A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors
Conditions: Congenital Bleeding Disorder; Haemophilia A With Inhibitors; Haemophilia B With Inhibitors
Interventions: Drug: Concizumab; Drug: Eptacog alfa
Sponsor: Novo Nordisk A/S
Not yet recruiting - verified June 2017
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A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors
Conditions: Haemostasis; Haemophilia A
Interventions: Drug: Concizumab; Drug: Turoctocog alfa
Sponsor: Novo Nordisk A/S
Not yet recruiting - verified June 2017
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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 - verified June 2017
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A Multicenter, Post-Marketing Registry Study of COAGADEX® in the Peri-operative Management of Patients With Moderate or Severe Hereditary Factor X Deficiency Undergoing Major Surgery
Condition: Factor 10 Deficiency
Intervention: Drug: Major surgery
Sponsor: Bio Products Laboratory
Not yet recruiting - verified May 2017
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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 - verified May 2017
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A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
Condition: Hemophilia A
Intervention: Drug: Emicizumab
Sponsor: Hoffmann-La Roche
Not yet recruiting - verified June 2017
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Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia
Conditions: Hemophilia A; Hemophilia B
Intervention: Other: Non-treatment, seroprevalence
Sponsor: Baxalta US Inc.
Recruiting - verified June 2017
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A Prospective Study on the Incidence and Related Risk Factors of Infantile Hemangioma in China
Conditions: Infantile Hemangioma; Capillary Hemangioma; Capillary Hemangiomas; Hemangioma, Capillary Infantile; Strawberry Hemangioma
Intervention:
Sponsor: Zhujiang Hospital
Recruiting - verified June 2017
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Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients
Condition: Hemophilia B
Interventions: Biological: ISU304/CB2679d; Biological: BeneFix
Sponsors: ISU Abxis Co., Ltd.; Catalyst Biosciences
Recruiting - verified June 2017
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ADYNOVATE Drug Use-Results Survey
Condition: Hemophilia A
Intervention: Biological: ADYNOVATE
Sponsors: Baxalta US Inc.; Baxalta Japan Limited
Recruiting - verified May 2017
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Hemophilia Mobile App Usability Pilot
Condition: Hemophilia
Intervention: Device: SureSource Engage application
Sponsors: Healthcare Innovation Technology Lab; Bayer
Recruiting - verified May 2017
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Bleeding Risk Evaluation in Haemophilia Patients Under Antiplatelet Therapies
Condition: Hemophilia
Intervention: Other: Non interventional study
Sponsor: Nantes University Hospital
Not yet recruiting - verified May 2017
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An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors
Condition: Hemophilia A
Intervention: Biological: Emicizumab
Sponsor: Genentech, Inc.
Available - verified June 2017
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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, Capital Medical University; West China Second University Hospital of Sichuan University; Jinshan Hospital of Fudan University; Fudan University
Recruiting - verified April 2017
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DDAVP vs. Exercise
Condition: Children With Mild Hemophilia A
Interventions: Drug: DDAVP intervention; Behavioral: Exercise Intervention
Sponsors: The Hospital for Sick Children; Canadian Hemophilia Society; St. Michael's Hospital, Toronto
Not yet recruiting - verified April 2017
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Efficacy and Safety of a 0.1% Tacrolimus Nasal Ointment as a Treatment for Epistaxis in Hemorrhagic Hereditary Telangiectasia (HHT)
Condition: Hemorrhagic Hereditary Telangiectasia (HHT)
Interventions: Drug: Protopic® (Tacrolimus) 0.1% ointment; Drug: Placebo
Sponsor: Hospices Civils de Lyon
Not yet recruiting - verified May 2017
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Study of rFVIIIFc for ITI in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies
Condition: Hemophilia A
Intervention: Biological: Recombinant coagulation factor (rFVIIIFc)
Sponsors: Swedish Orphan Biovitrum; Bioverativ Therapeutics Inc.
Not yet recruiting - verified March 2017
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A Trial Comparing Nonacog Beta Pegol (N9-GP) and ALPROLIX® in Patients With Haemophilia B
Conditions: Congenital Bleeding Disorder; Haemophilia B
Interventions: Drug: N9-GP; Drug: ALPROLIX®
Sponsor: Novo Nordisk A/S
Recruiting - verified June 2017
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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 - verified April 2017
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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 - verified March 2017
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Gingival Bleeding and Von Willebrand Disease Typ 2 and 3
Conditions: Gingival Bleeding; Von Willebrand Diseases
Intervention:
Sponsor: Goethe University
Recruiting - verified March 2017
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Alphanate in Immune Tolerance Induction Therapy
Condition: Hemophilia A, Congenital
Intervention: Biological: Alphanate
Sponsor: Grifols Therapeutics Inc.
Recruiting - verified May 2017
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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
Condition: Hemophilia A
Intervention: Biological: rFVIIIFc
Sponsor: Bioverativ Therapeutics Inc.
Not yet recruiting - verified March 2017
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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 - verified May 2017
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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
Not yet recruiting - verified June 2017
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Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A
Condition: Hemophilia A
Intervention: Biological: SB-525
Sponsor: Sangamo Therapeutics
Recruiting - verified June 2017
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Non Substitutive Strategies to Improve Haemophilia Care in Developing Countries. Experience in Ivory Coast.
