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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The Role of Standardized Bleeding Score in Diagnosis of Pediatric Von Willebrand's Disease
Condition: Von Willebrand's Diseased Children, Bleeding Questionnaire is Done
Intervention: Diagnostic Test: Bleeding score ( questionnaire )
Sponsor: Sohag University
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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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
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
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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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: Unity Health 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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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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TPO-Mimetic Use in Children for Hemotopoietic 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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World Bleeding Disorders Registry
Conditions: Hemophilia A; Hemophilia B
Intervention:
Sponsor: World Federation of Hemophilia
Recruiting
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Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding
Condition: Hemophilia A
Intervention: Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)
Sponsor: Bayer
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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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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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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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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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.
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
Not yet 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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Treatment of Hereditary Hemorrhagic Telangiectasia of the Nasal Mucosa by Intranasal Bevacizumab : Search for Effective Dose
Condition: Telangiectasia, Hereditary Hemorrhagic
Intervention: Drug: bevacuzimab spray
Sponsor: University Hospital, Caen
Recruiting
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Pazopanib Effects on Bleeding in Hereditary Hemorrhagic Telangiectasia
Conditions: Hereditary Hemorrhagic Telangiectasia; Epistaxis; Anemia; Nosebleed; HHT
Interventions: Drug: Pazopanib; Drug: Placebo oral capsule
Sponsors: Cure HHT; United States Department of Defense
Not yet 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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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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Assess Safety and Efficacy of VAD044 in HHT Patients
Condition: Hereditary Hemorrhagic Telangiectasia (HHT)
Intervention: Drug: VAD044
Sponsor: Vaderis Therapeutics AG
Not yet 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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A Gene Transfer Study for Hemophilia A
Condition: Hemophilia A
Intervention: Genetic: SPK-8011
Sponsor: Spark Therapeutics
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
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
Intervention:
Sponsor: Institute of Hematology & Blood Diseases Hospital
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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Safety and Efficacy in a ReAl-Life Study in Patients With Haemophilia Treated wIth NovoEight® for Surgery
Condition: Hemophilia
Intervention: Other: no intervention
Sponsor: Nantes University Hospital
Recruiting
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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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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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Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
Condition: Hemophilia A
Intervention: Drug: Gene therapy
Sponsors: Christian Medical College, Vellore, India; Centre for Stem Cell Research, (unit of inStem), CMC, Vellore
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
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: Washington Institute for Coagulation; Genentech, Inc.
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.
Not yet 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
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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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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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 TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Condition: Congenital Thrombotic Thrombocytopenic Purpura
Intervention: Biological: TAK-755
Sponsors: Takeda; Takeda Development Center Americas, Inc.; Shire
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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Von Willebrand Factor in Pregnancy (VIP) Study
Condition: Von Willebrand Diseases
Interventions: Other: Use of a postpartum diary and additional blood draws; Drug: VWF replacement therapy with Wilate; Drug: Tranexamic acid; Other: Use of a postpartum diary and additional blood draws.
Sponsors: Bloodworks; Mary M. Gooley Hemophilia Center; Ergomed; Octapharma
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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Pain and Joint Status With Psychosocial Factors in Patients With Hemophilia
Condition: Hemophilia Arthropathy
Intervention: Other: Observational group
Sponsor: Investigación en Hemofilia y Fisioterapia
Not yet recruiting
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PyROphosPHate Supplementation to Fight ECtopIc Calcification in PseudoXanthoma Elasticum
Condition: Pseudoxanthoma Elasticum
Intervention: Drug: study treatment PPI
Sponsor: Centre Hospitalier Universitaire de Nice
Not yet 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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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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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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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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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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ATHN 11: Liver Transplantation Outcomes Study
Conditions: Hemophilia A and B; Liver Transplantation
Intervention:
Sponsors: American Thrombosis and Hemostasis Network; BioMarin Pharmaceutical
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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A Study of the Burden of Illness and Treatment Patterns in Teenagers and Adults With Hereditary Angioedema
Condition: Hereditary Angioedema
Intervention:
Sponsor: Takeda
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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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 Fudan University; Fudan University
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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Accessibility of Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies
Conditions: Health Services Accessibility; Blood Coagulation Factor Deficiencies
Intervention:
Sponsors: RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône; Hospices Civils de Lyon
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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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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Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery
Condition: Congenital Antithrombin Deficiency
Intervention: Drug: Atenativ
Sponsor: Octapharma
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 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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ATHN 8: Previously Untreated Patients (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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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
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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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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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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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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Phase III Expansion Trial for Determining the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A
Condition: Hemophilia A
Intervention: Drug: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
Sponsors: Zhengzhou Gensciences Inc; Jiangsu Gensciences lnc.
Recruiting
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Study of TAK-672 in Participants With Acquired Hemophilia A
Condition: Acquired Hemophilia A
Intervention: Biological: TAK-672
Sponsor: Takeda
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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Rofecoxib Efficacy and Safety Evaluation Trial in Hemophilic Arthropathy
Condition: Hemophilic Arthropathy
Interventions: Drug: TRM-201 (Rofecoxib); Drug: Placebo
Sponsor: Tremeau Pharmceuticals, Inc.
Recruiting
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A Safety Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor
Condition: Hemophilia
Intervention: Drug: STSP-0601 for Injection
Sponsor: Staidson (Beijing) Biopharmaceuticals Co., Ltd
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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A Study of TAK-660 in Surgical Procedures for People With Hemophilia A.
Condition: Hemophilia A
Intervention: Biological: PEGylated Recombinant Factor VIII
Sponsor: Takeda
Recruiting
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