Prospective Psychometric Evaluation Study of a Patient-reported Outcomes (PRO) Instrument for Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP]
Condition: Congenital Thrombotic Thrombocytopenic Purpura
Intervention: Other: No intervention
Sponsor: Baxalta now part of Shire
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
Not yet recruiting
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Research Study to Look at Side Effects During Regular Injection With Factor VIII Medicine Named Turoctocog Alfa for a 8 Weeks Period
Conditions: Congenital Bleeding Disorder; Haemophilia A
Intervention: Drug: turoctocog alfa
Sponsor: Novo Nordisk A/S
Recruiting
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Quality of Life in Patients With Congenital Afibrinogenemia
Condition: Afibrinogenemia, Congenital
Intervention: Other: Questionnaire quality of life
Sponsor: University Hospital, Geneva
Recruiting
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Impact Study on Cerecare Compression Garments in the Treatment of Ehlers-Danlos Syndromes
Condition: Eds, Unspecified Type
Intervention: Device: Cerecare compression garments
Sponsor: Cerecare
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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A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors
Conditions: Hemophilia A; Hemophilia B
Interventions: Drug: fitusiran; Drug: recombinant Factor VIIa and FEIBA
Sponsors: Alnylam Pharmaceuticals; Genzyme, a Sanofi Company
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
Sponsors: Alnylam Pharmaceuticals; Genzyme, a Sanofi Company
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
Not yet recruiting
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Study of Platelets Sialylation by Flow Cytometry for the Differential Diagnosis of ICT
Condition: Idiopathic Thrombocytopenic Purpura
Intervention: Other: non interventional study
Sponsor: Nantes University Hospital
Not yet recruiting
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Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg
Condition: Hemophilia A
Intervention: Biological: Valoctocogene Roxaparvovec
Sponsor: BioMarin Pharmaceutical
Recruiting
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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
Not yet recruiting
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Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients
Condition: Hemophilia A
Intervention: Biological: valoctocogene roxaparvovec
Sponsor: BioMarin Pharmaceutical
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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Joint Health Study
Condition: Hemophilia B
Intervention:
Sponsors: Bloodworks (Puget Sound Blood Center); CSL Behring
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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World Bleeding Disorders Registry
Conditions: Hemophilia A; Hemophilia B
Intervention:
Sponsor: World Federation of Hemophilia
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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Comparison of Ideal vs. Actual Weight Base Factor Dosing
Condition: Hemophilia A
Interventions: Other: Ideal Body Weight First; Other: Actual Body Weight First
Sponsors: Bloodworks (Puget Sound Blood Center); Seattle Children's Hospital; Oregon Health and Science University; Providence Health & Services
Recruiting
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Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants
Condition: Hemophilia A
Intervention: Drug: Emicizumab
Sponsor: Hoffmann-La Roche
Recruiting
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Quality of Life and Adjustment Among Siblings of Children and Adolescents With Severe Hemophilia
Condition: Severe Haemophilia
Intervention:
Sponsor: Assistance Publique Hopitaux De Marseille
Not yet 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
Not yet recruiting
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BABH Study: Efficacy and Safety of Bevacizumab on Severe Bleedings Associated With Hemorrhagic Hereditary Telangiectasia (HHT).
Condition: Hemorrhagic Hereditary Telangiectasia (HHT)
Interventions: Drug: Bevacizumab; Drug: sodium chloride 0.9%
Sponsor: Hospices Civils de Lyon
Recruiting
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HEAD-US SCORING SYSTEM: Assessment of the Real-world Impact of Ultrasound
Condition: Moderate and Severe Haemophilia
Intervention: Other: HEAD-US scoring system
Sponsors: Guy's and St Thomas' NHS Foundation Trust; Pfizer
Not yet recruiting
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Study Investigating Novoeight®/NovoEight® (Turoctocog Alfa) in Mexican Haemophilia A Patients
Conditions: Congenital Bleeding Disorder; Haemophilia A
Intervention: Drug: turoctocog alfa
Sponsor: Novo Nordisk A/S
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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Factor XIII in Major Burns Coagulation
Conditions: Burns; Coagulation Disorder; Factor XIII Deficiency; Cicatrization; Endothelial Dysfunction
Intervention: Diagnostic Test: Coagulation tests
Sponsor: Hospital Universitari Vall d'Hebron Research Institute
Not yet recruiting
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Low Von Willebrand in Ireland Cohort Study
Condition: Von Willebrand Factor, Deficiency
Intervention:
Sponsor: St. James's Hospital, Ireland
Recruiting
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Bleeding Risk Evaluation in Haemophilia Patients Under Antiplatelet Therapies
Condition: Hemophilia
Intervention: Other: Non interventional study
Sponsor: Nantes University Hospital
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 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; Affiliated Hospital, Logistics University of People's Armed Police Force; Beijing Anzhen Hospital; West China Second University Hospital of Sichuan University; Jinshan Hospital of Fudan University; Fudan University
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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A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study)
Condition: Hemophilia A With Inhibitors
Intervention: Biological: rFVIIIFc
Sponsors: Bioverativ Therapeutics Inc.; Swedish Orphan Biovitrum
Recruiting
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Response of Continuous Recombinant Antithrombin Infusion in Postcardiotomy ECMO Patients
Condition: Antithrombin Deficiency Type 2
Intervention: Drug: ATryn continuous infusion
Sponsors: Mayo Clinic; rEVO Biologics
Not yet 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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UK - EHL Outcomes Registry
Conditions: Hemophilia A; Hemophilia B
Intervention:
Sponsor: Royal Free Hospital NHS Foundation Trust
Recruiting
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A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B
Conditions: Haemophilia A; Haemophilia B
