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
|