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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:
Rare Inherited Bleedig Disorders in Children at Sohag University Hospital
Condition:   Rare Inherited Bleeding Disorders in Children at Sohag University Hospital
Intervention:   Other: blood laboratory investigation(coagulation profile- platelet function test)
Sponsor:   Sohag University
Recruiting
The Role of Standardized Bleeding Score in Diagnosis of Pediatric Von Willebrand's Disease
Condition:   Von Willebrand's Diseased Children, Bleeding Questionnaire is Done
Intervention:   Diagnostic Test: Bleeding score ( questionnaire )
Sponsor:   Sohag University
Recruiting
Genetic Background of Patients With Low Von Willebrand Factor Levels
Condition:   Low Von Willebrand Factor
Intervention:   Diagnostic Test: Whole-exome sequencing
Sponsor:   Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Recruiting
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
Genomics of Fibrin Clot Structure in Patients With Constitutional Dysfibrinogenemia
Condition:   Hereditary Dysfibrinogenemia
Intervention:   Biological: Blood test
Sponsor:   University Hospital, Clermont-Ferrand
Not yet recruiting
AAV Gene Therapy Screening/Observational Protocol (ECLIPSE)
Conditions:   Hemophilia B;   Blood Coagulation Disorders, Inherited;   Blood Coagulation Disorder;   Haematologic Disease
Intervention:  
Sponsor:   Freeline Therapeutics
Recruiting
Chronic Pain and Hemophilia
Conditions:   Chronic Pain;   Hemophilia
Intervention:   Other: questionnaires
Sponsor:   University Hospital, Clermont-Ferrand
Not yet 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
Cerebral Hemorrhage Risk in Hereditary Hemorrhagic Telangiectasia
Condition:   Hereditary Hemorrhagic Telangiectasia
Intervention:  
Sponsors:   Unity Health Toronto;   National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting
Evaluation of Patients With Immune Function Abnormalities
Conditions:   Chronic Granulomatous Disease (CGD);   X-Linked Severe Combined Immune Deficiency (XSCID);   Leukocyte Adhesion Deficiency 1 (LAD);   Graft Versus Host Disease (cGvHD)
Intervention:  
Sponsor:   National Institute of Allergy and Infectious Diseases (NIAID)
Recruiting
A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
Condition:   Von Willebrand Disease
Interventions:   Biological: von Willebrand factor (Recombinant);   Biological: Antihemophilic Factor (Recombinant)
Sponsors:   Baxalta now part of Shire;   Takeda Development Center Americas, Inc.
Recruiting
Bevacizumab In Hereditary Hemorrhagic Telangiectasia
Condition:   Hereditary Hemorrhagic Telangiectasia
Intervention:   Drug: Bevacizumab
Sponsor:   Hanny Al-Samkari, MD
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;   Myelodysplastic Syndromes;   Myeloproliferative Diseases
Intervention:  
Sponsors:   St. Jude Children's Research Hospital;   Boston Children's Hospital;   University of Memphis;   Monroe Carell Jr. Children's Hospital at Vanderbilt;   Baylor College of Medicine;   Children's Hospital of Philadelphia;   Dana-Farber Cancer Institute
Recruiting
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
Conditions:   Hemophilia A;   Gene Therapy;   Clotting Disorders;   Blood Disorder
Intervention:   Biological: Valoctocogene Roxaparvovec
Sponsor:   BioMarin Pharmaceutical
Recruiting
TPO-Mimetic Use in Children for Hemotopoietic Failure
Conditions:   Bone Marrow Failure Disorders;   Aplastic Anemia;   Thrombocytopenia;   Refractory Cytopenia of Childhood;   Myelodysplastic Syndrome(MDS)
Intervention:   Drug: Romiplostim
Sponsors:   Anjali Sharathkumar;   Amgen
Recruiting
World Bleeding Disorders Registry
Conditions:   Hemophilia A;   Hemophilia B
Intervention:  
Sponsor:   World Federation of Hemophilia
Recruiting
Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding
Condition:   Hemophilia A
Intervention:   Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)
