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Clinical Trials

Below is a list of active clinical trials worldwide which involve patients with inherited bleeding disorders. The link from each study title will take you to ClinicalTrials.gov which gives you more information about the trial.
To see a list of trials for a particular bleeding disorder use the drop-down list below to select the condition you're interested in. A new window will open up with a list of trials for this condition only.

Please select the category:
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
Conditions:   Adeno-Associated Virus (AAV);   Blood Coagulation Disorder;   Blood Coagulation Disorders, Inherited;   Coagulation Protein Disorders;   Factor VIII (FVIII);   Factor VIII (FVIII) Deficiency;   Factor VIII (FVIII) Gene;   Factor VIII (FVIII) Protein;   Genetic Diseases, Inborn;   Genetic Diseases, X-Linked;   Gene Therapy;   Gene Transfer;   Hematologic Diseases;   Hemorrhagic Disorders;   Recombinant;   Vector;   Inhibitors
Intervention:   Genetic: SPK-8016
Sponsor:   Spark Therapeutics
Not yet recruiting
Platelet Glycoproteins in Inherited Thrombocytopathy: Association With Aggregation Studies and Bleeding Severity
Conditions:   Bleeding;   Platelet Dysfunction
Intervention:   Diagnostic Test: Flowcytometry analysis of platelets surface receptors
Sponsor:   Assiut University
Recruiting
Development of a Device for Evaluating Primary Hemostasis Under Whole Blood Flow Conditions
Conditions:   Von Willebrand Diseases;   Major Constitutional Thrombopathy;   Patient on Antiplatelet Drugs
Intervention:   Biological: Blood sampling
Sponsor:   Centre Hospitalier Universitaire Dijon
Not yet recruiting
Development of a Quality of Life Measurement Scale in Hereditary Haemorrhagic Telangiectasia (HHT) Disease.
Condition:   Hereditary Hemorrhagic Telangiectasia
Interventions:   Other: Phase 1 : fill out 1 scale;   Other: Phase 2 : fill out 1 scale and 4 questionnaires
Sponsor:   Hospices Civils de Lyon
Not yet recruiting
Evaluation of Obstetrical and Neonatal Complications in Hereditary Haemorrhagic Telangiectasia (HHT)
Conditions:   Osler Rendu Disease;   Hereditary Hemorrhagic Telangiectasia
Intervention:   Other: Questionnaire
Sponsor:   Hospices Civils de Lyon
Not yet recruiting
Von Willebrand Antigen and Activity as Novel Biomarkers of Hemostasis in Inflammatory Bowel Disease
Conditions:   Inflammatory Bowel Diseases;   Von Willebrand Diseases
Intervention:   Diagnostic Test: von willebrand antigen and activity
Sponsor:   Assiut University
Not yet recruiting
Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients
Condition:   Haemophilia A
Intervention:  
Sponsor:   Octapharma
Recruiting
Perioperative Eltrombopag in Patients With Inherited Thrombocytopenia
Condition:   Thrombocytopenia
Intervention:   Drug: Eltrombopag
Sponsors:   University Hospital, Toulouse;   French network for inherited hemorragic diseases;   National Reference Centre for Platelet Pathologies
Not yet recruiting
A Study of Haemophilia B Patients Who Have Undergone Gene Therapy
Condition:   Hemophilia B
Intervention:   Biological: FLT180a
Sponsor:   Freeline Therapeutics
Recruiting
A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
Condition:   Haemophilia B
Intervention:   Drug: Alprolix
Sponsor:   Swedish Orphan Biovitrum
Recruiting
Minimal Hepatic Encephalopathy in Hereditary Hemorrhagic Telangiectasian
Conditions:   Hepatic Encephalopathy;   Minimal Brain Dysfunction
Intervention:   Device: Critical flicker frequency assessment
Sponsor:   University of Bari
Recruiting
Prospective Descriptive Study of the Angiogenic T Cell Population in Subjects With Hereditary Hemorrhagic Telangiectasia (HHT)
Condition:   Hereditary Hemorrhagic Telangiectasia
Interventions:   Biological: Blood samples;   Other: Epistaxis charts
Sponsor:   Centre Hospitalier Universitaire Dijon
Recruiting
The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B
Condition:   Hemophilia
Interventions:   Other: Blood sample;   Other: Questionnaire;   Other: MRI-imaging;   Other: Physical examination
Sponsor:   Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Recruiting
Assessing Physician and Hemophilia A Patient Reasons and Expectations for Switching Treatment to Kovaltry: A Nested Study Within an Existing Registry
Condition:   Hemophilia A
Intervention:   Drug: Antihemophilic Factor (Kovaltry, BAY81-8973)
Sponsors:   Bayer;   American Thrombosis and Hemostasis Network
Recruiting
Von Willebrand Factor Concentrate During ECMO Support
Condition:   Acquired Von Willebrand Disease
Interventions:   Drug: Von Willebrand Factor;   Drug: Saline Solution