Conditions: Haemophilia; Developing Countries
Interventions: Other: Education; Diagnostic Test: Improve biological diagnosis and follow-up of haemophilia; Drug: Implement haemostatic non substitutive care of haemophilia; Other: Implement non haemostatic care of haemophilia
Sponsors: Cliniques universitaires Saint-Luc- Université Catholique de Louvain; World Federation of Hemophilia
Recruiting - verified February 2017
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FIBrinogen REplenishment in Surgery
Condition: Deficiency; Fibrinogen, Acquired
Interventions: Drug: Octafibrin; Drug: Cryoprecipitate
Sponsor: University Health Network, Toronto
Recruiting - verified February 2017
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A Gene Transfer Study for Hemophilia A
Condition: Hemophilia A
Intervention: Genetic: SPK-8011
Sponsor: Spark Therapeutics
Recruiting - verified April 2017
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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 - verified June 2017
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A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
Conditions: Wiskott-Aldrich Syndrome; Hematopoietic Stem Cell Transplantation; Graft Failure
Interventions: Biological: G-CSF for Conditioning before HSCT.; Biological: Plerixafor for Conditioning before HSCT.
Sponsor: Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Recruiting - verified January 2017
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Fascial Therapy in Elbow Hemophilic Arthropathy
Condition: Hemophilia
Intervention: Other: Experimental group
Sponsor: Real Fundación Victoria Eugenia
Not yet recruiting - verified January 2017
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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 - verified March 2017
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Immunmodulation in Patients With HHT
Condition: Hereditary Haemorrhagic Telangiectasia (HHT)
Intervention: Other: blood sample
Sponsor: University Hospital, Essen
Recruiting - verified December 2016
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UK - EHL Outcomes Registry
Conditions: Hemophilia A; Hemophilia B
Intervention:
Sponsor: Royal Free Hospital NHS Foundation Trust
Not yet recruiting - verified October 2016
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Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®
Conditions: Hemophilia A; Hemophilia; Factor VIII Deficiency
Intervention: Drug: Octocog Alfa
Sponsors: Spanish Society of Thrombosis and Haemostasis; Baxalta US Inc.; Unidad de Ensayos Clínicos e Investigación Clínica. Hospital La Paz; Instituto de Investigación Hospital Universitario La Paz
Not yet recruiting - verified December 2016
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A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A
Condition: Hemophilia A
Intervention: Drug: Emicizumab
Sponsors: Hoffmann-La Roche; Chugai Pharmaceutical
Recruiting - verified May 2017
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Individualizing Hemophilia Bypassing Agent Therapy Utilizing Thromboelastography
Condition: Hemophilia A With Inhibitors
Intervention: Other: Thromboelastography-Guided Treatment Regimen
Sponsor: Children's Hospital Los Angeles
Not yet recruiting - verified December 2016
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Gene Therapy for Haemophilia A.
Condition: Hemophilia A
Intervention: Biological: AAV2/8-HLP-FVIII-V3
Sponsors: University College, London; Medical Research Council
Recruiting - verified June 2017
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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 - verified February 2017
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Study With Atezolizumab Plus Bevacizumab in Patients With Chemotherapy Resistant, MSI-like, Colorectal Cancer
Conditions: MSI; ColoRectal Cancer; Chemotherapy; Resistance, APC
Interventions: Drug: Atezolizumab; Drug: Bevacizumab
Sponsors: Vall d'Hebron Institute of Oncology; European Organisation for Research and Treatment of Cancer - EORTC
Not yet recruiting - verified May 2017
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rVWF IN PROPHYLAXIS
Condition: Von Willebrand Disease
Interventions: Biological: von Willebrand factor (Recombinant); Biological: Antihemophilic Factor (Recombinant)
Sponsor: Baxalta US Inc.
Not yet recruiting - verified November 2016
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PF-06741086 Multiple Dose Study in Severe Hemophilia
Condition: Hemophilia A or B
Interventions: Biological: PF-06741086; Biological: PF-06741086; Biological: PF-06741086; Biological: PF-06741086
Sponsor: Pfizer
Recruiting - verified June 2017
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Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Condition: Hemophilia B
Intervention:
Sponsor: Dimension Therapeutics
Recruiting - verified January 2017
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Efficacy and Safety of Turoctocog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A
Conditions: Congenital Bleeding Disorder; Haemophilia A
Interventions: Drug: turoctocog alfa; Drug: turoctocog alfa
Sponsor: Novo Nordisk A/S
Recruiting - verified May 2017
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Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
Condition: Hemophilia A
Intervention:
Sponsor: Octapharma
Recruiting - verified November 2016
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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 US Inc.
Not yet recruiting - verified October 2016
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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 - verified October 2016
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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; Baxalta US Inc.
Not yet recruiting - verified October 2016
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Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
Condition: Severe Hemophilia A
Intervention: Drug: Wilate
Sponsor: Octapharma
Recruiting - verified March 2017
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Exit Interviews to Assess Impact of Infusion Frequency in Hemophilia A
Condition: Hemophilia A
Intervention: Biological: BAY94-9027
Sponsor: Bayer
Not yet recruiting - verified June 2017
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Drug Use Investigation of Kovaltry in Hemophilia A Patients
Condition: Hemophilia A
Intervention: Drug: Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973)
Sponsor: Bayer
Recruiting - verified June 2017
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RIXUBIS Drug Use-Result Survey (Japan)
Condition: Hemophilia B
Intervention: Biological: RIXUBIS
Sponsor: Baxalta US Inc.
