France, New Zealand, Singapore, Spain, Turkey
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National Cancer Institute (NCI)CompletedUnspecified Childhood Solid Tumor, Protocol Specific | Recurrent Childhood Medulloblastoma | Recurrent Childhood Ependymoma | Recurrent Melanoma | Recurrent Neuroblastoma | Recurrent Osteosarcoma | Recurrent Childhood Rhabdomyosarcoma | Recurrent Colon Cancer | Recurrent Wilms Tumor and Other Childhood... and other conditionsUnited States
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City of Hope Medical CenterNational Cancer Institute (NCI)Active, not recruitingAnaplastic Large Cell Lymphoma | Recurrent Angioimmunoblastic T-Cell Lymphoma | Recurrent Mycosis Fungoides | Refractory Angioimmunoblastic T-Cell Lymphoma | Refractory Mature T-Cell and NK-Cell Non-Hodgkin Lymphoma | Refractory Mycosis Fungoides | Refractory Anaplastic Large Cell Lymphoma | Subcutaneous... and other conditionsUnited States
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Diurnal LimitedEnrolling by invitationCongenital Adrenal HyperplasiaUnited States, France, Japan
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Diurnal LimitedCompletedCongenital Adrenal HyperplasiaFrance, United States, Japan
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Merck Sharp & Dohme LLCRecruitingMelanoma | Lymphoma | Solid Tumor | Classical Hodgkin Lymphoma | Microsatellite-instability-high Solid TumorFrance, Germany, Italy, Netherlands, Sweden, United Kingdom, Brazil, Israel, Korea, Republic of, United States
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Simons SearchlightBoston Children's Hospital; Geisinger Clinic; Simons FoundationRecruitingSMARCA4 Gene Mutation | DDX3X | 16P11.2 Deletion Syndrome | 16p11.2 Duplications | 1Q21.1 Deletion | 1Q21.1 Microduplication Syndrome (Disorder) | ACTL6B | ADNP | AHDC1 | ANK2 | ANKRD11 | ARID1B | ASH1L | BCL11A | CHAMP1 | CHD2 | CHD8 | CSNK2A1 | CTBP1 | CTNNB1 Gene Mutation | CUL3 | DNMT3A | DSCAM | DYRK1A | FOXP1 | GRIN2A | GRIN2B | HIVEP2-Related Intellectual... and other conditionsUnited States
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Novartis PharmaceuticalsCompletedFirst or Second Line HER2-negative Breast Cancer | Metastatic Disease Without Bone MetastasisUnited States
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PerspectumRecruitingLiver Diseases | Cirrhosis | Primary Sclerosing Cholangitis | Autoimmune Hepatitis | Hemochromatosis | Non-Alcoholic Fatty Liver Disease | Non-Alcoholic Steatohepatitis | Primary Biliary Cirrhosis | Viral HepatitisNetherlands, United Kingdom
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Genzyme, a Sanofi CompanyApproved for marketingGlycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease (Late-Onset)United States
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Synspira, Inc.TerminatedLung Diseases | Cystic Fibrosis | Pulmonary Disease | Antibiotic Resistant Infection | Respiratory Tract Disease | Cystic Fibrosis Pulmonary Exacerbation | Lung Inflammation | Burkholderia Infections | Lung Infection | Multi-antibiotic Resistance | Pulmonary Inflammation | Lung Infection Pseudomonal | Cystic Fibrosis... and other conditionsUnited Kingdom
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Vico Therapeutics B. V.RecruitingHuntington Disease | Spinocerebellar Ataxia Type 3 | Spinocerebellar Ataxia Type 1Israel, Germany, United Kingdom, Poland, Denmark, Netherlands, France
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Grifols Therapeutics LLCTerminatedType 1 Diabetes MellitusUnited States
