Inter Individual Variability in Initiation Pathway Activation and Regulation and Phenotypic Heterogeneity in Patients With Haemophilia A and B
Condition: Haemophilia
Interventions: Diagnostic Test: Thrombophilia screen; Diagnostic Test: Initiation pathway analysis
Sponsor: Royal Free Hospital NHS Foundation Trust
Recruiting
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Performance Evaluation of Von Willebrand:Collagen-Binding Assays to Diagnose Von Willebrand Factor Deficiency in Patients With Increased Risk of Bleeding
Condition: Von Willebrand Disease
Intervention: Other: Non interventional study
Sponsor: Nantes University Hospital
Recruiting
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UK - EHL Outcomes Registry
Conditions: Hemophilia A; Hemophilia B
Intervention:
Sponsor: Royal Free Hospital NHS Foundation Trust
Recruiting
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The Genetics and Functional Basis of Inherited Platelet, White Blood Cell, Red Blood Cell, and Blood Clotting Disorders.
Condition: Glanzmann Thrombasthenia
Intervention:
Sponsors: Rockefeller University; National Heart, Lung, and Blood Institute (NHLBI)
Recruiting
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Platelet Glycoproteins in Inherited Thrombocytopathy: Association With Aggregation Studies and Bleeding Severity
Conditions: Bleeding; Platelet Dysfunction
Intervention: Diagnostic Test: Flowcytometry analysis of platelets surface receptors
Sponsor: Assiut University
Recruiting
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A Research Study of How a New Medicine NNC0365-3769 (Mim8) Works in the Body of Healthy People and Patients With Bleeding Disorder
Conditions: Healthy Volunteers; Haemophilia A With or Without Inhibitors
Interventions: Drug: NNC0365-3769 A (Mim8); Drug: Placebo (Mim8)
Sponsor: Novo Nordisk A/S
Recruiting
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REAL-LIFE DATA OF CONSTITUTIONAL VON WILLEBRAND DISEASE IN WESTERN FRANCE (HOPSCOTcH-WILL)
Condition: Von Willebrand Diseases
Intervention: Other: VWD treatments
Sponsors: Nantes University Hospital; CHU de Brest; University Hospital, Angers; CH Le Mans; Rennes University Hospital
Not yet recruiting
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AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Conditions: Hemophilia B; Blood Coagulation Disorders, Inherited; Blood Coagulation Disorder; Haematologic Disease
Intervention:
Sponsor: Freeline Therapeutics
Not yet recruiting
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Real World Study of Deep Venous Thrombosis. Analysis of Prospective Cases in University Hospitals of São Paulo
Condition: Registry
Intervention: Other: deep venous thrombosis
Sponsors: Fundação Faculdade Regional de Medicina de São José do Rio Preto; University of Ribeirao Preto
Recruiting
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Cerebral Hemorrhage Risk in Hereditary Hemorrhagic Telangiectasia
Condition: Hereditary Hemorrhagic Telangiectasia
Intervention:
Sponsors: St. Michael's Hospital, Toronto; National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting
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Evaluation of Patients With Immune Function Abnormalities
Conditions: Chronic Granulomatous Disease (CGD); X-Linked Severe Combined Immune Deficiency (XSCID); Leukocyte Adhesion Deficiency 1 (LAD); Graft Versus Host Disease (cGvHD)
Intervention:
Sponsor: National Institute of Allergy and Infectious Diseases (NIAID)
Recruiting
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Development of a Quality of Life Measurement Scale in Hereditary Haemorrhagic Telangiectasia (HHT) Disease.