Recruiting - verified May 2017
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Efficacy and Safety Study of SCT800 for On-demand Treatment With in Patients With Hemophilia A
Condition: Hemophilia A
Intervention: Biological: SCT800
Sponsor: Sinocelltech Ltd.
Not yet recruiting - verified September 2016
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Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea
Condition: Hemophilia B
Intervention: Biological: RIXUBIS
Sponsor: Baxalta US Inc.
Recruiting - verified December 2016
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German Pediatric Hemophilia Research Database
Conditions: Hemophilia A; Hemophilia B; Children; Drug Specific Antibodies
Intervention: Other: documentation only
Sponsors: Ludwig-Maximilians - University of Munich; Pediatric Committee of the German Thrombosis and Hemostasis Research Society
Recruiting - verified January 2017
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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 - verified October 2016
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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 - verified December 2016
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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 - verified November 2016
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Octreotide in Patients With GI Bleeding Due to Rendu-Osler-Weber
Conditions: Hereditary Hemorrhagic Telangiectasia; Gastrointestinal Hemorrhage; Anemia
Intervention: Drug: Octreotide LAR
Sponsors: Radboud University; St. Antonius Hospital
Recruiting - verified August 2016
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Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A
Condition: Hemophilia A
Interventions: Biological: SCT800; Biological: Xyntha
Sponsor: Sinocelltech Ltd.
Not yet recruiting - verified August 2016
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BAX 802 in CHA With Inhibitors
Condition: Hemophilia A
Intervention: Biological: Antihemophilic Factor (Recombinant), Porcine Sequence (BAX802)
Sponsor: Baxalta US Inc.
Recruiting - verified April 2017
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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 - verified June 2016
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A Single-dose, Dose-escalation Study of a Long-acting MOD-5014 in Healthy Adult Male
Condition: Hemophilia A or B With Inhibitors
Interventions: Biological: MOD-5014; Other: MOD-5014 Placebo
Sponsor: Opko Biologics
Not yet recruiting - verified August 2016
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Effectiveness of Psychological Interventions in Haemophilia
Condition: Haemophilia
Interventions: Behavioral: Hypnosis; Behavioral: Cognitive-Behavioral Therapy
Sponsors: University of Minho; Hospital Sao Joao
Not yet recruiting - verified August 2016
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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 - verified August 2016
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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 - verified March 2017
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Molecular and Clinical Profile of Von Willebrand Disease in Spain
Condition: Von Willebrand Disease
Intervention: Genetic: VWF gene analysis
Sponsors: Spanish Society of Thrombosis and Haemostasis; Francisco Vidal Pérez; Maria Fernanda López Fernández; Almudena Pérez Rodríguez; Irene Corrales Insa; Ana Rosa Cid Haro
Not yet recruiting - verified August 2016
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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 - verified April 2017
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Effectiveness of the Myofascial Therapy in the Hemophilic Arthropathy of Ankle
Condition: Haemophilia
Intervention: Other: Physiotherapy sessions
Sponsor: Real Fundación Victoria Eugenia
Not yet recruiting - verified July 2016
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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 - verified May 2017
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Does the Thrombin Generation Test Performed During the Pharmacokinetic Profile of the Substitutive Factor VIII Bring Benefits to the Personalized Treatment of Pediatric Patients and Adult Hemophilia A Patients Under Prophylaxis ?
Condition: Hemophilia
Interventions: Device: Chronometric method; Device: Chromogenic method; Device: Thrombin generation test (TGT)
Sponsor: Brugmann University Hospital
Not yet recruiting - verified June 2016
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World Bleeding Disorders Registry
Condition: Hemophilia A and B
Intervention:
Sponsor: World Federation of Hemophilia
Not yet recruiting - verified May 2016
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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 - verified February 2017
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Immunosuppression During Penetrating Keratoplasty, Using a Subconjunctival Implant Releasing Dexamethasone : Tolerance and Safety Pilot Study
Conditions: Keratoconus; Fuchs' Endothelial Dystrophy; Congenital Hereditary Stromal Dystrophy of the Cornea
Intervention: Drug: Dexamethasone implant OZURDEX
Sponsor: Centre Hospitalier Universitaire de Saint Etienne
Recruiting - verified January 2017
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The Hemophilia Ultrasound Project
Conditions: Hemophilia A; Hemophilia B
Interventions: Other: Ankle and Knee Ultrasound Joint Assessment; Other: Ankle and Knee Magnetic Resonance Imaging
Sponsors: University of Miami; Baxalta US Inc.
Recruiting - verified August 2016
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The Proteins of the Contact Activation System
Conditions: Factor XI Deficiency; Factor XII Deficiency
Intervention: Other: Global Haemostasis assays
Sponsor: Hospices Civils de Lyon
Recruiting - verified May 2016
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Factor Product Utilization and Health Outcomes in Patients With Hemophilia
Conditions: Hemophilia A, Congenital; Hemophilia B, Congenital
Intervention:
Sponsors: University of British Columbia; Biogen
Recruiting - verified June 2016
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Topical Anesthesia for Closed PKP vs Retrobulbar Anesthesia for Open-sky PKP
Conditions: Corneal Opacity; Keratitis, Herpetic; Corneal Ulcer; Corneal Dystrophies, Hereditary
Interventions: Procedure: closed PKP under topical anesthesia; Procedure: open-sky PKP under retrobulbar anesthesia; Drug: Anti-Rejection Agents; Drug: Anti-Inflammatory Agents
Sponsor: Wenzhou Medical University
Recruiting - verified July 2016
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A Progressive Resistance Training Program in Patients With Haemophilia
Condition: Haemophilia
Interventions: Other: Progressive resistance training; Other: Normal daily activities
Sponsors: University of Valencia; Baxalta US Inc.