Sponsors:   Tirol Kiniken GmbH;   LFB BIOMEDICAMENTS
Recruiting
Nationwide Awareness Campaign and Call for Dental Screening for HHT in Germany
Conditions:   Hereditary Hemorrhagic Telangiectasia;   Dental Diseases
Intervention:  
Sponsors:   University Hospital, Essen;   Philipps University Marburg Medical Center;   University Hospital, Aachen;   German HHT self-help group
Not yet recruiting
Gait Examination in Patients With Hemophilia in Austria
Condition:   Hemophilia
Interventions:   Diagnostic Test: 3-d gait examination (level walking);   Diagnostic Test: Anthropometrics and body composition assessment;   Diagnostic Test: Biomarkers;   Diagnostic Test: Clinical examination;   Diagnostic Test: Habitual physical activity tracking (accelerometer based)
Sponsors:   FH Campus Wien, University of Applied Sciences;   Medical University of Vienna;   Shire
Recruiting
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
Recruiting
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
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
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
Quality of Life in Patients With Congenital Afibrinogenemia
Condition:   Afibrinogenemia, Congenital
Intervention:   Other: Questionnaire quality of life
Sponsor:   University Hospital, Geneva
Recruiting
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
Impact Study on Cerecare Compression Garments in the Treatment of Ehlers-Danlos Syndromes
Condition:   Eds, Unspecified Type
Intervention:   Device: Cerecare compression garments
Sponsor:   Cerecare
Recruiting
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
A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors
Conditions:   Hemophilia A;   Hemophilia B
Interventions:   Drug: fitusiran;   Drug: factor VIII or factor IX
Sponsor:   Genzyme, a Sanofi Company
Recruiting
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
Sponsor:   Genzyme, a Sanofi Company
Recruiting
Doxycycline for Hereditary Hemorrhagic Telangiectasia
Condition:   Hereditary Hemorrhagic Telangiectasia (HHT)
Interventions:   Drug: Doxycycline Hyclate;   Drug: Placebo
Sponsors:   St. Michael's Hospital, Toronto;   Barrow Neurological Institute;   Duke University;   Feinstein Institute for Medical Research;   University of Pittsburgh;   Sunnybrook Health Sciences Centre
Recruiting
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
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
A Factor IX Gene Therapy Study (FIX-GT)
Condition:   Hemophilia B
Intervention:   Biological: FLT180a
Sponsor:   University College, London
Recruiting
Joint Health Study
Condition:   Hemophilia B
Intervention:  
Sponsors:   Bloodworks;   CSL Behring
Recruiting
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
World Bleeding Disorders Registry
Conditions:   Hemophilia A;   Hemophilia B
Intervention:  
Sponsor:   World Federation of Hemophilia
Not yet recruiting
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
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;   Seattle Children's Hospital;   Oregon Health and Science University;   Providence Health & Services
Recruiting
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
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
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
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
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
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
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
Low Von Willebrand in Ireland Cohort Study
Condition:   Von Willebrand Factor, Deficiency
Intervention:  
Sponsor:   St. James's Hospital, Ireland
Recruiting
Bleeding Risk Evaluation in Haemophilia Patients Under Antiplatelet Therapies
Condition:   Hemophilia
Intervention:   Other: Non interventional study
Sponsor:   Nantes University Hospital
Recruiting
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;   Logistics University of Chinese People's Armed Police Forces;   Beijing Anzhen Hospital;   West China Second University Hospital of Sichuan University;   Jinshan Hospital of Fudan University;   Fudan University
Recruiting
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
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
Frequency of Hemorrhages Associated With the Functional Anomalies of Willebrand Factor in Emergency Patients
Condition:   Von Willebrand Diseases
Intervention:  
Sponsor:   University Hospital, Lille
Recruiting
UK - EHL Outcomes Registry
Conditions:   Hemophilia A;   Hemophilia B
Intervention:  
Sponsor:   Royal Free Hospital NHS Foundation Trust