Condition: Hereditary Hemorrhagic Telangiectasia
Interventions: Other: Phase 1 : fill out 1 scale; Other: Phase 2 : fill out 1 scale and 4 questionnaires
Sponsor: Hospices Civils de Lyon
Not yet recruiting
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BAX 111 rVWF in Pediatrics
Condition: Von Willebrand Disease
Interventions: Biological: von Willebrand factor (Recombinant); Biological: Antihemophilic Factor (Recombinant)
Sponsors: Baxalta now part of Shire; Baxalta Innovations GmbH, now part of Shire
Recruiting
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Investigation of the Genetics of Hematologic Diseases
Conditions: Bone Marrow Failure Syndromes; Erythrocyte Disorder; Leukocyte Disorder; Hemostasis; Blood Coagulation Disorder; Sickle Cell Disease; Dyskeratosis Congenita; Diamond-Blackfan Anemia; Congenital Thrombocytopenia; Severe Congenital Neutropenia; Fanconi Anemia
Intervention:
Sponsors: St. Jude Children's Research Hospital; Boston Children’s Hospital; Baylor College of Medicine; University of Memphis
Recruiting
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World Bleeding Disorders Registry
Conditions: Hemophilia A; Hemophilia B
Intervention:
Sponsor: World Federation of Hemophilia
Not yet recruiting
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Propranolol for Epistaxis in Hereditary Hemorrhagic Telangiectasia Patients
Conditions: Hereditary Hemorrhagic Telangiectasia; Osler Weber Rendu Disease
Interventions: Drug: Propranolol treatment; Drug: Placebo
Sponsors: University Hospital, Bordeaux; AMRO-HHT-France - Association Maladie de Rendu-Osler
Not yet recruiting
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Registry Study of COAGADEX® Patients With Moderate or Severe Hereditary Factor X Deficiency Undergoing Major Surgery
Condition: Factor 10 Deficiency
Intervention: Drug: Coagadex
Sponsor: Bio Products Laboratory
Recruiting
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Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
Conditions: Adeno-Associated Virus (AAV); Blood Coagulation Disorder; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Factor VIII (FVIII); Factor VIII (FVIII) Deficiency; Factor VIII (FVIII) Gene; Factor VIII (FVIII) Protein; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Gene Therapy; Gene Transfer; Hematologic Diseases; Hemorrhagic Disorders; Recombinant; Vector; Inhibitors
Intervention: Genetic: SPK-8016
Sponsor: Spark Therapeutics
Recruiting
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Latin-American Von Willebrand Disease Registry
Condition: Von Willebrand Disease
Intervention: Other: Observation
Sponsor: Academia Nacional de Medicina
Not yet recruiting
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Doxycycline for Hereditary Hemorrhagic Telangiectasia
Condition: Hereditary Hemorrhagic Telangiectasia (HHT)
Interventions: Drug: Doxycycline Hyclate; Drug: Placebo
Sponsors: St. Michael's Hospital, Toronto; Barrow Neurological Institute; Duke University; Feinstein Institute for Medical Research; University of Pittsburgh; Sunnybrook Health Sciences Centre
Recruiting
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Swiss Hemophilia Registry
Condition: Hemophilia and Other Severe Bleeding Disorders
Intervention: Other: Registry
Sponsor: Swiss Hemophilia Network
Recruiting
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Prospective Descriptive Study of the Angiogenic T Cell Population in Subjects With Hereditary Hemorrhagic Telangiectasia (HHT)
Condition: Hereditary Hemorrhagic Telangiectasia
Interventions: Biological: Blood samples; Other: Epistaxis charts
Sponsor: Centre Hospitalier Universitaire Dijon
Recruiting
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Pazopanib Effects on Bleeding in Hereditary Hemorrhagic Telangiectasia
Conditions: Hereditary Hemorrhagic Telangiectasia; Epistaxis Nosebleed; Anemia
Interventions: Drug: Pazopanib; Drug: Placebo oral capsule
Sponsor: Cure HHT
Not yet recruiting
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Timolol Gel for Epistaxis in Hereditary Hemorrhagic Telangiectasia
Condition: Hereditary Hemorrhagic Telangiectasia
Interventions: Drug: Timolol Gel; Drug: Placebo Gel
Sponsor: Washington University School of Medicine
Recruiting
