Rare Inherited Bleedig Disorders in Children at Sohag University Hospital
Condition: Rare Inherited Bleeding Disorders in Children at Sohag University Hospital
Intervention: Other: blood laboratory investigation(coagulation profile- platelet function test)
Sponsor: Sohag University
Recruiting
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Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
Conditions: Von Willebrand Disease, Type 3; Concomitant VWD and Hemophilia
Intervention: Drug: Emicizumab
Sponsors: Bleeding and Clotting Disorders Institute Peoria, Illinois; Genentech, Inc.
Recruiting
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Genetic Background of Patients With Low Von Willebrand Factor Levels
Condition: Low Von Willebrand Factor
Intervention: Diagnostic Test: Whole-exome sequencing
Sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Recruiting
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Embolization in Hereditary Coagulopathies
Conditions: Hemophilia; Embolization; Hemarthrosis; Clotting Factor Deficiency; Synovitis; Arthropathy
Intervention: Device: embolization with spherical microparticles embosphere
Sponsors: University of Sao Paulo General Hospital; Merit Medical Systems, Inc.
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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Evaluation of Hemostasis in Bleeding and Thrombotic Disorders
Conditions: Blood Coagulation Disorders, Inherited; Thrombotic Disorder
Intervention:
Sponsor: The University of Texas Health Science Center, Houston
Recruiting
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Genomics of Fibrin Clot Structure in Patients With Constitutional Dysfibrinogenemia
Condition: Hereditary Dysfibrinogenemia
Intervention: Biological: Blood test
Sponsor: University Hospital, Clermont-Ferrand
Recruiting
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Chronic Pain and Hemophilia
Conditions: Chronic Pain; Hemophilia
Intervention: Other: questionnaires
Sponsor: University Hospital, Clermont-Ferrand
Not yet recruiting
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Cerebral Hemorrhage Risk in Hereditary Hemorrhagic Telangiectasia
Condition: Hereditary Hemorrhagic Telangiectasia
Intervention:
Sponsors: Unity Health Toronto; National Institute of Neurological Disorders and Stroke (NINDS)
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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ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Conditions: Hematologic Disorder; Bleeding Disorder; Connective Tissue Disorder; Hemophilia; Thrombosis; Von Willebrand Diseases; Thrombophilia; Rare Bleeding Disorder; Platelet Disorder
Intervention:
Sponsors: American Thrombosis and Hemostasis Network; Pfizer; Hemophilia of Georgia, Inc.; Genentech, Inc.; Hemab Therapeutics; CSL Behring
Recruiting
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Prospective Study of Antiplatelet and Anticoagulation Therapy in Hereditary Haemorrhagic Telangiectasia
Conditions: Hereditary Hemorrhagic Telangiectasia; Rendu Osler Disease
Intervention: Other: monitoring the use of anticoagulant and/or antiplatelet therapy in patients with osler rendering disease
Sponsor: University Hospital, Clermont-Ferrand
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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A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
Condition: Von Willebrand Disease
Interventions: Biological: von Willebrand factor (Recombinant); Biological: Antihemophilic Factor (Recombinant)
Sponsors: Baxalta now part of Shire; Takeda Development Center Americas, Inc.