Not yet recruiting - verified October 2016
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Low Von Willebrand in Ireland Cohort Study
Condition: Von Willebrand Factor, Deficiency
Intervention:
Sponsor: St. James's Hospital, Ireland
Recruiting - verified May 2017
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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
Recruiting - verified September 2016
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FEIBA Reconstitution Volume Reduction and Faster Infusion Study
Condition: Hemophilia A or B With Inhibitors
Intervention: Biological: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)
Sponsor: Baxalta US Inc.
Not yet recruiting - verified October 2016
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Improved Factor VIII Inhibitor Evaluation
Conditions: Hemophilia; Hemophilia A
Intervention:
Sponsors: Boston VA Research Institute, Inc.; Baxalta US Inc.; Emory University; University of Michigan
Not yet recruiting - verified March 2016
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Symptoms and Treatment Results in Hereditary Hemorrhagic Telangiectasia
Condition: Hereditary Haemorrhagic Telangiectasia (HHT)
Intervention: Other: questionnaire
Sponsor: University Hospital, Essen
Recruiting - verified December 2016
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Efficacy & Safety of REFACTO AF in Prophylaxis Treatment
Condition: Severe Hemophilia A
Intervention:
Sponsor: Pfizer
Not yet recruiting - verified June 2017
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Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B
Condition: Hemophilia B
Intervention: Biological: SB-FIX
Sponsor: Sangamo Therapeutics
Recruiting - verified January 2017
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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 - verified August 2016
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BAX 855 Previously Untreated Patient (PUP)
Condition: Hemophilia A
Intervention: Biological: PEGylated Recombinant Factor VIII
Sponsor: Baxalta US Inc.
Recruiting - verified April 2017
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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
Sponsor: Baxalta US Inc.
Recruiting - verified August 2016
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Weight-based Dosing in Hemophilia A
Condition: Hemophilia
Intervention: Drug: rFVIII
Sponsor: University of Pittsburgh
Recruiting - verified May 2017
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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: University of Pittsburgh; University of North Carolina; Carnegie Mellon University; Duke University
Not yet recruiting - verified June 2017
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Biomarker for Patient With Alport Disease or Highly Suspected for Alport Disease
Conditions: Nephritis, Hereditary; Alport Syndrome; Alport Syndrome, Autosomal Dominant; Alport Syndrome, Autosomal Recessive; Hematuria-Nephropathy-Deafness Syndrome
Intervention:
Sponsors: University of Rostock; Centogene AG Rostock
Recruiting - verified September 2016
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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 - verified January 2017
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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 - verified July 2016
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Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)
Conditions: Lymphoma; Childhood Lymphoma; Solid Tumor; Childhood Solid Tumor; Neuroblastoma; Ewing Sarcoma; Hodgkin Lymphoma; Non-Hodgkin Lymphoma; Rhabdomyosarcoma; Soft Tissue Sarcoma; Medulloblastoma; Germ Cell Tumor; Wilms Tumor; Brain Neoplasms; Medulloblastoma, Childhood; Neuroectodermal Tumors, Primitive
Interventions: Drug: Genistein; Drug: Placebo
Sponsor: University of Virginia
Recruiting - verified June 2017
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Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A."
Condition: Hemophilia A
Interventions: Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222); Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Sponsor: Bayer
Recruiting - verified June 2017
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Vasculitis Pregnancy Registry
Conditions: Vasculitis; Behcet's Disease; CNS Vasculitis; Cryoglobulinemic Vasculitis; Eosinophilic Granulomatosis With Polyangiitis (EGPA); Churg-Strauss Syndrome (CSS); Granulomatosis With Polyangiitis (GPA); Wegener's Granulomatosis; IgA Vasculitis; Henoch-Schoenlein Purpura (HSP); Microscopic Polyangiitis (MPA); Polyarteritis Nodosa (PAN); Takayasu Arteritis (TAK); Urticarial Vasculitis; Systemic Vasculitis
Intervention: Other: Online questionnaires
Sponsors: University of Pennsylvania; Duke University; University of South Florida
Recruiting - verified April 2017
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Validation of a Specific Measure for Assessment of Compliance With Treatment in Patients With Hemophilia
Condition: Haemophilia
Intervention: Other: Patients with haemophilia
Sponsors: Real Fundación Victoria Eugenia; Medtep Inc.
Recruiting - verified October 2015
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Individualizing Hemophilia Prophylaxis Using Thromboelastography
Condition: Hemophilia A
Intervention: Other: Thromboelastography/Thromboelastometry-Guided Treatment Regimen
Sponsor: Children's Hospital Los Angeles
Recruiting - verified July 2016
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A Single Escalating Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors
Conditions: Hemophilia A; Hemophilia B
Intervention: Drug: BAY1093884
Sponsor: Bayer
Recruiting - verified June 2017
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Study of Voncento® in Subjects With Von Willebrand Disease
Condition: Von Willebrand Disease
Intervention: Biological: Voncento
Sponsor: CSL Behring
Recruiting - verified June 2017
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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; Bioverativ (Biogen); Shire (Baxalta); CSL Behring
Recruiting - verified June 2017
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Basic Study of Combination Therapy Based on APCC-induced FVllI Activation in Hemophilia A Patients With Inhibitors
Condition: Hemophilia A With Inhibitor
Intervention:
Sponsors: Nara Medical University; Baxalta US Inc.