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Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
Conditions: Blood Coagulation Disorder; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemophilia A; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Hematologic Diseases; Hemorrhagic Disorders; Factor VIII Deficiency
Intervention: Drug: Standard of Care FVIII Replacement therapy
Sponsor: Spark Therapeutics
Recruiting
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Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)
Conditions: Thrombotic Thrombocytopenic Purpura; Congenital Thrombotic Thrombocytopenic Purpura; Familial Thrombotic Thrombocytopenic Purpura; Thrombotic Thrombocytopenic Purpura, Congenital; Upshaw-Schulman Syndrome
Intervention: Other: Observation
Sponsors: University Hospital Inselspital, Berne; Swiss National Science Foundation; Mach Gaensslen Foundation; Baxalta Innovations GmbH, Wien, Austria
Recruiting
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Vitamin D and Hereditary Haemorrhagic Telangiectasia
Condition: Hereditary Haemorrhagic Telangiectasia
Interventions: Drug: Vit D; Drug: Placebo Oral Tablet
Sponsor: St. Paul's Hospital, Canada
Recruiting
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Molecular Studies on Hereditary Haemorrhagic Telangiectasia Families
Condition: Telangiectasia, Hereditary Hemorrhagic
Intervention:
Sponsors: Imperial College London; British Heart Foundation
Recruiting
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Pomalidomide in Hereditary Hemorrhagic Telangiectasia and Transfusion-Dependent Vascular Ectasia: a Phase I Study
Conditions: Hereditary Hemorrhagic Telangiectasia; Idiopathic Vascular Ectasia
Intervention: Drug: Pomalidomide
Sponsor: The Cleveland Clinic
Recruiting
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Pazopanib for the Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia
Conditions: Hereditary Hemorrhagic Telangiectasia; Epistaxis
Intervention: Drug: Pazopanib
Sponsors: Cure HHT; University of North Carolina
Not yet recruiting
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Pregnancy and Fibrinogen Disorders
Conditions: Hypofibrinogenemia, Congenital; Afibrinogenemia, Congenital; Dysfibrinogenemia, Congenital
Intervention:
Sponsors: University Hospital, Geneva; Swiss Hemophilia Network
Recruiting
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Pomalidomide for the Treatment of Bleeding in HHT
Condition: Telangiectasia, Hereditary Hemorrhagic
Interventions: Drug: Pomalidomide Oral Product; Drug: Placebo oral capsule
Sponsors: The Cleveland Clinic; RTI International
Recruiting
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Hereditary Hemorrhagic Telangiectasia (HHT) Research Outcomes Registry
Conditions: Hereditary Hemorrhagic Telangiectasia; HHT; Brain Arteriovenous Malformation (BAVM)
Intervention: Other: Registry and Saliva sample
Sponsors: St. Michael's Hospital, Toronto; HHT Foundation International; Dartmouth College
Recruiting
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Evaluation of Video-assisted Instructions of Nasal Self-packing in Patients With HHT
Condition: Hereditary Haemorrhagic Telangiectasia
Intervention: Procedure: video-assisted nasal self-packing
Sponsor: University Hospital, Essen
Recruiting
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An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
Conditions: Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor; Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor; Haemophilia A
Intervention: Drug: Standard of Care for Haemophilia A
Sponsor: Hoffmann-La Roche
Recruiting
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Prospective Pilot Study of Floseal for the Treatment of Anterior Epistaxis in Patients With (HHT)
Conditions: Hereditary Hemorrhagic Telangiectasia (HHT); Epistaxis
Interventions: Drug: Floseal; Other: Packing
Sponsors: St. Michael's Hospital, Toronto; The Ottawa Hospital
Recruiting
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Modifiers of Disease Severity in Cerebral Cavernous Malformations
Conditions: Cavernous Angioma, Familial; Cerebral Cavernous Malformations; Cerebral Cavernous Hemangioma
Intervention:
Sponsors: University of California, San Francisco; University of New Mexico; University of Chicago; National Institute of Neurological Disorders and Stroke (NINDS); Boston Children’s Hospital; Children's Hospital Medical Center, Cincinnati; Barrow Neurological Institute; Angioma Alliance
Recruiting
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ATHN 9: Severe VWD Natural History Study
Condition: Von Willebrand Diseases
Intervention:
Sponsors: American Thrombosis and Hemostasis Network; Takeda
Recruiting
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Characterization of New Candidate Genes in Cases of Human Inherited Thrombocytopenia (CATCH)
Conditions: Thrombocytopenia; Inherited Platelet Disorder
Intervention: Diagnostic Test: Validation of a new gene / protein variant implicated in familial CT
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
Not yet recruiting
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A Gene Transfer Study for Hemophilia A
Condition: Hemophilia A
Intervention: Genetic: SPK-8011
Sponsor: Spark Therapeutics
Recruiting
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Low VW Activity in Adolescent HMB
Condition: Von Willebrand Factor Deficiency
Interventions: Other: Genetic Analysis; Other: Medical Record Data Abstraction; Other: Pictorial Blood Assessment Chart (PBAC) score; Other: Complete Bleeding Symptom ISTH Bleeding Assessment Tool
Sponsors: Baylor College of Medicine; Shire
Recruiting
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Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B
Conditions: Congenital Bleeding Disorder; Haemophilia B
Intervention: Drug: nonacog beta pegol
Sponsor: Novo Nordisk A/S
Recruiting
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Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A
Condition: Hemophilia A
Intervention: Biological: valoctocogene roxaparvovec
Sponsor: BioMarin Pharmaceutical
Not yet recruiting
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Nationwide Awareness Campaign and Call for Dental Screening for HHT in Germany
Conditions: Hereditary Hemorrhagic Telangiectasia; Dental Diseases
Intervention:
Sponsors: University Hospital, Essen; Philipps University Marburg Medical Center; University Hospital, Aachen; German HHT self-help group
Recruiting
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Handheld Ultrasound (HHUS) for Home Use in Hemophilia
Conditions: Joint Bleed; Hemophilia A
Intervention: Device: handheld ultrasound: Philips Lumify integrated tele-ultrasound powered by Reacts collaborative platform
Sponsors: Bloodworks; Genentech, Inc.
Not yet recruiting
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A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Conditions: Hemophilia A; Hemophilia B
Interventions: Drug: fitusiran; Drug: factor VIII or factor IX
Sponsor: Genzyme, a Sanofi Company
Recruiting
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Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle".
Condition: Hemophilia A
Intervention: Drug: BAY2599023 (DTX201)
Sponsors: Bayer; Ultragenix pharmaceutical
Recruiting
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A Study to Evaluate the Safest Dose Range for FEIBA in Hemophilia A Patients With Inhibitors on Emicizumab
Condition: Hemophilia A With Inhibitor
Intervention: Drug: Feiba
Sponsors: Children's Hospital Los Angeles; Takeda
Not yet recruiting
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Perioperative Eltrombopag in Patients With Inherited Thrombocytopenia
Condition: Thrombocytopenia
Intervention: Drug: Eltrombopag
Sponsors: University Hospital, Toulouse; French network for inherited hemorragic diseases; National Reference Centre for Platelet Pathologies
Recruiting
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Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
Condition: Hemophilia A
Intervention: Other: assessment of joint health and bone density
Sponsors: Bloodworks; Genentech, Inc.
Recruiting
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Study Comparing the Pharmacokinetic of Biosimilar Eptacog Alfa With Novoseven®, in Patients With Congenital Factor VII Deficiency
Condition: Factor VII Deficiency
Interventions: Biological: Eptacog alfa, biosimilar and reference medicinal product Novoseven; Biological: Eptacog alfa biosimilar
Sponsor: AryoGen Pharmed Co.