Recruiting
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Bevacizumab In Hereditary Hemorrhagic Telangiectasia
Condition: Hereditary Hemorrhagic Telangiectasia
Intervention: Drug: Bevacizumab
Sponsor: Hanny Al-Samkari, MD
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; Myelodysplastic Syndromes; Myeloproliferative Diseases
Intervention:
Sponsors: St. Jude Children's Research Hospital; Boston Children's Hospital; University of Memphis; Monroe Carell Jr. Children's Hospital at Vanderbilt; Baylor College of Medicine; Children's Hospital of Philadelphia; Dana-Farber Cancer Institute
Recruiting
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Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
Conditions: Hemophilia A; Gene Therapy; Clotting Disorders; Blood Disorder
Intervention: Biological: Valoctocogene Roxaparvovec
Sponsor: BioMarin Pharmaceutical
Recruiting
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Belgian Antithrombin Deficiency Registry
Condition: Antithrombin III Deficiency
Intervention: Other: observation
Sponsor: Universitair Ziekenhuis Brussel
Not yet recruiting
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World Bleeding Disorders Registry
Conditions: Hemophilia A; Hemophilia B
Intervention:
Sponsor: World Federation of Hemophilia
Recruiting
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TPO-Mimetic Use in Children for Hematopoietic Failure
Conditions: Bone Marrow Failure Disorders; Aplastic Anemia; Thrombocytopenia; Refractory Cytopenia of Childhood; Myelodysplastic Syndrome(MDS)
Intervention: Drug: Romiplostim
Sponsors: Anjali Sharathkumar; Amgen
Recruiting
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Genotype-phenotype Association in Hereditary Hemorrhagic Telangiectasia
Conditions: Hereditary Hemorrhagic Telangiectasia; HHT; Rendu Osler Weber Disease
Intervention: Other: Registry
Sponsors: Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal; Sociedad Española De Medicina Interna
Recruiting
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Influence of Hypoxic Induced Factors in Patients With Hereditary Hemorrhagic Telangiectasia
Condition: Hereditary Hemorrhagic Telangiectasia
Intervention: Other: hypoxic induced factors
Sponsor: University Hospital, Essen
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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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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Efficacy of Nintedanib for Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) Patients
Condition: Telangiectasia, Hereditary Hemorrhagic
Interventions: Drug: Nintedanib; Drug: Placebo
Sponsors: Dr. Romain Lazor; Boehringer Ingelheim
Recruiting
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Tacrolimus Trial for Hereditary Hemorrhagic Telangiectasia (HHT)
Conditions: Hereditary Hemorrhagic Telangiectasia; Epistaxis Nosebleed
Intervention: Drug: Tacrolimus capsule (low-dose)
Sponsors: Unity Health Toronto; United States Department of Defense
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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National Longitudinal Cohort of Hematological Diseases
Conditions: Multiple Myeloma; Myeloma, Multiple; Myelomas, Multiple; Acute Myeloid Leukemia; Leukemias, Acute Myeloid; Myeloid Leukemias, Acute; Hemophilia; Hemophilia A; Hemophilia As; Hemophilia B; Hemophilia Bs; Myelodysplastic Syndrome; MDS; Lymphoma; Leukemia; Aplastic Anemia; Bleeding Disorder; Bone Marrow Transplantation; Blood Disease Infection
Intervention:
Sponsor: Institute of Hematology & Blood Diseases Hospital
Recruiting
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He-move-philia, Lifestyle Intervention for Patients With Hemophilia
Condition: Hemophilia
Interventions: Other: Lifestyle - group sessions; Other: Lifestyle - individual coaching
Sponsor: Radboud University Medical Center
Recruiting
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Effects of Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia (Paz)
Conditions: Hereditary Hemorrhagic Telangiectasia; Epistaxis; Anemia; Nosebleed; HHT
Interventions: Drug: Pazopanib; Drug: Placebo oral capsule
Sponsors: Cure HHT; United States Department of Defense; Food and Drug Administration (FDA)
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; Arteriovenous Malformation of Brain
Intervention: Other: Registry and Saliva sample
Sponsors: Unity Health Toronto; Cure HHT; 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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Assess Safety and Efficacy of VAD044 in HHT Patients
Condition: Hereditary Hemorrhagic Telangiectasia (HHT)
Intervention: Drug: VAD044
Sponsor: Vaderis Therapeutics AG
Recruiting
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A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood
Conditions: Treatment of Bleeding; Prophylaxis of Bleeding; Hemophilia A; Children
Intervention: Biological: Damoctocog alfa pegol (Jivi, BAY94-9027)
Sponsor: Bayer
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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Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
Conditions: Hemophilia A With Inhibitor; Hemophilia A With Anti Factor VIII
Intervention: Biological: Valoctocogene roxaparvovec
Sponsor: BioMarin Pharmaceutical
Recruiting
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Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Conditions: Severe Aplastic Anemia; Telomere Biology Disorders; Inherited Bone Marrow Failure Syndromes
Intervention:
Sponsor: National Heart, Lung, and Blood Institute (NHLBI)
Recruiting
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A Study to Learn More About Treatment With Damoctocog Alfa Pegol, How it is Used in Every Day Practice ("Real-World"), and How Satisfied People Who Receive Damoctocog Alfa Pegol Are in United States (US) Hemophilia Treatment Centers
Condition: Hemophilia A
Intervention: Other: No intervention
Sponsor: Bayer
Recruiting
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Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis
Condition: Hemophilia A
Interventions: Drug: Emicizumab; Drug: FVIII
Sponsors: Wayne State University; Genentech, Inc.