Sponsor:   Bayer
Recruiting
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
Conditions:   Adeno-Associated Virus (AAV);   Blood Coagulation Disorder;   Blood Coagulation Disorders, Inherited;   Coagulation Protein Disorders;   Factor VIII (FVIII);   Factor VIII (FVIII) Deficiency;   Factor VIII (FVIII) Gene;   Factor VIII (FVIII) Protein;   Genetic Diseases, Inborn;   Genetic Diseases, X-Linked;   Gene Therapy;   Gene Transfer;   Hematologic Diseases;   Hemorrhagic Disorders;   Recombinant;   Vector;   Inhibitors
Intervention:   Genetic: SPK-8016
Sponsor:   Spark Therapeutics
Recruiting
Influence of Hypoxic Induced Factors in Patients With Hereditary Hemorrhagic Telangiectasia
Condition:   Hereditary Hemorrhagic Telangiectasia
Intervention:   Other: hypoxic induced factors
Sponsor:   University Hospital, Essen
Recruiting
Latin-American Von Willebrand Disease Registry
Condition:   Von Willebrand Disease
Intervention:   Other: Observation
Sponsor:   Academia Nacional de Medicina
Not yet recruiting
Swiss Hemophilia Registry
Condition:   Hemophilia and Other Severe Bleeding Disorders
Intervention:   Other: Registry
Sponsor:   Swiss Hemophilia Network
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;   Baxalta Innovations GmbH, Wien, Austria
Recruiting
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Conditions:   Hematologic Disorder;   Bleeding Disorder;   Connective Tissue Disorder;   Hemophilia;   Thrombosis;   Von Willebrand Diseases;   Thrombophilia;   Rare Bleeding Disorder;   Platelet Disorder
Intervention:  
Sponsors:   American Thrombosis and Hemostasis Network;   Pfizer;   Hemophilia of Georgia, Inc.;   Genentech, Inc.
Recruiting
Efficacy of Nintedanib for Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) Patients
Condition:   Telangiectasia, Hereditary Hemorrhagic
Interventions:   Drug: Nintedanib;   Drug: Placebo
Sponsors:   Dr. Romain Lazor;   Boehringer Ingelheim
Not yet recruiting
Tacrolimus Trial for Hereditary Hemorrhagic Telangiectasia (HHT)
Conditions:   Hereditary Hemorrhagic Telangiectasia;   Epistaxis Nosebleed
Intervention:   Drug: Tacrolimus capsule (low-dose)
Sponsors:   Unity Health Toronto;   United States Department of Defense
Recruiting
Pazopanib for the Treatment of Epistaxis in Hereditary Hemorrhagic Telangiectasia
Conditions:   Hereditary Hemorrhagic Telangiectasia;   Epistaxis
Intervention:   Drug: Pazopanib
Sponsors:   Cure HHT;   University of North Carolina
Not yet recruiting
Treatment of Hereditary Hemorrhagic Telangiectasia of the Nasal Mucosa by Intranasal Bevacizumab : Search for Effective Dose
Condition:   Telangiectasia, Hereditary Hemorrhagic
Intervention:   Drug: bevacuzimab spray
Sponsor:   University Hospital, Caen
Recruiting
Pazopanib Effects on Bleeding in Hereditary Hemorrhagic Telangiectasia
Conditions:   Hereditary Hemorrhagic Telangiectasia;   Epistaxis;   Anemia;   Nosebleed;   HHT
Interventions:   Drug: Pazopanib;   Drug: Placebo oral capsule
Sponsors:   Cure HHT;   United States Department of Defense
Not yet recruiting
Pomalidomide for the Treatment of Bleeding in HHT
Condition:   Telangiectasia, Hereditary Hemorrhagic
Interventions:   Drug: Pomalidomide Oral Product;   Drug: Placebo oral capsule
Sponsors:   The Cleveland Clinic;   RTI International
Recruiting
Hereditary Hemorrhagic Telangiectasia (HHT) Research Outcomes Registry
Conditions:   Hereditary Hemorrhagic Telangiectasia;   HHT;   Arteriovenous Malformation of Brain
Intervention:   Other: Registry and Saliva sample
Sponsors:   Unity Health Toronto;   Cure HHT;   Dartmouth College
Recruiting
Evaluation of Video-assisted Instructions of Nasal Self-packing in Patients With HHT
Condition:   Hereditary Haemorrhagic Telangiectasia
Intervention:   Procedure: video-assisted nasal self-packing
Sponsor:   University Hospital, Essen
Recruiting