Recruiting
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Von Willebrand Disease in the Netherlands
Condition: Von Willebrand Disease
Intervention:
Sponsors: Erasmus Medical Center; Stichting Haemophilia (Dutch Haemophilia Foundation); CSL Behring; Shire
Not yet recruiting
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Longitudinal Studies of Patient With FPDMM
Conditions: Inherited Hematological Diseases; Rare Diseases; FPDMM
Intervention:
Sponsor: National Human Genome Research Institute (NHGRI)
Recruiting
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Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome
Condition: Hermansky-Pudlak Syndrome (HPS)
Intervention:
Sponsor: National Human Genome Research Institute (NHGRI)
Recruiting
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UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
Conditions: Adrenoleukodystrophy; Batten Disease; Mucopolysaccharidosis II; Leukodystrophy, Globoid Cell; Leukodystrophy, Metachromatic; Neimann Pick Disease; Pelizaeus-Merzbacher Disease; Sandhoff Disease; Tay-Sachs Disease; Brain Diseases, Metabolic, Inborn; Alpha-Mannosidosis; Sanfilippo Mucopolysaccharidoses
Intervention: Biological: DUOC-01
Sponsors: Joanne Kurtzberg, MD; The Marcus Foundation
Recruiting
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A Study of Factor Inhibitors in Adult Patients With Hemophilia and Von Willebrand's Disease in Upper Egypt
Conditions: Hemophilia; Von Willebrand Diseases; Christmas Disease
Intervention:
Sponsor: Safaa AA Khaled
Not yet recruiting
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Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
Condition: Hemophilia
Intervention:
Sponsor: Karolinska Institutet
Recruiting
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A Factor IX Gene Therapy Study (FIX-GT)
Condition: Hemophilia B
Intervention: Biological: FLT180a
Sponsor: University College, London
Recruiting
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A Long-Term Follow-Up Study of Haemophilia B Patients Who Have Undergone Gene Therapy
Condition: Hemophilia B
Intervention: Biological: FLT180a
Sponsor: Freeline Therapeutics
Recruiting
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TGA (Thrombin Generation Assay) and Prophylaxis in Haemophilia
Condition: Haemophilia A
Intervention: Other: Thrombin generation measurement.
Sponsor: Hospices Civils de Lyon
Recruiting
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Study to Gain More Information on How Safe and Effective Jivi Works in Patients With Severe Hemophila A (Post-marketing Investigation)
Condition: Hemophilia A
Intervention: Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)
Sponsor: Bayer
Recruiting
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Minimize Menorrhagia in Women With Von Willebrand Disease
Condition: Von Willebrand Diseases
Interventions: Drug: recombinant von Willebrand factor; Drug: tranexamic acid
Sponsors: Margaret Ragni; University of North Carolina; Duke University
Recruiting
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Procurement and Analysis of Specimens From Individuals With Pulmonary Fibrosis
Conditions: Pulmonary Fibrosis; Healthy Volunteers; Hermansky-Pudlak Syndrome (HPS)
Intervention:
Sponsor: National Human Genome Research Institute (NHGRI)
Recruiting
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Intranasal Bevacizumab for HHT-Related Epistaxis
Conditions: HHT; Hereditary Hemorrhagic Telangiectasia; Epistaxis; Nose Bleeds; Nasal Bleeding
Interventions: Drug: Bevacizumab; Drug: Placebo (Saline)
Sponsor: Stanford University
Recruiting
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A Phase 3, Randomized, Controlled Study of Prophylactic and On-demand Treatment of cTTP With BAX 930 (rADAMTS13)
Condition: Congenital Thrombotic Thrombocytopenic Purpura
Interventions: Biological: BAX930; Biological: Standard of care
Sponsors: Baxalta now part of Shire; Baxalta Innovations GmbH, now part of Shire
Recruiting
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At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