Not yet recruiting
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Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
Condition: Thrombotic Thrombocytopenic Purpura (TTP)
Intervention: Drug: TAK-755
Sponsor: Takeda
Available
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Haemophilia and Bone Loss - PHILEOS Study
Condition: Hemophilia
Interventions: Radiation: Bone densitometry (BMD); Biological: Blood sampling for patients only
Sponsors: Centre Hospitalier Universitaire de Saint Etienne; Ministry of Health, France
Recruiting
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Prevent Postpartum Hemorrhage in Women With Von Willebrand Disease: The VWD-WOMAN Trial
Conditions: Von Willebrand Diseases; Postpartum Hemorrhage
Interventions: Drug: Recombinant Von Willebrand factor; Drug: Tranexamic Acid Injection [Cyklokapron]
Sponsor: Margaret Ragni
Recruiting
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Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
Condition: Hemophilia A
Intervention: Biological: Auto CD34+PBSC transduced with a lentiviral vector encoding a novel coagulation factor VIII transgene
Sponsors: Christian Medical College, Vellore, India; Dr. H. Trent Spencer, Professor, Emory University of Medicine, Atlanta Ga, 30322
Recruiting
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Efficacy of Nintedanib Per os as a Treatment for Epistaxis in HHT Disease.
Conditions: Telangiectasia, Hereditary Hemorrhagic; Rendu Osler Disease
Interventions: Drug: Nintedanib 150 mg and 100 mg soft capsules; Drug: Oral treatment of placebo soft capsule
Sponsor: Hospices Civils de Lyon
Recruiting
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Prophylaxis Regimen for Hemophilia A Patients
Conditions: Hemophilia A; Prophylaxis of Bleeding
Intervention: Biological: Damoctocog alfa-pegol (Jivi, BAY94-9027)
Sponsor: Bayer
Recruiting
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Genetic Influence of Genetic Factors Influencing the Desmopressin's Efficacy in Mild/Moderate Hemophilia A
Conditions: Hemophilia A, Mild; Desmopressin; Factor VIII
Intervention: Drug: Desmopressin
Sponsor: Groupe Maladies hémorragiques de Bretagne
Recruiting
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Emotions in the Communication and Relationship Styles of Parents With Hemophilic Children.
Condition: Hemophilia A
Intervention:
Sponsor: Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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: Washington Institute for Coagulation; Genentech, Inc.
Recruiting
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An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A
Conditions: Hemophilia A; Prophylaxis of Bleeding
Intervention: Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)
Sponsor: Bayer
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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Emicizumab in Patients With Acquired Hemophilia A
Condition: Acquired Hemophilia A
Intervention: Drug: emicizumab
Sponsors: University of Washington; Genentech, Inc.
Recruiting
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Sirolimus for Nosebleeds in HHT
Conditions: Hereditary Hemorrhagic Telangiectasia; Nosebleeds; Epistaxis
Intervention: Drug: Sirolimus
Sponsors: Unity Health Toronto; National Institutes of Health (NIH)
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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A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Condition: Thrombotic Thrombocytopenic Purpura (TTP)
Intervention: Biological: TAK-755
Sponsors: Takeda; Takeda Development Center Americas, Inc.; Shire
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 Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP)
Condition: Thrombotic Thrombocytopenic Purpura (TTP)
Interventions: Biological: BAX930; Biological: Standard of care
Sponsors: Baxalta now part of Shire; Takeda Development Center Americas, Inc.
Recruiting
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Comparison of Efficiency of Closed Kinetic Chain Exercises Versus Proprioceptive Exercises Patient With Hemophilia
Conditions: Hemophilia A; Hemophilia B
Interventions: Other: Closed Chain Exercise Group; Other: Proprioceptive Exercise Group
Sponsor: Bahçeşehir University
Not yet 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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Gaining Insight Into the Complexity of Pain in Patients With Haemophilia
Conditions: Hemophilia A; Hemophilia B
Intervention: Diagnostic Test: Biopsychological pain assessment
Sponsors: Universiteit Antwerpen; Cliniques universitaires Saint-Luc- Université Catholique de Louvain; University Hospital, Antwerp
Recruiting
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Aspirin for Prophylaxis of TTP
Condition: Thrombotic Thrombocytopenic Purpura
Interventions: Drug: Aspirin tablet; Drug: Placebo
Sponsor: The First Affiliated Hospital of Soochow University
Not yet recruiting
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SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors
Conditions: Hemophilia A With Inhibitor; Hemophilia B With Inhibitor
Intervention: Drug: coagulation factor VIIa [recombinant]-jncw
Sponsors: American Thrombosis and Hemostasis Network; LFB USA, Inc.