A Study to Learn How Safe the Study Treatment BAY94-9027 is and How it Affects the Body in Previously Treated Children Aged 7 to Less Than 12 Years With Severe Hemophilia A, a Genetic Bleeding Disorder That is Caused by the Lack of a Protein Called Clotting Factor 8 (FVIII) in the Blood
Conditions:   Treatment of Bleeding;   Prophylaxis of Bleeding;   Hemophilia A;   Children
Intervention:   Biological: Damoctocog alfa pegol (Jivi, BAY94-9027)
Sponsor:   Bayer
Recruiting
Assess Safety and Efficacy of VAD044 in HHT Patients
Condition:   Hereditary Hemorrhagic Telangiectasia (HHT)
Intervention:   Drug: VAD044
Sponsor:   Vaderis Therapeutics AG
Not yet 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;   University of New Mexico;   University of Chicago;   National Institute of Neurological Disorders and Stroke (NINDS);   Boston Children's Hospital;   Children's Hospital Medical Center, Cincinnati;   Barrow Neurological Institute;   Angioma Alliance
Recruiting
A Gene Transfer Study for Hemophilia A
Condition:   Hemophilia A
Intervention:   Genetic: SPK-8011
Sponsor:   Spark Therapeutics
Recruiting
ATHN 9: Severe VWD Natural History Study
Condition:   Von Willebrand Diseases
Intervention:  
Sponsors:   American Thrombosis and Hemostasis Network;   Takeda
Recruiting
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
Conditions:   Hemophilia A With Inhibitor;   Hemophilia A With Anti Factor VIII
Intervention:   Biological: Valoctocogene roxaparvovec
Sponsor:   BioMarin Pharmaceutical
Recruiting
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Conditions:   Severe Aplastic Anemia;   Telomere Biology Disorders;   Inherited Bone Marrow Failure Syndromes
Intervention:  
Sponsor:   National Heart, Lung, and Blood Institute (NHLBI)
Recruiting
A Study to Learn More About Treatment With Damoctocog Alfa Pegol, How it is Used in Every Day Practice ("Real-World"), and How Satisfied People Who Receive Damoctocog Alfa Pegol Are in United States (US) Hemophilia Treatment Centers
Condition:   Hemophilia A
Intervention:   Other: No intervention
Sponsor:   Bayer
Not yet recruiting
National Longitudinal Cohort of Hematological Diseases
Conditions:   Multiple Myeloma;   Myeloma, Multiple;   Myelomas, Multiple;   Acute Myeloid Leukemia;   Leukemias, Acute Myeloid;   Myeloid Leukemias, Acute;   Hemophilia;   Hemophilia A;   Hemophilia As;   Hemophilia B;   Hemophilia Bs;   Myelodysplastic Syndrome;   MDS;   Lymphoma;   Leukemia;   Aplastic Anemia;   Bleeding Disorder;   Bone Marrow Transplantation
Intervention:  
Sponsor:   Institute of Hematology & Blood Diseases Hospital
Recruiting
Prevention of Bleeding in Patients With Moderate and Severe Hemophilia A Playing Sports: A Comparison Between Factor VIII and Emicizumab Prophylaxis
Condition:   Hemophilia A
Interventions:   Drug: Emicizumab;   Drug: FVIII
Sponsors:   Wayne State University;   Genentech, Inc.
Not yet recruiting
Safety and Efficacy in a ReAl-Life Study in Patients With Haemophilia Treated wIth NovoEight® for Surgery
Condition:   Hemophilia
Intervention:   Other: no intervention
Sponsor:   Nantes University Hospital
Recruiting
Haemophilia and Bone Loss - PHILEOS Study
Condition:   Hemophilia
Interventions:   Radiation: Bone densitometry (BMD);   Biological: Blood sampling for patients only
Sponsors:   Centre Hospitalier Universitaire de Saint Etienne;   Ministry of Health, France
Recruiting
Prevent Postpartum Hemorrhage in Women With Von Willebrand Disease: The VWD-WOMAN Trial
Conditions:   Von Willebrand Diseases;   Postpartum Hemorrhage
Interventions:   Drug: Recombinant Von Willebrand factor;   Drug: Tranexamic Acid Injection [Cyklokapron]
Sponsor:   Margaret Ragni
Recruiting
Study to Test the Safety and How Well Patients With Severe Hemophilia A Respond to Treatment With BAY 2599023 (DTX 201), a Drug Therapy That Delivers a Healthy Version of the Defective Factor VIII Gene Into the Nucleus of Liver Cells Using an Altered, Non-infectious Virus (AAV) as a "Shuttle".
