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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
Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A
Conditions:   Von Willebrand Disease, Type 3;   Concomitant VWD and Hemophilia
Intervention:   Drug: Emicizumab
Sponsors:   Bleeding and Clotting Disorders Institute Peoria, Illinois;   Genentech, Inc.
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
Embolization in Hereditary Coagulopathies
Conditions:   Hemophilia;   Embolization;   Hemarthrosis;   Clotting Factor Deficiency;   Synovitis;   Arthropathy
Intervention:   Device: embolization with spherical microparticles embosphere
Sponsors:   University of Sao Paulo General Hospital;   Merit Medical Systems, Inc.
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
Recruiting
Chronic Pain and Hemophilia
Conditions:   Chronic Pain;   Hemophilia
Intervention:   Other: questionnaires
Sponsor:   University Hospital, Clermont-Ferrand
Not yet 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
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
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.;   Hemab Therapeutics;   CSL Behring
Recruiting
Prospective Study of Antiplatelet and Anticoagulation Therapy in Hereditary Haemorrhagic Telangiectasia
Conditions:   Hereditary Hemorrhagic Telangiectasia;   Rendu Osler Disease
Intervention:   Other: monitoring the use of anticoagulant and/or antiplatelet therapy in patients with osler rendering disease
Sponsor:   University Hospital, Clermont-Ferrand
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
Belgian Antithrombin Deficiency Registry
Condition:   Antithrombin III Deficiency
Intervention:   Other: observation
Sponsor:   Universitair Ziekenhuis Brussel
Not yet recruiting
World Bleeding Disorders Registry
Conditions:   Hemophilia A;   Hemophilia B
Intervention:  
Sponsor:   World Federation of Hemophilia
Recruiting
TPO-Mimetic Use in Children for Hematopoietic Failure
Conditions:   Bone Marrow Failure Disorders;   Aplastic Anemia;   Thrombocytopenia;   Refractory Cytopenia of Childhood;   Myelodysplastic Syndrome(MDS)
Intervention:   Drug: Romiplostim
Sponsors:   Anjali Sharathkumar;   Amgen
Recruiting
Genotype-phenotype Association in Hereditary Hemorrhagic Telangiectasia
Conditions:   Hereditary Hemorrhagic Telangiectasia;   HHT;   Rendu Osler Weber Disease
Intervention:   Other: Registry
Sponsors:   Fundacion para la Investigacion Biomedica del Hospital Universitario Ramon y Cajal;   Sociedad Española De Medicina Interna
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
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
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
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
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;   Blood Disease Infection
Intervention:  
Sponsor:   Institute of Hematology & Blood Diseases Hospital
Recruiting
He-move-philia, Lifestyle Intervention for Patients With Hemophilia
Condition:   Hemophilia
Interventions:   Other: Lifestyle - group sessions;   Other: Lifestyle - individual coaching
Sponsor:   Radboud University Medical Center
Recruiting
Effects of Pazopanib on Hereditary Hemorrhagic Telangiectasia Related Epistaxis and Anemia (Paz)
Conditions:   Hereditary Hemorrhagic Telangiectasia;   Epistaxis;   Anemia;   Nosebleed;   HHT
Interventions:   Drug: Pazopanib;   Drug: Placebo oral capsule
Sponsors:   Cure HHT;   United States Department of Defense;   Food and Drug Administration (FDA)
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
Assess Safety and Efficacy of VAD044 in HHT Patients
Condition:   Hereditary Hemorrhagic Telangiectasia (HHT)
Intervention:   Drug: VAD044
Sponsor:   Vaderis Therapeutics AG
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
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
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
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
Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
Condition:   Thrombotic Thrombocytopenic Purpura (TTP)
Intervention:   Drug: TAK-755
Sponsor:   Takeda
Available
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
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
Condition:   Hemophilia A
Intervention:   Biological: Auto CD34+PBSC transduced with a lentiviral vector encoding a novel coagulation factor VIII transgene
Sponsors:   Christian Medical College, Vellore, India;   Dr. H. Trent Spencer, Professor, Emory University of Medicine, Atlanta Ga, 30322
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
Recruiting
Genetic Influence of Genetic Factors Influencing the Desmopressin's Efficacy in Mild/Moderate Hemophilia A
Conditions:   Hemophilia A, Mild;   Desmopressin;   Factor VIII
Intervention:   Drug: Desmopressin
Sponsor:   Groupe Maladies hémorragiques de Bretagne
Recruiting
Emotions in the Communication and Relationship Styles of Parents With Hemophilic Children.