Recruiting
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Efficacy and Safety of Fanhdi®, a High-purity Von Willebrand Containing FVIII Concentrate, in Pediatric Patients With Von Willebrand Disease
Condition: Von Willebrand Disease
Intervention: Drug: plasma-derived FVIII/VWF concentrate
Sponsors: Grifols Therapeutics LLC; Instituto Grifols, S.A.
Recruiting
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Bone Microarchitecture in Men With Hemophilia
Conditions: Severe Hemophilia A; Osteoporosis
Interventions: Radiation: HR-pQCT; Biological: Blood sample; Radiation: Dual energy X-ray absorptiometry; Other: medical data collection
Sponsor: Hospices Civils de Lyon
Recruiting
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Pre-clinical Models for Mesenchymal Stem Cell Therapy in Hemophilic Arthropathy
Conditions: Hemophilia A; Hemophilia B; Arthropathy
Intervention:
Sponsor: University Hospital, Montpellier
Recruiting
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PPI Supplementation to Fight ECtopIc Calcification in PXE
Condition: Pseudoxanthoma Elasticum
Interventions: Dietary Supplement: study treatment PPI; Dietary Supplement: Placebo comparator
Sponsor: Centre Hospitalier Universitaire de Nice
Recruiting
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Proximal Aortopathy in Scotland - Epidemiology and Surgical Outcomes
Conditions: Aortic Diseases; Aortic Dissection; Aortic Aneurysm, Thoracic; Aortic Ectasia; Aortic Valve Insufficiency; Bicuspid Aortic Valve; Marfan Syndrome; Ehlers-Danlos Syndrome; Loeys-Dietz Syndrome
Interventions: Procedure: Surgery on the proximal aorta (aortic root +/- ascending aorta); Other: Patients diagnosed with thoracic aortopathy but not operated upon
Sponsors: Golden Jubilee National Hospital; Aberdeen Royal Infirmary; Royal Infirmary of Edinburgh; Network for Inherited Cardiac Conditions Scotland; University of Glasgow
Not yet recruiting
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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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Study of Calcium-phosphate Complications Induced by the Administration of IV Iron Supplementation in Patients With Rendu-Osler Disease
Condition: Rendu Osler Disease
Intervention:
Sponsor: University Hospital, Strasbourg, France
Recruiting
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Baby Detect : Genomic Newborn Screening
Conditions: Congenital Adrenal Hyperplasia; Familial Hyperinsulinemic Hypoglycemia 1; Phosphoglucomutase 1 Deficiency; Maturity Onset Diabetes of the Young; Cystic Fibrosis; Hypophosphatasia, Infantile; Congenital Hypothyroidism; DAVID; Pituitary Hormone Deficiency, Combined; Diamond Blackfan Anemia; Wiskott-Aldrich Syndrome; Fanconi Anemia; Hemophilia A; Hemophilia B; Glucose 6 Phosphate Dehydrogenase Deficiency; Alpha-Thalassemia; Sickle Cell Disease; Shwachman-Diamond Syndrome; Alpha 1-Antitrypsin Deficiency; Inflammatory Bowel Disease 25, Autosomal Recessive; Wilson Disease; Progressive Familial Intrahepatic Cholestasis; Crigler-Najjar Syndrome; DIAR4; Familial Chylomicronemia; Lysosomal Acid Lipase Deficiency; Familial Hemophagocytic Lymphocytosis; Griscelli Syndrome; Chediak-Higashi Syndrome; Severe Congenital Neutropenia; SCID; Chronic Granulomatous Disease; Menkes Disease; X-ALD; Smith-Lemli-Opitz Syndrome; Ataxia With Vitamin E Deficiency; THMD5; THMD4; Thiamine-Responsive Megaloblastic Anemia; Thiamine Metabolism Dysfunction Syndrome 2; GOT2 DEFICIENCY; Cerebral Folate Transport Deficiency; Segawa Syndrome, Autosomal Recessive; Congenital Myasthenic Syndrome; Metachromatic Leukodystrophy; Sepiapterin Reductase Deficiency; Dopamine Beta Hydroxylase Deficiency; Glut1 Deficiency Syndrome; Late-Infantile Neuronal Ceroid Lipofuscinosis; Aromatic L-amino Acid Decarboxylase Deficiency; Charcot-Marie-Tooth Disease, Type 6C; Hereditary Hyperekplexia; Brain Dopamine-Serotonin Vesicular Transport Disease; Very Long Chain Hydroxy Acyl Dehydrogenase Deficiency; Tyrosinemia, Type I; Disaccharide Intolerance I; Beta Ketothiolase Deficiency; Phosphoglycerate Dehydrogenase Deficiency; Succinyl-Coa 