Conditions: All Diagnosed Health Conditions; ADD/ADHD; Alopecia Areata; Ankylosing Spondylitis; Asthma; Atopic Dermatitis Eczema; Beta Thalassemia; Bipolar Disorder; Breast Cancer; Celiac Disease; Cervical Cancer; Chronic Inflammatory Demyelinating Polyneuropathy; Chronic Kidney Diseases; Chronic Obstructive Pulmonary Disease; Colon Cancer; Colorectal Cancer; Crohn's Disease; Cystic Fibrosis; Depression; Diabetes Mellitus; Duchenne Muscular Dystrophy; Endometriosis; Epilepsy; Facioscapulohumeral Muscular Dystrophy; G6PD Deficiency; General Anxiety Disorder; Hepatitis B; Hereditary Hemorrhagic Telangiectasia; HIV/AIDS; Human Papilloma Virus; Huntington's Disease; Idiopathic Thrombocytopenic Purpura; Insomnia; Kidney Cancer; Leukemia; Lung Cancer; Lupus Nephritis; Lymphoma; Melanoma; Multiple Myeloma; Multiple Sclerosis; Myositis; Myotonic Dystrophy; Ovarian Cancer; Pancreatic Cancer; Parkinson's Disease; Polycystic Kidney Diseases; Prostate Cancer; Psoriasis; Psoriatic Arthritis; Rosacea; Scleroderma; Sickle Cell Anemia; Sickle Cell Trait; Sjogren's Syndrome; Skin Cancer; Spinal Muscular Atrophy; Systemic Lupus Erythematosus; Thrombotic Thrombocytopenic Purpura; Trisomy 21; Ulcerative Colitis
Intervention:
Sponsor: Sanguine Biosciences
Recruiting
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Study of Chediak-Higashi Syndrome
Condition: Chediak Higashi Syndrome
Intervention:
Sponsor: National Human Genome Research Institute (NHGRI)
Recruiting
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Medication Exposure in Older Patients With Constitutional Hemorrhagic Disease
Condition: Hemophilia
Intervention: Other: Telephone interview
Sponsor: Hospices Civils de Lyon
Not yet recruiting
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Videomicroscopy for the Prediction of Bleeding in Constitutional Haemorrhagic Diseases
Conditions: Von Willebrand Diseases; Glanzmann Thrombasthenia
Interventions: Device: Sublingual videomicroscopy; Biological: blood sample
Sponsor: University Hospital, Lille
Not yet recruiting
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An International Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B
Condition: Hemophilia B
Intervention: Drug: Alprolix
Sponsor: Swedish Orphan Biovitrum
Recruiting
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Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
Condition: Von Willebrand Diseases
Intervention: Drug: VWF-containing products
Sponsor: Octapharma
Recruiting
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Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry & Jivi: A Nested Study Within an Existing Registry
Condition: Hemophilia A
Intervention: Drug: New FVIII products
Sponsors: Bayer; American Thrombosis and Hemostasis Network
Recruiting
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A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Conditions: Hematologic Malignancies; Inherited Disorders of Metabolism; Inherited Abnormalities of Platelets; Histiocytic Disorders; Acute Myelogenous Leukemia (AML or ANLL); Acute Lymphoblastic Leukemia (ALL); Other Acute Leukemia; Chronic Myelogenous Leukemia (CML); Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases; Other Leukemia; Hodgkin Lymphoma; Non-hodgkin Lymphoma; Multiple Myeloma/ Plasma Cell Disorder (PCD); Inherited Abnormalities of Erythrocyte Differentiation or Function; Disorders of the Immune System; Automimmune Diseases; Severe Aplastic Anemia
Intervention: Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
Sponsors: Center for International Blood and Marrow Transplant Research; National Marrow Donor Program
Recruiting
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Study Evaluating "Real World" Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog Alfa) for Routine Prophylaxis
Condition: Hemophilia A, Congenital
Intervention: Biological: Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973
Sponsor: Bayer
Recruiting
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Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A
Condition: Hemophilia A
Intervention: Drug: Damoctocog alfa pegol (Jivi, Bay94-9027)
Sponsor: Bayer
Recruiting
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ATHN 7: Hemophilia Natural History Study
Conditions: Hemophilia A With Inhibitor; Haemophilia A Without Inhibitor; Hemophilia B With Inhibitor; Haemophilia B Without Inhibitor
Intervention:
Sponsors: American Thrombosis and Hemostasis Network; Genentech, Inc.