Condition:   Hemophilia A
Intervention:   Drug: BAY2599023 (DTX201)
Sponsors:   Bayer;   Ultragenix pharmaceutical
Recruiting
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
Condition:   Hemophilia A
Intervention:   Drug: Gene therapy
Sponsors:   Christian Medical College, Vellore, India;   Centre for Stem Cell Research, (unit of inStem), CMC, Vellore
Recruiting
Efficacy of Nintedanib Per os as a Treatment for Epistaxis in HHT Disease.
Conditions:   Telangiectasia, Hereditary Hemorrhagic;   Rendu Osler Disease
Interventions:   Drug: Nintedanib 150 mg and 100 mg soft capsules;   Drug: Oral treatment of placebo soft capsule
Sponsor:   Hospices Civils de Lyon
Recruiting
Prophylaxis Regimen for Hemophilia A Patients
Conditions:   Hemophilia A;   Prophylaxis of Bleeding
Intervention:   Biological: Damoctocog alfa-pegol (Jivi, BAY94-9027)
Sponsor:   Bayer
Not yet recruiting
Perioperative Eltrombopag in Patients With Inherited Thrombocytopenia
Condition:   Thrombocytopenia
Intervention:   Drug: Eltrombopag
Sponsors:   University Hospital, Toulouse;   French network for inherited hemorragic diseases;   National Reference Centre for Platelet Pathologies
Recruiting
Effects of Emicizumab vs. Factor VIII Prophylaxis on Joint and Bone Health in Severe Hemophilia A
Condition:   Hemophilia A
Intervention:   Other: assessment of joint health and bone density
Sponsors:   Washington Institute for Coagulation;   Genentech, Inc.
Recruiting
Emicizumab in Patients With Acquired Hemophilia A
Condition:   Acquired Hemophilia A
Intervention:   Drug: emicizumab
Sponsors:   University of Washington;   Genentech, Inc.
Not yet recruiting
Von Willebrand Disease in the Netherlands
Condition:   Von Willebrand Disease
Intervention:  
Sponsors:   Erasmus Medical Center;   Stichting Haemophilia (Dutch Haemophilia Foundation);   CSL Behring;   Shire
Recruiting
Sirolimus for Nosebleeds in HHT
Conditions:   Hereditary Hemorrhagic Telangiectasia;   Nosebleeds;   Epistaxis
Intervention:   Drug: Sirolimus
Sponsors:   Unity Health Toronto;   National Institutes of Health (NIH)
Recruiting
Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome
Condition:   Hermansky-Pudlak Syndrome (HPS)
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
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
Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
Condition:   Hemophilia
Intervention:  
Sponsor:   Karolinska Institutet
Recruiting
A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Condition:   Congenital Thrombotic Thrombocytopenic Purpura
Intervention:   Biological: TAK-755
Sponsors:   Takeda;   Takeda Development Center Americas, Inc.;   Shire
Recruiting
A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP)
Condition:   Thrombotic Thrombocytopenic Purpura (TTP)
Interventions:   Biological: BAX930;   Biological: Standard of care
Sponsors:   Baxalta now part of Shire;   Takeda Development Center Americas, Inc.
Recruiting
Von Willebrand Factor in Pregnancy (VIP) Study
Condition:   Von Willebrand Diseases
Interventions:   Other: Use of a postpartum diary and additional blood draws;   Drug: VWF replacement therapy with Wilate;   Drug: Tranexamic acid;   Other: Use of a postpartum diary and additional blood draws.
Sponsors:   Bloodworks;   Mary M. Gooley Hemophilia Center;   Ergomed;   Octapharma
Recruiting
Efficacy and Safety of Fanhdi®, a High-purity Von Willebrand Containing FVIII Concentrate, in Pediatric Patients With Von Willebrand Disease
Condition:   Von Willebrand Disease
Intervention:   Drug: plasma-derived FVIII/VWF concentrate
Sponsors:   Grifols Therapeutics LLC;   Instituto Grifols, S.A.