Interventions: Drug: ELOCTA; Drug: ALPROLIX
Sponsor: Swedish Orphan Biovitrum
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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Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A
Conditions: Congenital Bleeding Disorder; Haemophilia A
Intervention: Drug: turoctocog alfa pegol
Sponsor: Novo Nordisk A/S
Recruiting
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MRA With Feraheme in HHT
Condition: Hereditary Hemorrhagic Telangiectasia
Intervention: Procedure: Feraheme MRI/MRA
Sponsor: University of California, Los Angeles
Recruiting
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Immunmodulation in Patients With HHT
Condition: Hereditary Haemorrhagic Telangiectasia (HHT)
Intervention: Other: blood sample
Sponsor: University Hospital, Essen
Recruiting
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Fascial Therapy in Elbow Hemophilic Arthropathy
Condition: Hemophilia
Intervention: Other: Experimental group
Sponsor: Real Fundación Victoria Eugenia
Recruiting
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Treatment of Nasal Staphylococcus Aureus Colonization in Patients With HHT
Conditions: Hereditary Hemorrhagic Telangiectasia; Epistaxis
Interventions: Drug: Mupirocin; Other: Placebo
Sponsor: Hospital Italiano de Buenos Aires
Not yet recruiting
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A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children
Condition: Haemophilia
Intervention: Other: Observe the patient's condition and then change the regimen
Sponsor: Beijing Children's Hospital
Recruiting
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Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products
Condition: Hemophilia A
Interventions: Drug: efmoroctocog alfa; Drug: Factor VIII
Sponsor: Swedish Orphan Biovitrum
Recruiting
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BAX 111 rVWF in Pediatrics
Condition: Von Willebrand Disease
Interventions: Biological: von Willebrand factor (Recombinant); Biological: Antihemophilic Factor (Recombinant)
Sponsor: Baxalta now part of Shire
Recruiting
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rVWF IN PROPHYLAXIS
Condition: Von Willebrand Disease
Interventions: Biological: von Willebrand factor (Recombinant); Biological: Antihemophilic Factor (Recombinant)
Sponsor: Baxalta now part of Shire
Recruiting
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Graded TTCE for Post-Embolization PAVM Monitoring
Condition: Hereditary Hemorrhagic Telangiectasia (HHT)
Intervention: Other: Transthoracic Contrast Echocardiogram
Sponsor: University of Pennsylvania
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; Texas Children's Hospital; Shire
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
Not yet recruiting
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Evaluate Efficacy and Safety of Recombinant Factor VIII （rFVIII）Treatment of Severe or Moderately Severe Hemophilia A
Condition: Hemophilia A
Interventions: Drug: Recombinant Factor VIII (50 IU/kg); Drug: Recombinant Factor VIII (On-demand treatment)
Sponsor: Jiangsu Chia-tai Tianqing Pharmaceutical Co.,Ltd
Recruiting
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BAX 802 in CHA With Inhibitors
Condition: Hemophilia A
Intervention: Biological: Antihemophilic Factor (Recombinant), Porcine Sequence (BAX802)
Sponsor: Baxalta now part of Shire
Recruiting
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The Comorbidity of Benign Hypermobility Joint Syndrome and Functional Constipation in Children
Conditions: Benign Hypermobility Syndrome; Functional Constipation
Intervention:
Sponsor: Medical University of Warsaw
Recruiting
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Human Fibrinogen Concentrate in Pediatric Cardiac Surgery
Conditions: Hypofibrinogenemia; Afibrinogenemia; Bleeding Disorders
Interventions: Drug: RiaStAP; Drug: Saline
Sponsor: Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Recruiting
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Determinants and Consequences of the Transition to Adulthood Among Adolescents With Haemophilia
Condition: Haemophilia
Intervention: Other: Patient questionnaire
Sponsor: Assistance Publique Hopitaux De Marseille
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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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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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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Personalized Prophylactic Treatment With Advate® in Severe or Moderate Haemophilia A Patients
Conditions: Hemophilia A; Factor VIII
Intervention: Drug: Recombinant VIII factor
Sponsor: Hospital Universitario La Fe
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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Ultrasonography in Hemophilic Joint Disease and Serum Markers
Conditions: Hemophilia A; Hemophilia B; Hemarthroses
Intervention: Other: ultrasound
Sponsors: Northwell Health; National Cancer Institute (NCI); University of California, San Diego
Recruiting
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Weight-based Dosing in Hemophilia A
Condition: Hemophilia
Intervention: Drug: rFVIII
Sponsor: Craig Seaman
Recruiting
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Post-Marketing Non-Interventional Safety Evaluation of Obizur in the Treatment of Bleeding Episodes for Patients With Acquired Hemophilia A
Condition: Acquired Hemophilia A
Intervention: Biological: OBIZUR
Sponsors: Baxalta now part of Shire; Baxalta Innovations GmbH, now part of Shire
Recruiting
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Minimize Menorrhagia in Women With Type 1 Von Willebrand Disease
Condition: Type 1 Von Willebrand Disease
Interventions: Drug: recombinant von Willebrand factor; Drug: tranexamic acid
Sponsors: Margaret Ragni; University of North Carolina; Carnegie Mellon University; Duke University
Not yet recruiting
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Investigating a Von Willebrand Factor (VWF) Functional Screening Assay for Assigning the Phenotypic Variants of Von Willebrand Disease (VWD)
Condition: Von Willebrands Disease
Intervention:
Sponsors: Jonathan Roberts; Blood Center of Wisconsin
Recruiting
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ATHN 2: Factor Switching Study
Condition: Hemophilia
Interventions: Biological: Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013; Biological: Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013
Sponsors: American Thrombosis and Hemostasis Network; Shire; CSL Behring; Bioverativ Therapeutics Inc.; Bayer
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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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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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 Fanhdi
Sponsor: Instituto Grifols, S.A.