Recruiting
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China Registry for Genetic / Metabolic Liver Diseases
Condition: Genetic/Metabolic Liver Diseases
Intervention: Drug: Standard of care
Sponsors: Beijing Friendship Hospital; Beijing YouAn Hospital; Henan Provincial People's Hospital; Beijing Ditan Hospital; Hebei Medical University Third Hospital; Peking University First Hospital; Xinjiang Uygur Autonomous Region Traditional Chinese Medicine Hospital; Nanfang Hospital of Southern Medical University; Logistics University of Chinese People's Armed Police Forces; Beijing Anzhen Hospital; West China Second University Hospital of Sichuan University; Jinshan Hospital of Fudan University; Fudan University
Recruiting
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Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and B
Conditions: Haemophilia A; Haemophilia B
Intervention:
Sponsor: Swedish Orphan Biovitrum
Recruiting
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Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Patients With Severe Hemophilia A or B
Conditions: Hemophilia A; Hemophilia B
Intervention: Drug: PF-06741086
Sponsor: Pfizer
Recruiting
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Frequency of Hemorrhages Associated With the Functional Anomalies of Willebrand Factor in Emergency Patients
Condition: Von Willebrand Diseases
Intervention:
Sponsor: University Hospital, Lille
Recruiting
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ATHN 8: PUPs Matter Study
Conditions: Hemophilia; Hemophilia A; Hemophilia B; Hemophilia A With Inhibitor; Hemophilia B With Inhibitor; Haemophilia B Without Inhibitor; Haemophilia A Without Inhibitor; Haemophilia
Intervention:
Sponsors: American Thrombosis and Hemostasis Network; CSL Behring; Octapharma; Takeda
Recruiting
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HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
Condition: Hemophilia B
Intervention: Genetic: AAV5-hFIXco-Padua
Sponsor: UniQure Biopharma B.V.
Recruiting
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rVWF Pediatric and Adult Study
Condition: Von Willebrand Disease
Interventions: Biological: rVWF; Biological: rFVIII
Sponsors: Baxalta now part of Shire; Baxalta Innovations GmbH, now part of Shire
Recruiting
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Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
Condition: Von Willebrand Diseases
Intervention: Drug: Wilate
Sponsor: Octapharma
Not yet recruiting
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Effectiveness and Tolerability of Eqwilate in Real-life Conditions
Condition: VWD - Von Willebrand's Disease
Intervention: Biological: Eqwilate
Sponsor: Octapharma
Recruiting
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Treatment of Hemophilia A Patients With FVIII Inhibitors
Condition: Hemophilia A
Interventions: Biological: Nuwiq; Biological: Octanate; Biological: Wilate; Biological: Emicizumab; Biological: Recombinant factor VIIa (rFVIIa); Biological: Activated prothrombin complex concentrate (aPCC)
Sponsors: Emory University; Octapharma
Not yet recruiting
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Evaluation of Immune Status Before and After Splenectomy in Immune Thrombocytopenia Patients
Conditions: Thrombocytopenia; Splenectomy
Intervention: Procedure: splenectomy
Sponsor: Zhang Lei
Recruiting
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Evaluation of Bleeding Score in Egyptian Patients With vWD Type I and Correlate it With Laboratory Parameters
Condition: Von Willebrand Disease, Type 1
Intervention: Diagnostic Test: The von Willebrand Antigen level
Sponsor: Assiut University
Not yet recruiting
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Differences in Preparation for Small Bowel Capsule Endoscopy
Conditions: Capsule Endoscopy; Inflammatory Bowel Diseases; Celiac Disease
Interventions: Drug: Klean Prep; Other: Clear liquids only
Sponsors: Sheffield Teaching Hospitals NHS Foundation Trust; Hotel Dieu Hospital
Recruiting
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Von Willebrand Antigen and Activity as Novel Biomarkers of Hemostasis in Inflammatory Bowel Disease
Conditions: Inflammatory Bowel Diseases; Von Willebrand Diseases
Intervention: Diagnostic Test: von willebrand antigen and activity
Sponsor: Assiut University
Recruiting
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ADVATE/ ADYNOVI Hemophilia A Outcome Database (AHEAD)
Condition: Hemophilia A
Interventions: Biological: ADVATE; Biological: ADYNOVI
Sponsors: Baxalta now part of Shire; Baxalta Innovations GmbH, now part of Shire
Recruiting