Condition:   Hemophilia A
Intervention:  
Sponsor:   Fondazione Policlinico Universitario Agostino Gemelli IRCCS
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
An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A
Conditions:   Hemophilia A;   Prophylaxis of Bleeding
Intervention:   Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)
Sponsor:   Bayer
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
Emicizumab in Patients With Acquired Hemophilia A
Condition:   Acquired Hemophilia A
Intervention:   Drug: emicizumab
Sponsors:   University of Washington;   Genentech, Inc.
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
A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Condition:   Thrombotic Thrombocytopenic Purpura (TTP)
Intervention:   Biological: TAK-755
Sponsors:   Takeda;   Takeda Development Center Americas, Inc.;   Shire
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 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
Comparison of Efficiency of Closed Kinetic Chain Exercises Versus Proprioceptive Exercises Patient With Hemophilia
Conditions:   Hemophilia A;   Hemophilia B
Interventions:   Other: Closed Chain Exercise Group;   Other: Proprioceptive Exercise Group
Sponsor:   Bahçeşehir University
Not yet 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
Gaining Insight Into the Complexity of Pain in Patients With Haemophilia
Conditions:   Hemophilia A;   Hemophilia B
Intervention:   Diagnostic Test: Biopsychological pain assessment
Sponsors:   Universiteit Antwerpen;   Cliniques universitaires Saint-Luc- Université Catholique de Louvain;   University Hospital, Antwerp
Recruiting
Aspirin for Prophylaxis of TTP
Condition:   Thrombotic Thrombocytopenic Purpura
Interventions:   Drug: Aspirin tablet;   Drug: Placebo
Sponsor:   The First Affiliated Hospital of Soochow University
Not yet 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
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
PPI Supplementation to Fight ECtopIc Calcification in PXE
Condition:   Pseudoxanthoma Elasticum
Interventions:   Dietary Supplement: study treatment PPI;   Dietary Supplement: Placebo comparator
Sponsor:   Centre Hospitalier Universitaire de Nice
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
Study of Calcium-phosphate Complications Induced by the Administration of IV Iron Supplementation in Patients With Rendu-Osler Disease
Condition:   Rendu Osler Disease
Intervention:  
Sponsor:   University Hospital, Strasbourg, France
Recruiting
Baby Detect : Genomic Newborn Screening
Conditions:   Congenital Adrenal Hyperplasia;   Familial Hyperinsulinemic Hypoglycemia 1;   Phosphoglucomutase 1 Deficiency;   Maturity Onset Diabetes of the Young;   Cystic Fibrosis;   Hypophosphatasia, Infantile;   Congenital Hypothyroidism;   DAVID;   Pituitary Hormone Deficiency, Combined;   Diamond Blackfan Anemia;   Wiskott-Aldrich Syndrome;   Fanconi Anemia;   Hemophilia A;   Hemophilia B;   Glucose 6 Phosphate Dehydrogenase Deficiency;   Alpha-Thalassemia;   Sickle Cell Disease;   Shwachman-Diamond Syndrome;   Alpha 1-Antitrypsin Deficiency;   Inflammatory Bowel Disease 25, Autosomal Recessive;   Wilson Disease;   Progressive Familial Intrahepatic Cholestasis;   Crigler-Najjar Syndrome;   DIAR4;   Familial Chylomicronemia;   Lysosomal Acid Lipase Deficiency;   Familial Hemophagocytic Lymphocytosis;   Griscelli Syndrome;   Chediak-Higashi Syndrome;   Severe Congenital Neutropenia;   SCID;   Chronic Granulomatous Disease;   Menkes Disease;   X-ALD;   Smith-Lemli-Opitz Syndrome;   Ataxia With Vitamin E Deficiency;   THMD5;   THMD4;   Thiamine-Responsive Megaloblastic Anemia;   Thiamine Metabolism Dysfunction Syndrome 2;   GOT2 DEFICIENCY;   Cerebral Folate Transport Deficiency;   Segawa Syndrome, Autosomal Recessive;   Congenital Myasthenic Syndrome;   Metachromatic Leukodystrophy;   Sepiapterin Reductase Deficiency;   Dopamine Beta Hydroxylase Deficiency;   Glut1 