Not yet recruiting - verified September 2015
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An Open-label Extension Study of an Investigational Drug, ALN-AT3SC, in Patients With Moderate or Severe Hemophilia A or B
Conditions: Hemophilia A; Hemophilia B
Intervention: Drug: ALN-AT3SC
Sponsor: Alnylam Pharmaceuticals
Recruiting - verified July 2016
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A Study of the Safety and Efficacy of Coagulation Factor VIIa (Recombinant) LR769 for the Prevention of Excessive Bleeding in Congenital Hemophilia A or B Patients With Inhibitors to Factor VIII or IX Undergoing Elective Surgery or Other Invasive Procedure
Condition: Hemophilia
Intervention: Biological: Coagulation Factor VIIa (Recombinant)
Sponsors: LFB USA, Inc.; Laboratoire français de Fractionnement et de Biotechnologies
Recruiting - verified January 2017
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Swiss Hemophilia Registry
Condition: Hemophilia and Other Severe Bleeding Disorders
Intervention: Other: Registry
Sponsor: Swiss Hemophilia Network
Recruiting - verified March 2017
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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 - verified August 2016
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Natural History Study of Factor IX Treatment and Complications
Condition: Factor IX Deficiency
Intervention: Other: Standard care with blood and urine sample collection
Sponsors: Skane University Hospital; Indiana Hemophilia and Thrombosis Center; University of Bonn; Biogen; Swedish Orphan Biovitrum
Recruiting - verified May 2017
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Efficacy of a Timolol Nasal Spray as a Treatment for Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) - (TEMPO)
Condition: Hemorrhagic Hereditary Telangiectasia (HHT)
Interventions: Drug: Timolol nasal spray; Drug: Placebo nasal spray
Sponsor: Hospices Civils de Lyon
Recruiting - verified November 2016
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"Incidence and Consequences of Disorders of Glycosylation in Patients With Conotruncal and Septal Heart Defects"
Conditions: Congenital Heart Diseases; Conotruncal Defects; Congenital Disorder of Glycosylation; Antithrombin III Deficiency
Intervention: Other: none intervention
Sponsors: Hospital Universitari Vall d'Hebron Research Institute; Universidad de Murcia
Recruiting - verified March 2017
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A Gene Therapy Study for Hemophilia B
Condition: Hemophilia B
Intervention: Genetic: SPK-9001
Sponsors: Spark Therapeutics; Pfizer
Recruiting - verified February 2017
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Study of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) for On-demand Treatment of Bleeding Episodes in Patients With Hemophilia A or B With Inhibitors
Conditions: Hemophilia A With Inhibitors; Hemophilia B With Inhibitors
Interventions: Drug: CSL689; Drug: Eptacog alfa (activated)
Sponsor: CSL Behring
Recruiting - verified June 2017
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Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients
Condition: Hemophilia A
Intervention: Drug: Plasma-derived FVIII/VWF concentrate
Sponsors: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico; Sintesi Research Srl
Recruiting - verified June 2015
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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 - verified September 2015
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Cardiopulmonary Exercise Testing to Evaluate Pulmonary AVMs With and Without Airflow Obstruction
Conditions: Pulmonary Arteriovenous Malformations; Hereditary Hemorrhagic Telangiectasia; Asthma; COPD
Intervention: Other: Cardiopulmonary exercise test
Sponsor: Imperial College London
Not yet recruiting - verified April 2015
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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 - verified March 2017
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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 - verified January 2017
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Myofascial Therapy in Patients With Hemophilic Arthropathy
Condition: Hemophilia
Intervention: Other: Myofascial therapy
Sponsor: Universidad Católica San Antonio de Murcia
Recruiting - verified September 2015
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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 - verified September 2016
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Study to Assess the Efficacy, Safety and Pharmacokinetic of Octafibrin in Paediatric Subjects With Fibrinogen Deficiency
Condition: Congenital Fibrinogen Deficiency
Intervention: Biological: Octafibrin
Sponsor: Octapharma
Recruiting - verified May 2016
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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 - verified April 2017
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Patient Functioning and Well-being, Economic, and Clinical Impact of Hemophilia A and Its Treatment
Condition: Hemophilia A
Intervention: Drug: Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)
Sponsor: Bayer
Recruiting - verified June 2017
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European Alport Therapy Registry - European Initiative Towards Delaying Renal Failure in Alport Syndrome
Conditions: Alport Syndrome; Hereditary Kidney Disease; Pediatric Kidney Disease; Thin Basement Membrane Disease; Familial Benign Hematuria
Interventions: Drug: ACE-inhibitor; Drug: AT1-inhibitor; Drug: HMG-Coenzyme inhibitor (statin); Drug: Spironolactone; Drug: Paricalcitol
Sponsors: University Hospital Goettingen; Gesellschaft für Pädiatrische Nephrologie; Deutsche Gesellschaft für Nephrologie; Alport Selbsthilfe e.V.; Association pour l'Information et la Recherche sur les Maladies Rénales Génétiques (AIRG); KfH Foundation Preventive Medicine
Recruiting - verified October 2016
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A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis
Condition: Hermansky Pudlak Syndrome
Interventions: Other: Pulmonary function test; Other: Chest CT; Other: Sample collection
Sponsors: Vanderbilt University; University of South Florida; National Institutes of Health (NIH); National Heart, Lung, and Blood Institute (NHLBI)
Recruiting - verified January 2017
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Prospective Biomarkers of Bone Metabolism in Hemophilia A
Conditions: Hemophilia; Bone Disease
Intervention: Drug: Advate
Sponsors: Oregon Health and Science University; Baxter Healthcare Corporation
Recruiting - verified September 2016
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Atenativ Effect on Uterine Blood Flow and Preeclampsia
Condition: Antithrombin III Deficiency
Intervention: Drug: Atenativ
Sponsors: Vastra Gotaland Region; Octapharma
Recruiting - verified March 2016
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Pharmacokinetics, Efficacy, and Safety of Human Plasma-Derived Fibrinogen (FIB Grifols) in Patients With Congenital Afibrinogenaemia
Condition: Congenital Afibrinogenaemia
Intervention: Biological: Human Plasma-Derived Fibrinogen Concentrate
Sponsor: Instituto Grifols, S.A.