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The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
Condition: Hemophilia
Interventions: Other: Blood sample; Other: Questionnaire; Other: MRI-imaging; Other: Physical examination
Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Recruiting
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Development of a Device for Evaluating Primary Hemostasis Under Whole Blood Flow Conditions
Conditions: Von Willebrand Diseases; Major Constitutional Thrombopathy; Patient on Antiplatelet Drugs
Intervention: Biological: Blood sampling
Sponsor: Centre Hospitalier Universitaire Dijon
Recruiting
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Trial of Acquired Haemophilia With Steroid Combined With Cyclophosphamide Versus Steroid Combined With Rituximab
Condition: Acquired Hemophilia A
Interventions: Drug: Steroid; Drug: Rituximab; Drug: Cyclophosphamide
Sponsors: Zhang Lei; Qilu Hospital of Shandong University; Tianjin First Central Hospital; The Second Affiliated Hospital of Kunming Medical University; Henan Cancer Hospital
Recruiting
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Mutation p.Ile112Thr : Discrepancy Between Factor IX Level and Bleeding Phenotype
Condition: Hemophilia B
Intervention: Other: data collection
Sponsor: Centre Hospitalier Universitaire Dijon
Not yet recruiting
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The Safety and Tolerability of SerpinPC in Healthy Men and in Men With Severe Blood Disorders (Haemophilia A and B)
Conditions: Hemophilia A; Hemophilia B
Interventions: Drug: SerpinPC; Drug: Placebo
Sponsor: ApcinteX Ltd
Recruiting
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A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
Condition: Hemophilia A
Intervention: Drug: ELOCTA
Sponsor: Swedish Orphan Biovitrum
Not yet recruiting
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PHYSICAL ACTIVITY AND FVIII CLEARANCE: RELEVANCE FOR PERSONALIZED THERAPY IN SEVERE HAEMOPHILIA A (PHYSEMO)
Condition: Hemophilia A
Intervention: Device: SenseWear® armband device
Sponsor: Catholic University of the Sacred Heart
Recruiting
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Safety, Efficacy Evaluation of Recombinant Coagulation Factor VIII Injection in Subjects With Hemophilia A.
Condition: Hemophilia A
Intervention: Drug: Recombinant Human Coagulation FVIII
Sponsor: Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Recruiting
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Extended Half-Life (EHL) Clotting Factor for Improving the Musculoskeletal Health of Patients With Hemophilia
Condition: Haemophilia
Intervention: Other: EHL clotting factor
Sponsor: Investigación en Hemofilia y Fisioterapia
Not yet recruiting
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Post-Marketing Surveillance (Use-results Surveillance) With Esperoct®
Condition: Haemophilia A
Intervention: Drug: Turoctocog alfa pegol
Sponsor: Novo Nordisk A/S
Not yet recruiting
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Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study)
Condition: Hemophilia A
Intervention: Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)
Sponsor: Bayer
Not yet recruiting
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A Phase 3, Open-label Interventional Study of an Intravenous Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein, BIVV001, in Patients With Severe Hemophilia A (XTEND-1)
Condition: Factor VIII Deficiency
Intervention: Drug: BIVV001
Sponsor: Sanofi
Recruiting
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A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis
Condition: Hemophilia
Intervention: Drug: Fitusiran
Sponsor: Genzyme, a Sanofi Company
Recruiting
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Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A.
Condition: Hemophilia A
Intervention: Drug: Recombinant Human Coagulation FVIII
Sponsor: Sinocelltech Ltd.
Not yet recruiting
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Update in VWD Laboratory Diagnosis
Condition: VWD - Von Willebrand's Disease
Intervention:
Sponsor: Assiut University
Not yet recruiting
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Pharmacokinetic, Efficacy and Safety of BT524 in Patients With Congenital Fibrinogen Deficiency
Conditions: Congenital Afibrinogenemia; Congenital Hypofibrinogenemia
Intervention: Biological: BT524 (fibrinogen concentrate from human plasma)
Sponsors: Biotest; ICON plc; SYNLAB Analytics & Services Germany GmbH; Phoenix Clinical Research; Accovion GmbH
Recruiting
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