Recruiting
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
A Gene Transfer Study for Hemophilia A
Condition:   Hemophilia A
Intervention:   Genetic: SPK-8011
Sponsor:   Spark Therapeutics
Recruiting
MRA With Feraheme in HHT
Condition:   Hereditary Hemorrhagic Telangiectasia
Intervention:   Procedure: Feraheme MRI/MRA
Sponsor:   University of California, Los Angeles
Recruiting
Immunmodulation in Patients With HHT
Condition:   Hereditary Haemorrhagic Telangiectasia (HHT)
Intervention:   Other: blood sample
Sponsor:   University Hospital, Essen
Recruiting
Fascial Therapy in Elbow Hemophilic Arthropathy
Condition:   Hemophilia
Intervention:   Other: Experimental group
Sponsor:   Real Fundación Victoria Eugenia
Recruiting
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
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
rVWF IN PROPHYLAXIS
Condition:   Von Willebrand Disease
Interventions:   Biological: von Willebrand factor (Recombinant);   Biological: Antihemophilic Factor (Recombinant)
Sponsor:   Baxalta now part of Shire
Recruiting
Graded TTCE for Post-Embolization PAVM Monitoring
Condition:   Hereditary Hemorrhagic Telangiectasia (HHT)
Intervention:   Other: Transthoracic Contrast Echocardiogram
Sponsor:   University of Pennsylvania
Recruiting
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
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
BAX 802 in CHA With Inhibitors
Condition:   Hemophilia A
Intervention:   Biological: Antihemophilic Factor (Recombinant), Porcine Sequence (BAX802)
Sponsors:   Baxalta now part of Shire;   Baxalta Innovations GmbH, now part of Shire
Recruiting
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
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
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
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
TGA (Thrombin Generation Assay) and Prophylaxis in Haemophilia
Condition:   Haemophilia A
Intervention:   Other: Thrombin generation measurement.
Sponsor:   Hospices Civils de Lyon
Recruiting
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
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
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
Weight-based Dosing in Hemophilia A
Condition:   Hemophilia
Intervention:   Drug: rFVIII
Sponsor:   Craig Seaman
Recruiting
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
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;   Duke University
Not yet recruiting
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
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
Swiss Hemophilia Registry
Condition:   Hemophilia and Other Severe Bleeding Disorders
Intervention:   Other: Registry
Sponsor:   Swiss Hemophilia Network
Recruiting
Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
Condition:   Hemophilia
Intervention:  
Sponsor:   Karolinska Institutet
Recruiting
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
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
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
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
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
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
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
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
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
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
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
Modifiers of Disease Severity in Cerebral Cavernous Malformations
Conditions:   Cavernous Angioma, Familial;   Cerebral Cavernous Malformations;   Cerebral Cavernous Hemangioma
Intervention:  
Sponsors:   University of California, San Francisco;   Barrow Neurological Institute;   Angioma Alliance;   National Institute of Neurological Disorders and Stroke (NINDS);   University of New Mexico;   National Center for Advancing Translational Science (NCATS)
Recruiting
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
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
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
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
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
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
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
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
Molecular Studies on Hereditary Haemorrhagic Telangiectasia Families
Condition:   Telangiectasia, Hereditary Hemorrhagic
Intervention:  
Sponsors:   Imperial College London;   British Heart Foundation
Recruiting
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