Recruiting - verified July 2016
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Gene Therapy for WAS Follow-up
Condition: Wiskott-Aldrich Syndrome
Intervention: Genetic: Autologous CD34+ cells transduced with WASP lentiviral vector
Sponsor: Genethon
Recruiting - verified August 2016
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The European Paediatric Network for Haemophilia Management ( PedNet Registry)
Conditions: Factor VIII Deficiency; Factor IX Deficiency
Intervention: Drug: Coagulation proteins
Sponsors: UMC Utrecht; Lund University Hospital
Recruiting - verified November 2016
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Randomized Controlled Feasibility Trial of the Fearon Algorithm to Improve Management of Unstable Warfarin
Condition: Hypoprothrombinemia
Interventions: Other: Warfarin adjustment using the Fearon algorithm; Other: Warfarin adjustment using standard dosing
Sponsors: McMaster University; Epitome Pharmaceuticals
Recruiting - verified March 2016
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Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
Condition: Severe Haemophilia A
Intervention: Biological: Human cl rhFVIII
Sponsor: Octapharma
Recruiting - verified October 2016
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An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia A
Condition: Hemophilia A
Intervention: Biological: rFVIIIFc
Sponsors: Bioverativ Therapeutics Inc.; Swedish Orphan Biovitrum
Recruiting - verified May 2017
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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 - verified June 2017
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Efficacy and Safety Study of Octafibrin for On-demand Treatment of Acute Bleeding and to Prevent Bleeding During and After Surgery
Condition: Congenital Fibrinogen Deficiency
Intervention: Drug: Octafibrin
Sponsor: Octapharma
Recruiting - verified May 2016
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Hemophilia Inhibitor Prevention (INHIBIT) Trial
Condition: Severe Hemophilia A
Intervention: Drug: Long-acting recombinant factor VIII Fc fusion protein
Sponsor: University of Pittsburgh
Not yet recruiting - verified April 2017
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Development of a Specific Measure for Assessing Adherence in Adult Patients With Hemophilia.
Condition: Haemophilia
Intervention:
Sponsor: Universidad de Murcia
Recruiting - verified September 2015
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Adherence to Treatment in Hemophilia
Condition: Haemophilia
Intervention:
Sponsors: ANA TORRES-ORTUÑO; Pfizer
Recruiting - verified September 2015
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Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B
Condition: Hemophilia B
Intervention: Biological: rFIXFc
Sponsors: Bioverativ Therapeutics Inc.; Swedish Orphan Biovitrum
Recruiting - verified February 2017
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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 - verified May 2017
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Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
Conditions: Sickle Cell Disease; Transfusion Dependent Alpha- or Beta- Thalassemia; Diamond Blackfan Anemia; Paroxysmal Nocturnal Hemoglobinuria; Glanzmann Thrombasthenia; Severe Congenital Neutropenia; Shwachman-Diamond Syndrome; Non-Malignant Hematologic Disorders
Interventions: Drug: Reduced Toxicity Ablative Regimen; Drug: Reduced Intensity Preparative Regimen; Drug: Myeloablative Preparative Regimen
Sponsor: Masonic Cancer Center, University of Minnesota
Recruiting - verified February 2017
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Assessment of Proprioceptive and Functional Characteristics in Patients With Hemophilia
Condition: Hemophilia
Intervention:
Sponsor: Universidad Católica San Antonio de Murcia
Recruiting - verified September 2015
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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 - verified February 2016
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A Survey on the Success of Inhibitor Elimination Using Individualized Concentrate Selection and Controlled ITI
Condition: Haemophilia A
Intervention:
Sponsors: Haemophilia Centre Rhine Main; University of Bonn; Malmö University; University Hospital, Paris; Hospital Louis Pradel, Haematology Department, Lyon; Department of Hematology, CHU Montpellier; Octapharma Molecular Biochemistry, Berlin
Recruiting - verified May 2017
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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 - verified February 2017
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ADVATE Hemophilia A Outcome Database (AHEAD)
Condition: Hemophilia A
Intervention: Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Sponsor: Baxalta US Inc.