Recruiting
Bone Microarchitecture in Men With Hemophilia
Conditions:   Severe Hemophilia A;   Osteoporosis
Interventions:   Radiation: HR-pQCT;   Biological: Blood sample;   Radiation: Dual energy X-ray absorptiometry;   Other: medical data collection
Sponsor:   Hospices Civils de Lyon
Recruiting
Pre-clinical Models for Mesenchymal Stem Cell Therapy in Hemophilic Arthropathy
Conditions:   Hemophilia A;   Hemophilia B;   Arthropathy
Intervention:  
Sponsor:   University Hospital, Montpellier
Recruiting
Pain and Joint Status With Psychosocial Factors in Patients With Hemophilia
Condition:   Hemophilia Arthropathy
Intervention:   Other: Observational group
Sponsor:   Investigación en Hemofilia y Fisioterapia
Not yet recruiting
PyROphosPHate Supplementation to Fight ECtopIc Calcification in PseudoXanthoma Elasticum
Condition:   Pseudoxanthoma Elasticum
Intervention:   Drug: study treatment PPI
Sponsor:   Centre Hospitalier Universitaire de Nice
Not yet recruiting
Proximal Aortopathy in Scotland - Epidemiology and Surgical Outcomes
Conditions:   Aortic Diseases;   Aortic Dissection;   Aortic Aneurysm, Thoracic;   Aortic Ectasia;   Aortic Valve Insufficiency;   Bicuspid Aortic Valve;   Marfan Syndrome;   Ehlers-Danlos Syndrome;   Loeys-Dietz Syndrome
Interventions:   Procedure: Surgery on the proximal aorta (aortic root +/- ascending aorta);   Other: Patients diagnosed with thoracic aortopathy but not operated upon
Sponsors:   Golden Jubilee National Hospital;   Aberdeen Royal Infirmary;   Royal Infirmary of Edinburgh;   Network for Inherited Cardiac Conditions Scotland;   University of Glasgow
Not yet recruiting
Analysis of Specimens From Individuals With Pulmonary Fibrosis
Conditions:   Pulmonary Fibrosis;   Healthy Volunteers;   Hermansky-Pudlak Syndrome (HPS)
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Recruiting
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
Conditions:   All Diagnosed Health Conditions;   ADD/ADHD;   Alopecia Areata;   Ankylosing Spondylitis;   Asthma;   Atopic Dermatitis Eczema;   Beta Thalassemia;   Bipolar Disorder;   Breast Cancer;   Celiac Disease;   Cervical Cancer;   Chronic Inflammatory Demyelinating Polyneuropathy;   Chronic Kidney Diseases;   Chronic Obstructive Pulmonary Disease;   Colon Cancer;   Colorectal Cancer;   Crohn's Disease;   Cystic Fibrosis;   Depression;   Diabetes Mellitus;   Duchenne Muscular Dystrophy;   Endometriosis;   Epilepsy;   Facioscapulohumeral Muscular Dystrophy;   G6PD Deficiency;   General Anxiety Disorder;   Hepatitis B;   Hereditary Hemorrhagic Telangiectasia;   HIV/AIDS;   Human Papilloma Virus;   Huntington's Disease;   Idiopathic Thrombocytopenic Purpura;   Insomnia;   Kidney Cancer;   Leukemia;   Lung Cancer;   Lupus Nephritis;   Lymphoma;   Melanoma;   Multiple Myeloma;   Multiple Sclerosis;   Myositis;   Myotonic Dystrophy;   Ovarian Cancer;   Pancreatic Cancer;   Parkinson's Disease;   Polycystic Kidney Diseases;   Prostate Cancer;   Psoriasis;   Psoriatic Arthritis;   Rosacea;   Scleroderma;   Sickle Cell Anemia;   Sickle Cell Trait;   Sjogren's Syndrome;   Skin Cancer;   Spinal Muscular Atrophy;   Systemic Lupus Erythematosus;   Thrombotic Thrombocytopenic Purpura;   Trisomy 21;   Ulcerative Colitis
Intervention:  
Sponsor:   Sanguine Biosciences
Recruiting
Study of Chediak-Higashi Syndrome
Condition:   Chediak-Higashi Syndrome
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Recruiting
A Non-interventional Cohort Safety Study of Patients With hATTR-PN
Condition:   Hereditary Transthyretin Amyloidosis With Polyneuropthy
Intervention:   Other: Data Collection
Sponsors:   Akcea Therapeutics;   United BioSource, LLC
Recruiting
Videomicroscopy for the Prediction of Bleeding in Constitutional Haemorrhagic Diseases
Conditions:   Von Willebrand Diseases;   Glanzmann Thrombasthenia
Interventions:   Device: Sublingual videomicroscopy;   Biological: blood sample
Sponsor:   University Hospital, Lille
Not yet recruiting
Longitudinal Studies of Patient With FPDMM
