- ICH GCP
- US Clinical Trials Registry
- Search trials
Clinical Trials on X-linked Severe Combined Immunodeficiency
Total 1627 results
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University of PittsburghRespirtech, Inc.CompletedDuchenne Muscular DystrophyUnited States
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American Thrombosis and Hemostasis NetworkTakeda; CSL Behring; OctapharmaCompletedHemophilia A | Hemophilia B | Hemophilia | Hemophilia A With Inhibitor | Haemophilia | Hemophilia B With Inhibitor | Haemophilia A Without Inhibitor | Haemophilia B Without InhibitorUnited States
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Ultragenyx Pharmaceutical IncTerminatedOrnithine Transcarbamylase DeficiencyUnited States
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National Institute of Allergy and Infectious Diseases...Rare Diseases Clinical Research Network; Primary Immune Deficiency Treatment...Enrolling by invitationGranulomatous Disease, ChronicUnited States, Canada
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National Institute of Allergy and Infectious Diseases...CompletedAtherosclerosis | Chronic Granulomatous Disease (CGD)United States
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Masonic Cancer Center, University of MinnesotaCompletedAplastic Anemia | Dyskeratosis CongenitaUnited States
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia BUnited States, Netherlands, Austria, Spain, Germany, Macedonia, The Former Yugoslav Republic of, Taiwan, United Kingdom, Italy, Malaysia, Thailand, France, Greece, Turkey, Japan, South Africa, Romania
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National Institute of Allergy and Infectious Diseases...CompletedChronic Granulomatous Disease | Communicable DiseaseUnited States
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia BUnited States, Sweden, Spain, Germany, United Kingdom, Denmark, France, Japan
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia B | HaemostasisUnited States
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American Thrombosis and Hemostasis NetworkLFB USA, Inc.RecruitingHemophilia A With Inhibitor | Hemophilia B With InhibitorUnited States
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American Thrombosis and Hemostasis NetworkGenentech, Inc.Active, not recruitingHemophilia A With Inhibitor | Hemophilia B With Inhibitor | Haemophilia A Without Inhibitor | Haemophilia B Without InhibitorUnited States
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia BUnited States, Switzerland, Germany
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Baylor College of MedicineActive, not recruitingFocal Dermal Hypoplasia (FDH) | Goltz SyndromeUnited States
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Stealth BioTherapeutics Inc.AvailableMitochondrial Diseases | Barth Syndrome
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Hacettepe UniversitySuleyman Demirel UniversityCompletedDuchenne Muscular Dystrophy | Ambulation Difficulty | Posture Disorders in Children | Ambulation Disorder, NeurologicTurkey
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Catalyst BiosciencesCompletedHemophilia A | Hemophilia B | Hemophilia A With Inhibitor | Hemophilia B With Inhibitor | Hemophilia A Without Inhibitor | Hemophilia B Without InhibitorBulgaria, Russian Federation
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Pixium Vision SACompletedRetinitis Pigmentosa | Choroideremia | Cone Rod DystrophyAustria, Spain, United Kingdom, France, Germany
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University of California, San FranciscoTerminatedMyeloid Malignancy | Metabolic Disease | Bone Marrow Failure Syndrome | Transfusion-dependent Red Blood Cell (RBC) Defect | Congenital Immunodeficiency | Severe Immune DysregulationUnited States
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University of California, San FranciscoNational Cancer Institute (NCI)CompletedMyelodysplastic Syndromes | Leukemia | Congenital Amegakaryocytic Thrombocytopenia | Severe Congenital NeutropeniaUnited States
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Institut National de la Santé Et de la Recherche...Not yet recruitingAutoimmune Diseases | Systemic Lupus Erythematosus | Primary Immunodeficiency | Autoimmune Hepatitis | Juvenile Idiopathic Arthritis | IBD | Autoimmune Rheumatologic Disease | Autoimmune Diabetes | APECED | IPEX | Hemophagocytic Lymphohistiocytoses | Autoimmune Lymphoproliferative Syndrome | Autoimmune Thrombocytopenia and other conditions
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Ionis Pharmaceuticals, Inc.RecruitingPelizaeus-Merzbacher DiseaseFrance, Australia, Germany, Netherlands