3-Oxoacid Transferase Deficiency; Pyridoxine-5'-Phosphate Oxidase Deficiency; Pyridoxine-Dependent Epilepsy; Propionic Acidemia; Pompe Disease; Phenylalanine Hydroxylase Deficiency; Ornithine Transcarbamylase Deficiency; N Acetyl Glutamate Synthethase Deficiency; Riboflavin Deficiency; Maple Syrup Urine Disease; Medium Chain Acyl CoA Dehydrogenase Deficiency; Malonic Acidemia; Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency; Isovaleric Acidemia; Phosphoserine Aminotransferase Deficiency; Phosphoserine Phosphatase Deficiency; Spatccm; Hyperornithinemia-Hyperammonemia-Homocitrullinuria; MRT8, FORMERLY; S-Adenosylhomocysteine Hydrolase Deficiency; Mucopolysaccharidosis VII; Mucopolysaccharidosis VI; Mucopolysaccharidosis IV A; Mucopolysaccharidosis II; Mucopolysaccharidosis I; Transcobalamin Deficiency; Isolated Methylmalonic Acidemia; Cobalamin Deficiency; Homocystinuria; Holocarboxylase Synthetase Deficiency; Fanconi Bickel Syndrome; Glycogen Storage Disease; Glycine Encephalopathy; Glutaric Acidemia I; Glucose Galactose Malabsorption; Gaucher Disease, Type 1; Galactosemias; Fructosemia; Fructose-1,6-Diphosphatase Deficiency; Carbamoyl Phosphate Synthase 1 Deficiency; Citrullinemia Type II; Citrullinemia 1; Creatine Deficiency Syndrome; Systemic Primary Carnitine Deficiency; Carnitine Palmitoyltransferase Deficiency 2; Carnitine Palmitoyltransferase Deficiency 1; Carnitine Acylcarnitine Translocase Deficiency; Riboflavin Transporter Deficiency; Branched-Chain Keto Acid Dehydrogenase Kinase Deficiency; Andersen Tawil Syndrome; Timothy Syndrome; Jervell-Lange Nielsen Syndrome; Catecholaminergic Polymorphic Ventricular Tachycardia; Familial Hypertrophic Cardiomyopathy Type 4; Pseudohypoaldosteronism, Type II; Pseudohypoaldosteronism Type 1; Primary Hyperoxaluria; X Linked Hypophosphatemia; Hereditary Nephrogenic Diabetes Insipidus; Cystinosis; Congenital Nephrotic Syndrome, Finnish Type; Alport Syndrome; Hereditary Retinoblastoma; Biotinidase Deficiency; Aciduria, Argininosuccinic; Arginemia; ACAD9 Deficiency; 3-Hydroxy 3-Methyl Glutaric Aciduria; 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency
Intervention:
Sponsors: Laurent Servais; Centre Hospitalier Universitaire de Liege; University of Liege
Recruiting
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A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa
Condition: Hemophilia A, Severe
Intervention: Drug: Efanesoctocog alfa
Sponsors: Swedish Orphan Biovitrum; Syneos Health
Not yet 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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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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A Non-interventional Cohort Safety Study of Patients With hATTR-PN
Condition: Hereditary Transthyretin Amyloidosis With Polyneuropthy
Intervention: Other: Data Collection
Sponsors: Akcea Therapeutics; United BioSource, LLC
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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Association Between Individual Clotting Factor Level Monitoring and the Risk of Bleeding Whilst Physical Active Conditions
Condition: Hemophilia A
Intervention: Other: Observation of the association between clotting factor level and risk of bleeding whilst physical activity
Sponsors: Prof. Dr. Dr. Thomas Hilberg; Takeda; University of Bonn
Recruiting
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A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
Condition: Hemophilia
Interventions: Drug: Fitusiran; Drug: Clotting factor concentrates (CFC) or bypassing agents (BPA); Drug: Antithrombin concentrate (ATIIIC)
Sponsor: Sanofi
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; Autoimmune 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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Personalized Therapy of Patients Suffering From Rare Genodermatoses
Condition: Rare Diseases
Intervention: Other: If a targetable cytokine is increased in the lesional skin of a patient, this patient is treated with the respective antibody therapy (off-label use of approved drug).