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Jonsson Comprehensive Cancer CenterNational Cancer Institute (NCI); Highlight TherapeuticsActive, not recruitingLeiomyosarcoma | Malignant Peripheral Nerve Sheath Tumor | Synovial Sarcoma | Undifferentiated Pleomorphic Sarcoma | Undifferentiated High Grade Pleomorphic Sarcoma of Bone | Myxofibrosarcoma | Stage II Soft Tissue Sarcoma of the Trunk and Extremities AJCC v8 | Stage III Soft Tissue Sarcoma of the... and other conditionsUnited States
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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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Royal Marsden NHS Foundation TrustRecruitingLynch Syndrome | Familial Adenomatous Polyposis | Hereditary Diffuse Gastric Cancer | Peutz-Jeghers Syndrome | Cowden Syndrome | Li Fraumeni Syndrome | MutYH-associated Polyposis | SMAD4 Gene MutationUnited Kingdom
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National Tuberous Sclerosis AssociationRecruitingTuberous Sclerosis | LymphangioleiomyomatosisUnited States, Canada
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Novartis PharmaceuticalsRecruitingMSIhi or dMMR Advanced Unresectable or Metastatic Solid Tumors, Including Colorectal CancersJapan, Italy, Spain, Singapore, Belgium, Germany, Korea, Republic of, Sweden, Taiwan, France, Israel, Norway, United States
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Marinus PharmaceuticalsActive, not recruitingTuberous Sclerosis ComplexUnited States, Spain, United Kingdom, Israel, Italy, Germany, France, Canada
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Avidity Biosciences, Inc.RecruitingPhase 1/2 Study of AOC 1020 in Adults With Facioscapulohumeral Muscular Dystrophy (FSHD) (FORTITUDE)Muscular Dystrophies | Muscular Dystrophy, Facioscapulohumeral | FSHD | Facio-Scapulo-Humeral Dystrophy | FMD | Facioscapulohumeral Muscular Dystrophy 1 | FSHD2 | FSHD1 | FMD2 | Fascioscapulohumeral Muscular Dystrophy | Fascioscapulohumeral Muscular Dystrophy Type 1 | Fascioscapulohumeral Muscular Dystrophy... and other conditionsUnited States, United Kingdom, Canada
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Edgar JaeggiLabatt Family Heart CentreRecruitingAtrial Flutter | Tachycardia, Supraventricular | Tachycardia, Atrioventricular Nodal Reentry | Tachycardia Atrial | Tachycardia, Reciprocating | Tachycardia, Paroxysmal | Fetal Hydrops | Tachycardia, Atrial EctopicUnited States, Hong Kong, Canada, Netherlands, Sweden, Australia, Brazil, Finland, France, Russian Federation, Spain, Czechia, Switzerland, United Kingdom
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Neonc Technologies, Inc.RecruitingMelanoma | Renal Cell Carcinoma | Cervical Cancer | Small Cell Lung Cancer | Gastric Cancer | Colorectal Cancer | Esophageal Cancer | Non-small Cell Lung Cancer | Head and Neck Squamous Cell Carcinoma | Esophageal Squamous Cell Carcinoma | Urothelial Carcinoma | Merkel Cell Carcinoma | Gastroesophageal Junction... and other conditionsUnited States
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Bristol-Myers SquibbWithdrawnAcute Lymphoblastic Leukemia | Chronic Myeloid LeukemiaNetherlands, Italy, United Kingdom, Russian Federation, Poland, Hungary
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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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Astellas Pharma Global Development, Inc.Active, not recruitingAcute Myeloid Leukemia (AML) | Acute Myeloid Leukemia With FMS-like Tyrosine Kinase (FLT3) MutationUnited States, Japan, Taiwan, Spain, Australia, Belgium, Canada, France, Germany, Italy, Korea, Republic of, Poland, United Kingdom
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Astellas Pharma Global Development, Inc.Approved for marketingAcute Myeloid Leukemia (AML) | FMS-like Tyrosine Kinase-3 (FLT3) MutationsUnited States, Canada, Japan
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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