Recruiting - verified March 2017
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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 - verified August 2016
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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 - verified September 2016
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Pharmacokinetic, Efficacy and Safety of BT524 in Patients With Congenital Fibrinogen Deficiency
Conditions: Congenital Afibrinogenemia; Congenital Hypofibrinogenemia
Intervention: Biological: BT524 (fibrinogen concentrate from human plasma)
Sponsors: Biotest; ICON plc; Interlab; Phoenix Clinical Research
Recruiting - verified June 2016
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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 - verified May 2017
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Web-based Application for the Population Pharmacokinetic Service - Phase 1
Conditions: Hemophilia A; Hemophilia B
Intervention: Device: Population pharmacokinetic estimation
Sponsors: McMaster University; Canadian Hemophilia Society
Recruiting - verified March 2016
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Integrated Cancer Repository for Cancer Research
Conditions: Pancreatic Cancer; Thyroid Cancer; Lung Cancer; Esophageal Cancer; Thymus Cancer; Colon Cancer; Rectal Cancer; GIST; Anal Cancer; Bile Duct Cancer; Duodenal Cancer; Gallbladder Cancer; Gastric Cancer; Liver Cancer; Small Intestine Cancer; Peritoneal Surface Malignancies; Familial Adenomatous Polyposis; Lynch Syndrome; Bladder Cancer; Kidney Cancer; Penile Cancer; Prostate Cancer; Testicular Cancer; Ureter Cancer; Urethral Cancer; Hypopharyngeal Cancer; Laryngeal Cancer; Lip Cancer; Oral Cavity Cancer; Nasopharyngeal Cancer; Oropharyngeal Cancer; Paranasal Sinus Cancer; Nasal Cavity Cancer; Salivary Gland Cancer; Skin Cancer; CNS Tumor; CNS Cancer; Mesothelioma; Breastcancer; Leukemia; Melanoma; Sarcoma; Unknown Primary Tumor; Multiple Myeloma; Ovarian Cancer; Endometrial Cancer; Vaginal Cancer
Intervention:
Sponsor: University of Nebraska
Recruiting - verified May 2017
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Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
Condition: Severe Hemophilia A
Intervention: Biological: Human-cl rhFVIII
Sponsor: Octapharma
Recruiting - verified October 2016
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Willebrand International Non-interventional Global Surveillance
Condition: Von Willebrand Disease
Intervention:
Sponsor: Laboratoire français de Fractionnement et de Biotechnologies
Recruiting - verified March 2016
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Participation in a Research Registry for Immune Disorders
Conditions: ADA-SCID; Adenosine Deaminase Deficiency; Wiskott- Aldrich Syndrome; Primary Immunodeficiencies; Severe Combined Immune Deficiency
Intervention:
Sponsors: National Human Genome Research Institute (NHGRI); National Institute of Allergy and Infectious Diseases (NIAID); National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Recruiting - verified March 6, 2017
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Iron Deficiency and Hereditary Haemorrhagic Telangiectasia
Condition: Hereditary Haemorrhagic Telangiectasia
Intervention: Drug: Ferrous sulphate 200mg oral tablet
Sponsor: Imperial College London
Recruiting - verified July 2013
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BMT Abatacept for Non-Malignant Diseases
Conditions: Hurler Syndrome; Fanconi Anemia; Glanzmann Thrombasthenia; Wiskott-Aldrich Syndrome; Chronic Granulomatous Disease; Severe Congenital Neutropenia; Leukocyte Adhesion Deficiency; Shwachman-Diamond Syndrome; Diamond-Blackfan Anemia; Dyskeratosis-congenita; Chediak-Higashi Syndrome; Severe Aplastic Anemia; Thalassemia Major; Hemophagocytic Lymphohistiocytosis; Sickle Cell Disease
Intervention: Drug: Abatacept
Sponsor: Emory University
Recruiting - verified August 2016
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Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa
Condition: Haemophilia
Intervention:
Sponsor: Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus
Recruiting - verified December 2015
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Reperfusion of Pulmonary Arteriovenous Malformations After Embolotherapy
Condition: Pulmonary Arteriovenous Malformations
Intervention: Procedure: Angiography and embolotherapy
Sponsor: St. Michael's Hospital, Toronto
Recruiting - verified May 2017
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Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
Conditions: Severe Combined Immunodeficiency (SCID); Immunodeficiency With Predominant T-cell Defect, Unspecified; Severe Chronic Neutropenia; Chronic Granulomatous Disease (CGD); Hyper IgE Syndromes; Hyper IgM Deficiencies; Wiskott-Aldrich Syndrome; Mendelian Susceptibility to Mycobacterial Disease; Common Variable Immune Deficiency (CVID)
Intervention: Biological: CD3/CD19 negative allogeneic hematopoietic stem cells
Sponsor: University of Pittsburgh
Recruiting - verified June 2017
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Immune Disorder HSCT Protocol
Conditions: Immune Deficiency Disorders; Severe Combined Immunodeficiency; Chronic Granulomatous Disease; X-linked Agammaglobulinemia; Wiskott-Aldrich Syndrome; Hyper-IgM; DiGeorge Syndrome; Chediak-Higashi Syndrome; Common Variable Immune Deficiency; Immune Dysregulatory Disorders; Hemophagocytic Lymphohistiocytosis; IPEX; Autoimmune Lymphoproliferative Syndrome; X-linked Lymphoproliferative Syndrome
Intervention: Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Sponsor: Washington University School of Medicine
Recruiting - verified November 2016
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Multicentre, Non-controlled, Prospective, Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A
Condition: Haemophilia A
Intervention: Biological: Optivate 500IU
Sponsor: Bio Products Laboratory
Recruiting - verified December 2016
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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 - verified May 2016