Sponsor: Johannes Kepler University of Linz
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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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
Recruiting
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An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa
Conditions: Hemophilia A; Hemophilia B
Interventions: Procedure: Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Other: Haemophilia Joint Health Score (HJHS)
Sponsors: Swedish Orphan Biovitrum; IQVIA Pvt. Ltd
Not yet 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; Jinshan Hospital Fudan University; Fudan University
Recruiting
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Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Conditions: Hemophilia A; Hemophilia B
Intervention: Drug: PF-06741086
Sponsor: Pfizer
Recruiting
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SCOPE HIM SCOPE HIM
Condition: Hemophilia
Intervention: Biological: Coagulation Factor VIIa (Recombinant)
Sponsor: Laboratoire français de Fractionnement et de Biotechnologies
Not yet recruiting
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Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Conditions: Hemophilia A; Hemophilia B
Intervention: Drug: PF-06741086
Sponsor: Pfizer
Recruiting
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A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Conditions: Hemophilia A; Hemophilia B
Intervention: Drug: marstacimab
Sponsor: Pfizer
Recruiting
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A Study of Recombinant Von Willebrand Factor (rVWF) (TAK-577) in Children With Severe Von Willebrand Disease (vWD)
Condition: Von Willebrand Disease (VWD)
Interventions: Biological: Recombinant von Willebrand Factor (rVWF); Biological: ADVATE
Sponsor: Takeda
Not yet recruiting
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Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery
Condition: Congenital Antithrombin Deficiency
Intervention: Drug: Atenativ
Sponsor: Octapharma
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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A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
Condition: Von Willebrand Disease (VWD)
Interventions: Biological: rVWF; Biological: rFVIII
Sponsors: Baxalta now part of Shire; Takeda Development Center Americas, Inc.
Recruiting
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Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease VWD) Patients <6 Years of Age
Condition: Von Willebrand Disease
Intervention: Drug: wilate
Sponsor: Octapharma
Recruiting
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Biomechanical and Microstructural Properties of Ascending Aortic Aneurysms
Condition: Thoracic Aortic Aneurysms
Interventions: Other: Biomechanical and microstructural analysis of ATAA; Radiation: ECG-gated CT
Sponsor: Universitaire Ziekenhuizen KU Leuven
Recruiting
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A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers
Condition: Von Willebrand Disease (VWD)
Intervention: Other: No Intervention
Sponsor: Takeda
Recruiting
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A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
Condition: Hemophilia B
Intervention: Other: Non-Interventional
Sponsor: Regeneron Pharmaceuticals
Not yet 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
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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Gene Therapy for Chinese Hemophilia A
Conditions: Hemophilia A; Gene Therapy
Intervention: Genetic: Injection of GS001
Sponsor: Institute of Hematology & Blood Diseases Hospital
Recruiting
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Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.
Condition: Hemophilia A
Intervention: Drug: Recombinant Human Coagulation FVIII
Sponsor: Sinocelltech Ltd.
Recruiting
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Acquired Hemophilia A and Autoimmunity. Study of Lymphocyte Populations and Myeloid-Derived Suppressor Cells
Condition: Hemophilia A
Intervention: Other: no intervention
Sponsor: Nantes University Hospital
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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Update in VWD Laboratory Diagnosis
Condition: VWD - Von Willebrand's Disease
Intervention:
Sponsor: Assiut University
Not yet recruiting
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Impact of Von Willebrand Factor and Its Multimers on Angiogenesis
Condition: Von Willebrand Diseases
Intervention: Other: no intervention
Sponsor: Nantes University Hospital
Not yet recruiting
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The Efficacy and Safety of ZS802 in Chinese Hemophilia A Patients.
Condition: Hemophilia A
Intervention: Genetic: ZS802
Sponsor: Institute of Hematology & Blood Diseases Hospital
Not yet recruiting
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