Home News Info Guidelines Trials EUHASS Travel Products Organisations Genes QC FAQs About Links

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:
A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors
Conditions:   Congenital Bleeding Disorder;   Haemophilia A With Inhibitors;   Haemophilia B With Inhibitors
Interventions:   Drug: Concizumab;   Drug: Eptacog alfa
Sponsor:   Novo Nordisk A/S
Not yet recruiting - verified June 2017
A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors
Conditions:   Haemostasis;   Haemophilia A
Interventions:   Drug: Concizumab;   Drug: Turoctocog alfa
Sponsor:   Novo Nordisk A/S
Not yet recruiting - verified June 2017
Study Investigating Novoeight®/NovoEight® (Turoctocog Alfa) in Mexican Haemophilia A Patients
Conditions:   Congenital Bleeding Disorder;   Haemophilia A
Intervention:   Drug: turoctocog alfa
Sponsor:   Novo Nordisk A/S
Not yet recruiting - verified June 2017
A Multicenter, Post-Marketing Registry Study of COAGADEX® in the Peri-operative Management of Patients With Moderate or Severe Hereditary Factor X Deficiency Undergoing Major Surgery
Condition:   Factor 10 Deficiency
Intervention:   Drug: Major surgery
Sponsor:   Bio Products Laboratory
Not yet recruiting - verified May 2017
Factor XIII in Major Burns Coagulation
Conditions:   Burns;   Coagulation Disorder;   Factor XIII Deficiency;   Cicatrization;   Endothelial Dysfunction
Intervention:   Diagnostic Test: Coagulation tests
Sponsor:   Hospital Universitari Vall d'Hebron Research Institute
Not yet recruiting - verified May 2017
A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors
Condition:   Hemophilia A
Intervention:   Drug: Emicizumab
Sponsor:   Hoffmann-La Roche
Not yet recruiting - verified June 2017
Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia
Conditions:   Hemophilia A;   Hemophilia B
Intervention:   Other: Non-treatment, seroprevalence
Sponsor:   Baxalta US Inc.
Recruiting - verified June 2017
A Prospective Study on the Incidence and Related Risk Factors of Infantile Hemangioma in China
Conditions:   Infantile Hemangioma;   Capillary Hemangioma;   Capillary Hemangiomas;   Hemangioma, Capillary Infantile;   Strawberry Hemangioma
Intervention:  
Sponsor:   Zhujiang Hospital
Recruiting - verified June 2017
Dose-escalation Study to Investigate the Safety, PK, and PD of ISU304/CB2679d in Hemophilia B Patients
Condition:   Hemophilia B
Interventions:   Biological: ISU304/CB2679d;   Biological: BeneFix
Sponsors:   ISU Abxis Co., Ltd.;   Catalyst Biosciences
Recruiting - verified June 2017
ADYNOVATE Drug Use-Results Survey
Condition:   Hemophilia A
Intervention:   Biological: ADYNOVATE
Sponsors:   Baxalta US Inc.;   Baxalta Japan Limited
Recruiting - verified May 2017
Hemophilia Mobile App Usability Pilot
Condition:   Hemophilia
Intervention:   Device: SureSource Engage application
Sponsors:   Healthcare Innovation Technology Lab;   Bayer
Recruiting - verified May 2017
Bleeding Risk Evaluation in Haemophilia Patients Under Antiplatelet Therapies
Condition:   Hemophilia
Intervention:   Other: Non interventional study
Sponsor:   Nantes University Hospital
Not yet recruiting - verified May 2017
An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors
Condition:   Hemophilia A
Intervention:   Biological: Emicizumab
Sponsor:   Genentech, Inc.