Recruiting
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A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa
Conditions: Hemophilia A With Inhibitors; Hemophilia B With Inhibitors
Intervention: Biological: Coagulation FVIIa (Recombinant)
Sponsors: LFB USA, Inc.; Laboratoire français de Fractionnement et de Biotechnologies
Recruiting
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A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B
Conditions: Hemophilia A; Hemophilia B
Intervention: Drug: MOD-5014
Sponsor: Opko Biologics
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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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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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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To Investigate Safety and Efficacy of NovoEight® (rFVIII) During Long-term Treatment of Haemophilia A in Japan
Conditions: Congenital Bleeding Disorder; Haemophilia A
Intervention: Drug: turoctocog alfa
Sponsor: Novo Nordisk A/S
Recruiting
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Use of Proteomics for the Diagnosis of a Platelet-related Bleeding Disorder
Condition: Inherited Platelet Disorders
Intervention: Other: Blood collection for platelet protein samples
Sponsors: Hadassah Medical Organization; Hebrew University of Jerusalem
Not yet 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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An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
Conditions: Hemophilia A; Severe Hemophilia A
Intervention: Biological: rVIII‑SingleChain
Sponsor: CSL Behring
Recruiting
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Global Hemostatic Methods in Hemophilia and Von Willebrand's Disease
Conditions: Hemophilia A; Hemophilia B; Von Willebrand's Disease
Intervention:
Sponsors: Karolinska University Hospital; The Swedish Society of Medicine; Karolinska Institutet
Recruiting
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ADVATE Hemophilia A Outcome Database (AHEAD)
Condition: Hemophilia A
Intervention: Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Sponsor: Baxalta now part of Shire
Recruiting
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Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990
Condition: Wiskott-Aldrich Syndrome
Intervention:
Sponsors: National Institute of Allergy and Infectious Diseases (NIAID); Primary Immune Deficiency Treatment Consortium; Rare Diseases Clinical Research Network
Recruiting
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A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
Condition: Hemophilia B
Intervention: Biological: rIX-FP
Sponsor: CSL Behring
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 New Mexico; University of California, San Francisco; Barrow Neurological Institute; Angioma Alliance; National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting
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Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept
Condition: Hereditary Hemorrhagic Telangiectasia
Interventions: Drug: Topical timolol maleate; Drug: placebo saline drops
Sponsors: St. Michael's Hospital, Toronto; University of California, San Francisco; The Hospital for Sick Children; University of Toronto; Sunnybrook Health Sciences Centre; Ryerson University; National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting
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Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A.
Conditions: Congenital Bleeding Disorder; Haemophilia A
Intervention: Drug: turoctocog alfa pegol
Sponsor: Novo Nordisk A/S
Recruiting
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Thrombocytopathy in Gaucher Disease Patients
Conditions: Gaucher Disease; Thrombocytopathy
Intervention:
Sponsors: Rabin Medical Center; Genzyme, a Sanofi Company
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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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 Switzerland; Baxalta Innovations GmbH, Wien, Austria
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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Registry for Vascular Anomalies Associated With Coagulopathy
Conditions: Multifocal Lymphangioendotheliomatosis With Thrombocytopenia; Cutaneovisceral Angiomatosis With Thrombocytopenia; Vascular Anomaly With Thrombocytopenia; Hemangiomas
Intervention: Other: no intervention
Sponsor: Medical College of Wisconsin
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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Molecular Studies on Hereditary Haemorrhagic Telangiectasia Families
Condition: Telangiectasia, Hereditary Hemorrhagic
Intervention:
Sponsors: Imperial College London; British Heart Foundation
Recruiting
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Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome
Conditions: Albinism; Intestinal Disease; Kidney Disease; Myocardial Disease; Pulmonary Fibrosis
Intervention:
Sponsor: National Human Genome Research Institute (NHGRI)
Recruiting
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