Deficiency Syndrome;   Late-Infantile Neuronal Ceroid Lipofuscinosis;   Aromatic L-amino Acid Decarboxylase Deficiency;   Charcot-Marie-Tooth Disease, Type 6C;   Hereditary Hyperekplexia;   Brain Dopamine-Serotonin Vesicular Transport Disease;   Very Long Chain Hydroxy Acyl Dehydrogenase Deficiency;   Tyrosinemia, Type I;   Disaccharide Intolerance I;   Beta Ketothiolase Deficiency;   Phosphoglycerate Dehydrogenase Deficiency;   Succinyl-Coa 3-Oxoacid Transferase Deficiency;   Pyridoxine-5'-Phosphate Oxidase Deficiency;   Pyridoxine-Dependent Epilepsy;   Propionic Acidemia;   Pompe Disease;   Phenylalanine Hydroxylase Deficiency;   Ornithine Transcarbamylase Deficiency;   N Acetyl Glutamate Synthethase Deficiency;   Riboflavin Deficiency;   Maple Syrup Urine Disease;   Medium Chain Acyl CoA Dehydrogenase Deficiency;   Malonic Acidemia;   Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency;   Isovaleric Acidemia;   Phosphoserine Aminotransferase Deficiency;   Phosphoserine Phosphatase Deficiency;   Spatccm;   Hyperornithinemia-Hyperammonemia-Homocitrullinuria;   MRT8, FORMERLY;   S-Adenosylhomocysteine Hydrolase Deficiency;   Mucopolysaccharidosis VII;   Mucopolysaccharidosis VI;   Mucopolysaccharidosis IV A;   Mucopolysaccharidosis II;   Mucopolysaccharidosis I;   Transcobalamin Deficiency;   Isolated Methylmalonic Acidemia;   Cobalamin Deficiency;   Homocystinuria;   Holocarboxylase Synthetase Deficiency;   Fanconi Bickel Syndrome;   Glycogen Storage Disease;   Glycine Encephalopathy;   Glutaric Acidemia I;   Glucose Galactose Malabsorption;   Gaucher Disease, Type 1;   Galactosemias;   Fructosemia;   Fructose-1,6-Diphosphatase Deficiency;   Carbamoyl Phosphate Synthase 1 Deficiency;   Citrullinemia Type II;   Citrullinemia 1;   Creatine Deficiency Syndrome;   Systemic Primary Carnitine Deficiency;   Carnitine Palmitoyltransferase Deficiency 2;   Carnitine Palmitoyltransferase Deficiency 1;   Carnitine Acylcarnitine Translocase Deficiency;   Riboflavin Transporter Deficiency;   Branched-Chain Keto Acid Dehydrogenase Kinase Deficiency;   Andersen Tawil Syndrome;   Timothy Syndrome;   Jervell-Lange Nielsen Syndrome;   Catecholaminergic Polymorphic Ventricular Tachycardia;   Familial Hypertrophic Cardiomyopathy Type 4;   Pseudohypoaldosteronism, Type II;   Pseudohypoaldosteronism Type 1;   Primary Hyperoxaluria;   X Linked Hypophosphatemia;   Hereditary Nephrogenic Diabetes Insipidus;   Cystinosis;   Congenital Nephrotic Syndrome, Finnish Type;   Alport Syndrome;   Hereditary Retinoblastoma;   Biotinidase Deficiency;   Aciduria, Argininosuccinic;   Arginemia;   ACAD9 Deficiency;   3-Hydroxy 3-Methyl Glutaric Aciduria;   3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency
Intervention:  
Sponsors:   Laurent Servais;   Centre Hospitalier Universitaire de Liege;   University of Liege
Recruiting
A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa
Condition:   Hemophilia A, Severe
Intervention:   Drug: Efanesoctocog alfa
Sponsors:   Swedish Orphan Biovitrum;   Syneos Health
Not yet 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
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
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
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
A Study to Test a Medicine (Fitusiran) Injected Under the Skin for Preventing Bleeding Episodes in Male Adolescent or Adult Participants With Severe Hemophilia
Condition:   Hemophilia
Interventions:   Drug: Fitusiran;   Drug: Clotting factor concentrates (CFC) or bypassing agents (BPA);   Drug: Antithrombin concentrate (ATIIIC)
Sponsor:   Sanofi
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
Personalized Therapy of Patients Suffering From Rare Genodermatoses
Condition:   Rare Diseases
Intervention:   Other: If a targetable cytokine is increased in the lesional skin of a patient, this patient is treated with the respective antibody therapy (off-label use of approved drug).