Available - verified June 2017
China Registry for Genetic / Metabolic Liver Diseases
Condition:   Genetic/Metabolic Liver Diseases
Intervention:   Drug: Standard of care
Sponsors:   Beijing Friendship Hospital;   Beijing YouAn Hospital;   Henan Provincial Hospital;   Beijing Ditan Hospital;   Hebei Medical University Third Hospital;   Peking University First Hospital;   Xinjiang Uygur Autonomous Region Traditional Chinese Medicine Hospital;   Nanfang Hospital of Southern Medical University;   Affiliated Hospital, Logistics University of People's Armed Police Force;   Beijing Anzhen Hospital, Capital Medical University;   West China Second University Hospital of Sichuan University;   Jinshan Hospital of Fudan University;   Fudan University
Recruiting - verified April 2017
DDAVP vs. Exercise
Condition:   Children With Mild Hemophilia A
Interventions:   Drug: DDAVP intervention;   Behavioral: Exercise Intervention
Sponsors:   The Hospital for Sick Children;   Canadian Hemophilia Society;   St. Michael's Hospital, Toronto
Not yet recruiting - verified April 2017
Efficacy and Safety of a 0.1% Tacrolimus Nasal Ointment as a Treatment for Epistaxis in Hemorrhagic Hereditary Telangiectasia (HHT)
Condition:   Hemorrhagic Hereditary Telangiectasia (HHT)
Interventions:   Drug: Protopic® (Tacrolimus) 0.1% ointment;   Drug: Placebo
Sponsor:   Hospices Civils de Lyon
Not yet recruiting - verified May 2017
Study of rFVIIIFc for ITI in Haemophilia A Patients With Inhibitors Who Have Failed Previous ITI Therapies
Condition:   Hemophilia A
Intervention:   Biological: Recombinant coagulation factor (rFVIIIFc)
Sponsors:   Swedish Orphan Biovitrum;   Bioverativ Therapeutics Inc.
Not yet recruiting - verified March 2017
A Trial Comparing Nonacog Beta Pegol (N9-GP) and ALPROLIX® in Patients With Haemophilia B
Conditions:   Congenital Bleeding Disorder;   Haemophilia B
Interventions:   Drug: N9-GP;   Drug: ALPROLIX®
Sponsor:   Novo Nordisk A/S
Recruiting - verified June 2017
Response of Continuous Recombinant Antithrombin Infusion in Postcardiotomy ECMO Patients
Condition:   Antithrombin Deficiency Type 2
Intervention:   Drug: ATryn continuous infusion
Sponsors:   Mayo Clinic;   rEVO Biologics
Not yet recruiting - verified April 2017
Study Comparing the Pharmacokinetic of Biosimilar Eptacog Alfa With Novoseven®, in Patients With Congenital Factor VII Deficiency
Condition:   Factor VII Deficiency
Interventions:   Biological: Eptacog alfa, biosimilar and reference medicinal product Novoseven;   Biological: Eptacog alfa biosimilar
Sponsor:   AryoGen Pharmed Co.
Recruiting - verified March 2017
Gingival Bleeding and Von Willebrand Disease Typ 2 and 3
Conditions:   Gingival Bleeding;   Von Willebrand Diseases
Intervention:  
Sponsor:   Goethe University
Recruiting - verified March 2017
Alphanate in Immune Tolerance Induction Therapy
Condition:   Hemophilia A, Congenital
Intervention:   Biological: Alphanate
Sponsor:   Grifols Therapeutics Inc.
Recruiting - verified May 2017
A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment
Condition:   Hemophilia A
Intervention:   Biological: rFVIIIFc
Sponsor:   Bioverativ Therapeutics Inc.
Not yet recruiting - verified March 2017
A Study to Evaluate the Real-world Usage and Effectiveness of Elocta and Alprolix in Patients With Haemophilia A or B
Conditions:   Haemophilia A;   Haemophilia B
Interventions:   Drug: ELOCTA;   Drug: ALPROLIX
Sponsor:   Swedish Orphan Biovitrum
Recruiting - verified May 2017
Frequency of Hemorrhages Associated With the Functional Anomalies of Willebrand Factor in Emergency Patients
Condition:   Von Willebrand Diseases
Intervention:  
Sponsor:   University Hospital, Lille
Not yet recruiting - verified June 2017
Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A
Condition:   Hemophilia A
Intervention:   Biological: SB-525
Sponsor:   Sangamo Therapeutics
Recruiting - verified June 2017
Non Substitutive Strategies to Improve Haemophilia Care in Developing Countries. Experience in Ivory Coast.