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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 - verified May 2017
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Cardiopulmonary Exercise Testing to Evaluate Pulmonary AVMs
Conditions: Pulmonary Arteriovenous Malformations; Hereditary Hemorrhagic Telangiectasia
Interventions: Other: Cardiopulmonary exercise test; Procedure: Blood test
Sponsor: Imperial College London
Recruiting - verified October 2015
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Outcome of Acquired Haemophilia With Steroid Combined With Cyclophosphamide Versus Steroid Combined With Rituximab (CREHA Study)
Condition: Acquired Haemophilia
Interventions: Drug: Steroid + cyclophosphamide; Drug: Steroid + rituximab
Sponsor: University Hospital, Rouen
Recruiting - verified November 2016
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Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
Conditions: SCID; Omenn's Syndrome; Reticular Dysgenesis; Wiskott-Aldrich Syndrome; Bare Lymphocyte Syndrome; Common Variable Immunodeficiency; Chronic Granulomatous Disease; CD40 Ligand Deficiency; Hyper IgM Syndrome; X-linked Lymphoproliferative Disease; Hemophagocytic Lymphohistiocytosis; Griscelli Syndrome; Chediak-Higashi Syndrome; Langerhan's Cell Histiocytosis
Interventions: Drug: Alemtuzumab 0.3 mg; Drug: Cyclophosphamide; Drug: Busulfan; Biological: Stem Cell Transplantation; Drug: Fludarabine phosphate 40 mg; Drug: Melphalan; Drug: Alemtuzumab 0.2 mg; Drug: Busulfan; Drug: Fludarabine phosphate 30 mg; Drug: MESNA
Sponsor: Masonic Cancer Center, University of Minnesota
Recruiting - verified February 2017
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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 - verified September 2016
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Clinical and Basic Investigations Into Erdheim Chester Disease
Conditions: Myelofibrosis; Gaucher Disease; Pulmonary Fibrosis; Hermansky-Pudlak Syndrome (HPS); Cancer
Intervention:
Sponsor: National Human Genome Research Institute (NHGRI)
Recruiting - verified April 3, 2017
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Hemophilia Inhibitor Previously Untreated Patient Study
Condition: Hemophilia A
Intervention: Drug: FVIII concentrate
Sponsors: The University of Texas Health Science Center, Houston; Rho, Inc.; Baxter Healthcare Corporation
Recruiting - verified May 2016
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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 - verified May 2017
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Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
Condition: Wiskott-Aldrich Syndrome
Intervention: Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
Sponsors: Genethon; Great Ormond Street Hospital for Children NHS Foundation Trust; Institute of Child Health
Recruiting - verified February 2016
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Gene Therapy for WAS
Condition: Wiskott-Aldrich Syndrome
Intervention: Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
Sponsors: Genethon; Hôpital Necker-Enfants Malades
Recruiting - verified February 2016
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Thrombocytopathy in Gaucher Disease Patients
Conditions: Gaucher Disease; Thrombocytopathy
Intervention:
Sponsors: Rabin Medical Center; Genzyme, a Sanofi Company
Recruiting - verified October 2016
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BAY81-8973 Pediatric Safety and Efficacy Trial
Condition: Haemophilia A
Intervention: Biological: Recombinant Factor VIII (BAY81-8973)
Sponsor: Bayer
Recruiting - verified June 2017
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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 - verified September 2016
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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 - verified May 2017
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Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
Condition: Hemophilia B
Interventions: Genetic: Gene Transfer; Drug: scAAV2/8-LP1-hFIXco
Sponsors: St. Jude Children's Research Hospital; National Heart, Lung, and Blood Institute (NHLBI); Hemophilia of Georgia, Inc.; Children's Hospital of Philadelphia
Recruiting - verified April 2017
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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 - verified March 2017
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Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
Condition: Von Willebrand Disease
Intervention: Biological: Alphanate SD/HT
Sponsor: Grifols Biologicals Inc.
Recruiting - verified January 2017
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Safety, Pharmacokinetics and Efficacy of an AT-III Concentrate.
Condition: Antithrombin III Deficiency
Intervention: Drug: Plasma-derived AT-III concentrate
Sponsor: Grifols Biologicals Inc.
Recruiting - verified January 2017
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Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A
Condition: Severe Hemophilia A
Intervention: Drug: Alphanate SD/HT
Sponsor: Grifols Biologicals Inc.
Recruiting - verified January 2017
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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 - verified August 2016
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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 - verified May 2016
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Molecular Studies on Hereditary Haemorrhagic Telangiectasia Families
Condition: Telangiectasia, Hereditary Hemorrhagic
Intervention:
Sponsors: Imperial College London; British Heart Foundation
Recruiting - verified August 2008
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Case Notes Review on Patients With Hereditary Haemorrhagic Telangiectasia
Condition: Telangiectasia, Hereditary Hemorrhagic
Intervention:
Sponsors: Imperial College London; The Margaret Hayton HHT Fund
Recruiting - verified August 2008
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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 - verified March 9, 2017
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