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Seoul National University HospitalRecruitingDuchenne Muscular Dystrophy | Scoliosis Neuromuscular | Lordosis LumbarKorea, Republic of
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National Cancer Institute (NCI)RecruitingCancer | HIV | Chronic Granulomatous Disease | Dock 8 DeficiencyUnited States
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Andrea GropmanChildren's National Research InstituteCompletedOrnithine Transcarbamylase Deficiency
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AryoGen Pharmed Co.CompletedFactor VII Deficiency | Hemophilia A With Inhibitor | Hemophilia B With Inhibitor | Glanzmann's ThrombastheniaIran, Islamic Republic of
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BUnited States, Spain, Taiwan, Turkey, Poland, Croatia, Italy, Malaysia, Brazil, United Kingdom, Hungary, Israel, Thailand, Japan, South Africa, Canada, France, Argentina
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BSpain, Italy, Greece, Germany, United Kingdom
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BUnited Kingdom
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Rigshospitalet, DenmarkCompletedBecker Muscular Dystrophy | Limb-Girdle Muscular Dystrophy Type 2IDenmark
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BFrance, Israel, Germany
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BMorocco, United Arab Emirates, Algeria, Oman, Saudi Arabia
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Sardocor Corp.Not yet recruitingDMD-Associated Dilated Cardiomyopathy
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National Institute for Mother and Child Health...Marie Curie Emergency Children's Hospital, Bucharest; Victor Gomoiu Children...RecruitingChronic Granulomatous DiseaseRomania
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Ultragenyx Pharmaceutical IncActive, not recruitingOrnithine Transcarbamylase (OTC) DeficiencyCanada, United States, France, Spain, United Kingdom
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Hôpital CochinAssociation Monégasque contre les MyopathiesUnknownDuchenne Muscular Dystrophy | Duchenne Muscular Dystrophy-Associated Dilated CardiomyopathyFrance
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BSpain, Germany, Japan
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BSpain
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BUnited Kingdom
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Emory UniversityChildren's Healthcare of AtlantaTerminatedSickle Cell Disease | Fanconi Anemia | Thalassemia | Severe Congenital Neutropenia | Wiskott-Aldrich Syndrome | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Glanzmann Thrombasthenia | Leukocyte Adhesion Deficiency | Dyskeratosis-congenita | Chediak-Higashi Syndrome | Chronic-granulomatous Disease | Schwachman-Diamond...United States
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World Federation of HemophiliaRecruitingHemophilia A | Hemophilia B | Von Willebrand DiseasesCanada
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia BSpain, United Kingdom, Australia, Taiwan, France, Malaysia, United States, Austria, Canada, Israel, Japan, Thailand, Algeria, Argentina
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Novo Nordisk A/SCompletedHealthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia BAustria, Sweden, Spain, Switzerland, United Kingdom, Germany, Malaysia, Denmark, South Africa, Thailand, Italy
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Novo Nordisk A/SCompletedHaemophilia A, Haemophilia B, ObesityUnited States
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia BUnited States, Netherlands, Germany, Macedonia, The Former Yugoslav Republic of, United Kingdom, Italy, Malaysia, Hungary, Thailand, France, Russian Federation, Turkey, Japan, South Africa, Canada
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Novo Nordisk A/SCompletedHealthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia BUnited Kingdom
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Novo Nordisk A/SCompletedHealthy | Congenital Bleeding Disorder | Haemophilia A | Haemophilia BUnited Kingdom
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BSpain
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Andrea GropmanNational Center for Research Resources (NCRR); Rare Diseases Clinical Research... and other collaboratorsCompletedBrain Diseases, Metabolic, Inborn | Ornithine Transcarbamylase Deficiency | Urea Cycle DisorderUnited States
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Novo Nordisk A/SCompletedCongenital Bleeding Disorder | Haemophilia A | Haemophilia BBelgium, Germany, France, United Kingdom, Italy, Hungary, Austria, Sweden, Iran, Islamic Republic of, Argentina, Ireland, Poland, Portugal, Slovakia