Sponsor:   Johannes Kepler University of Linz
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
An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa
Conditions:   Hemophilia A;   Hemophilia B
Interventions:   Procedure: Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US);   Other: Haemophilia Joint Health Score (HJHS)
Sponsors:   Swedish Orphan Biovitrum;   IQVIA Pvt. Ltd
Not yet 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;   Jinshan Hospital Fudan University;   Fudan University
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
SCOPE HIM SCOPE HIM
Condition:   Hemophilia
Intervention:   Biological: Coagulation Factor VIIa (Recombinant)
Sponsor:   Laboratoire français de Fractionnement et de Biotechnologies
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 Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Conditions:   Hemophilia A;   Hemophilia B
Intervention:   Drug: marstacimab
Sponsor:   Pfizer
Recruiting
A Study of Recombinant Von Willebrand Factor (rVWF) (TAK-577) in Children With Severe Von Willebrand Disease (vWD)
Condition:   Von Willebrand Disease (VWD)
Interventions:   Biological: Recombinant von Willebrand Factor (rVWF);   Biological: ADVATE
Sponsor:   Takeda
Not yet recruiting
Efficacy of Atenativ in Patients With Congenital Antithrombin Deficiency Undergoing Surgery or Delivery
Condition:   Congenital Antithrombin Deficiency
Intervention:   Drug: Atenativ
Sponsor:   Octapharma
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
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
Biomechanical and Microstructural Properties of Ascending Aortic Aneurysms
Condition:   Thoracic Aortic Aneurysms
Interventions:   Other: Biomechanical and microstructural analysis of ATAA;   Radiation: ECG-gated CT
Sponsor:   Universitaire Ziekenhuizen KU Leuven
Recruiting
A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers
Condition:   Von Willebrand Disease (VWD)
Intervention:   Other: No Intervention
Sponsor:   Takeda
Recruiting
A Trial That Evaluates Disease Characteristics in Hemophilia B Adult Male Participants Receiving Prophylaxis With Standard of Care Factor IX Protein (FIX) Replacement Therapy
Condition:   Hemophilia B
Intervention:   Other: Non-Interventional
Sponsor:   Regeneron Pharmaceuticals
Not yet 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
Gene Therapy for Chinese Hemophilia A
Conditions:   Hemophilia A;   Gene Therapy
Intervention:   Genetic: Injection of GS001
Sponsor:   Institute of Hematology & Blood Diseases Hospital
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
Acquired Hemophilia A and Autoimmunity. Study of Lymphocyte Populations and Myeloid-Derived Suppressor Cells
Condition:   Hemophilia A
Intervention:   Other: no intervention
Sponsor:   Nantes University Hospital
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
Update in VWD Laboratory Diagnosis
Condition:   VWD - Von Willebrand's Disease
Intervention:  
Sponsor:   Assiut University
Not yet 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
The Efficacy and Safety of ZS802 in Chinese Hemophilia A Patients.
Condition:   Hemophilia A
Intervention:   Genetic: ZS802
Sponsor:   Institute of Hematology & Blood Diseases Hospital
Not yet recruiting