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.

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