Conditions:   Inherited Hematological Diseases;   Rare Diseases;   FPDMM
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Recruiting
Association Between Individual Clotting Factor Level Monitoring and the Risk of Bleeding Whilst Physical Active Conditions
Condition:   Hemophilia A
Intervention:   Other: Observation of the association between clotting factor level and risk of bleeding whilst physical activity
Sponsors:   Prof. Dr. Dr. Thomas Hilberg;   Takeda;   University of Bonn
Recruiting
ATHN 11: Liver Transplantation Outcomes Study
Conditions:   Hemophilia A and B;   Liver Transplantation
Intervention:  
Sponsors:   American Thrombosis and Hemostasis Network;   BioMarin Pharmaceutical
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;   Autoimmune Diseases;   Severe Aplastic Anemia
Intervention:   Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
Sponsors:   Center for International Blood and Marrow Transplant Research;   National Marrow Donor Program
Recruiting
A Study of the Burden of Illness and Treatment Patterns in Teenagers and Adults With Hereditary Angioedema
Condition:   Hereditary Angioedema
Intervention:  
Sponsor:   Takeda
Recruiting
Evaluating Effectiveness and Long Term Safety of Damoctocog Alfa Pegol in Patients, Who Have Been Diagnosed With Hemophilia A
Condition:   Hemophilia A
Intervention:   Drug: Damoctocog alfa pegol (Jivi, Bay94-9027)
Sponsor:   Bayer
Recruiting
Study to Learn More About the Physical Activity Level of Patients Suffering From Hemophilia A Treated With Damoctocog Alfa Pegol (LIFE ACTIVE Study)
Condition:   Hemophilia A
Intervention:   Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)
Sponsor:   Bayer
Recruiting
China Registry for Genetic / Metabolic Liver Diseases
Condition:   Genetic/Metabolic Liver Diseases
Intervention:   Drug: Standard of care
Sponsors:   Beijing Friendship Hospital;   Beijing YouAn Hospital;   Henan Provincial People's Hospital;   Beijing Ditan Hospital;   Hebei Medical University Third Hospital;   Peking University First Hospital;   Xinjiang Uygur Autonomous Region Traditional Chinese Medicine Hospital;   Nanfang Hospital of Southern Medical University;   Logistics University of Chinese People's Armed Police Forces;   Beijing Anzhen Hospital;   West China Second University Hospital of Sichuan University;   Jinshan Hospital Fudan University;   Fudan University
Recruiting
SEVENFACT® for Bleeding Events in Hemophilia With Inhibitors
Conditions:   Hemophilia A With Inhibitor;   Hemophilia B With Inhibitor
Intervention:   Drug: coagulation factor VIIa [recombinant]-jncw
Sponsors:   American Thrombosis and Hemostasis Network;   LFB USA, Inc.
Recruiting
Accessibility of Prophylaxis and On-demand Treatment for Persons With Haemophilia and Other Coagulation Deficiencies
Conditions:   Health Services Accessibility;   Blood Coagulation Factor Deficiencies
Intervention:  
Sponsors:   RESCUe - RESeau Cardiologie Urgence / RESUVal - RESeau des Urgences de la vallée du Rhône;   Hospices Civils de Lyon
Recruiting
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Conditions:   Hemophilia A;   Hemophilia B
Intervention:   Drug: PF-06741086
Sponsor:   Pfizer
Recruiting
Frequency of Hemorrhages Associated With the Functional Anomalies of Willebrand Factor in Emergency Patients
Condition:   Von Willebrand Diseases
Intervention:  
Sponsor:   University Hospital, Lille
Recruiting
Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery
Condition:   Congenital Antithrombin Deficiency
Intervention:   Drug: Atenativ
Sponsor:   Octapharma
Not yet recruiting
Open-Label Extension Study of Marstacimab in Hemophilia Participants With or Without Inhibitors
Conditions:   Hemophilia A;   Hemophilia B
Intervention:   Drug: PF-06741086
Sponsor:   Pfizer
Recruiting
A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD)
Condition:   Von Willebrand Disease (VWD)
Interventions:   Biological: rVWF;   Biological: rFVIII
Sponsors:   Baxalta now part of Shire;   Takeda Development Center Americas, Inc.
Recruiting
Efficacy, PK, Immunogenicity and Safety of Wilate in Severe Von Willebrand Disease VWD) Patients <6 Years of Age
Condition:   Von Willebrand Disease
Intervention:   Drug: wilate
Sponsor:   Octapharma
Recruiting
ATHN 8: Previously Untreated Patients (PUPs) Matter Study
Conditions:   Hemophilia;   Hemophilia A;   Hemophilia B;   Hemophilia A With Inhibitor;   Hemophilia B With Inhibitor;   Haemophilia B Without Inhibitor;   Haemophilia A Without Inhibitor;   Haemophilia
Intervention:  
Sponsors:   American Thrombosis and Hemostasis Network;   CSL Behring;   Octapharma;   Takeda
Recruiting
Effectiveness and Tolerability of Eqwilate in Real-life Conditions
Condition:   VWD - Von Willebrand's Disease
Intervention:   Biological: Eqwilate
Sponsor:   Octapharma
Recruiting
Treatment of Hemophilia A Patients With FVIII Inhibitors
Condition:   Hemophilia A
Interventions:   Biological: Nuwiq;   Biological: Octanate;   Biological: Wilate;   Biological: Emicizumab;   Biological: Recombinant factor VIIa (rFVIIa);   Biological: Activated prothrombin complex concentrate (aPCC)
Sponsors:   Emory University;   Octapharma
Recruiting
Evaluation of Immune Status Before and After Splenectomy in Immune Thrombocytopenia Patients
Conditions:   Thrombocytopenia;   Splenectomy
Intervention:   Procedure: splenectomy
Sponsor:   Zhang Lei
Recruiting
Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.
Condition:   Hemophilia A
Intervention:   Drug: Recombinant Human Coagulation FVIII
Sponsor:   Sinocelltech Ltd.
Recruiting
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
Gene Therapy for Chinese Hemophilia A
Conditions:   Hemophilia A;   Gene Therapy
Intervention:   Genetic: Injection of GS001
Sponsor:   Institute of Hematology & Blood Diseases Hospital
Recruiting
Phase III Expansion Trial for Determining the Safety and Efficacy of Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection in Adolescent and Adult Patients With Hemophilia A
Condition:   Hemophilia A
Intervention:   Drug: Recombinant Human Coagulation Factor VIII-Fc Fusion Protein for Injection
Sponsors:   Zhengzhou Gensciences Inc;   Jiangsu Gensciences lnc.
Recruiting
Study of TAK-672 in Participants With Acquired Hemophilia A
Condition:   Acquired Hemophilia A
Intervention:   Biological: TAK-672
Sponsor:   Takeda
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
Recruiting
Rofecoxib Efficacy and Safety Evaluation Trial in Hemophilic Arthropathy
Condition:   Hemophilic Arthropathy
Interventions:   Drug: TRM-201 (Rofecoxib);   Drug: Placebo
Sponsor:   Tremeau Pharmceuticals, Inc.
Recruiting
A Safety Study of STSP-0601 in Adult Patients With Hemophilia A or B With Inhibitor
Condition:   Hemophilia
Intervention:   Drug: STSP-0601 for Injection
Sponsor:   Staidson (Beijing) Biopharmaceuticals Co., Ltd
Recruiting
Impact of Von Willebrand Factor and Its Multimers on Angiogenesis
Condition:   Von Willebrand Diseases
Intervention:   Other: no intervention
Sponsor:   Nantes University Hospital
Not yet recruiting
A Study of TAK-660 in Surgical Procedures for People With Hemophilia A.
Condition:   Hemophilia A
Intervention:   Biological: PEGylated Recombinant Factor VIII
Sponsor:   Takeda
Recruiting