Conditions:   Haemophilia;   Developing Countries
Interventions:   Other: Education;   Diagnostic Test: Improve biological diagnosis and follow-up of haemophilia;   Drug: Implement haemostatic non substitutive care of haemophilia;   Other: Implement non haemostatic care of haemophilia
Sponsors:   Cliniques universitaires Saint-Luc- Université Catholique de Louvain;   World Federation of Hemophilia
Recruiting - verified February 2017
FIBrinogen REplenishment in Surgery
Condition:   Deficiency; Fibrinogen, Acquired
Interventions:   Drug: Octafibrin;   Drug: Cryoprecipitate
Sponsor:   University Health Network, Toronto
Recruiting - verified February 2017
A Gene Transfer Study for Hemophilia A
Condition:   Hemophilia A
Intervention:   Genetic: SPK-8011
Sponsor:   Spark Therapeutics
Recruiting - verified April 2017
Safety, Tolerability, and Pharmacokinetics Study of Turoctocog Alfa Pegol Injected Under the Skin in Patients With Haemophilia A
Conditions:   Congenital Bleeding Disorder;   Haemophilia A
Intervention:   Drug: turoctocog alfa pegol
Sponsor:   Novo Nordisk A/S
Recruiting - verified June 2017
A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
Conditions:   Wiskott-Aldrich Syndrome;   Hematopoietic Stem Cell Transplantation;   Graft Failure
Interventions:   Biological: G-CSF for Conditioning before HSCT.;   Biological: Plerixafor for Conditioning before HSCT.
Sponsor:   Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Recruiting - verified January 2017
Fascial Therapy in Elbow Hemophilic Arthropathy
Condition:   Hemophilia
Intervention:   Other: Experimental group
Sponsor:   Real Fundación Victoria Eugenia
Not yet recruiting - verified January 2017
MRA With Feraheme in HHT
Condition:   Hereditary Hemorrhagic Telangiectasia
Intervention:   Procedure: Feraheme MRI/MRA
Sponsor:   University of California, Los Angeles
Recruiting - verified March 2017
Immunmodulation in Patients With HHT
Condition:   Hereditary Haemorrhagic Telangiectasia (HHT)
Intervention:   Other: blood sample
Sponsor:   University Hospital, Essen
Recruiting - verified December 2016
UK - EHL Outcomes Registry
Conditions:   Hemophilia A;   Hemophilia B
Intervention:  
Sponsor:   Royal Free Hospital NHS Foundation Trust
Not yet recruiting - verified October 2016
Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®
Conditions:   Hemophilia A;   Hemophilia;   Factor VIII Deficiency
Intervention:   Drug: Octocog Alfa
Sponsors:   Spanish Society of Thrombosis and Haemostasis;   Baxalta US Inc.;   Unidad de Ensayos Clínicos e Investigación Clínica. Hospital La Paz;   Instituto de Investigación Hospital Universitario La Paz
Not yet recruiting - verified December 2016
A Study to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Emicizumab Given Every 4 Weeks in Participants With Hemophilia A
Condition:   Hemophilia A
Intervention:   Drug: Emicizumab
Sponsors:   Hoffmann-La Roche;   Chugai Pharmaceutical
Recruiting - verified May 2017
Individualizing Hemophilia Bypassing Agent Therapy Utilizing Thromboelastography
Condition:   Hemophilia A With Inhibitors
Intervention:   Other: Thromboelastography-Guided Treatment Regimen
Sponsor:   Children's Hospital Los Angeles
Not yet recruiting - verified December 2016
Gene Therapy for Haemophilia A.
Condition:   Hemophilia A
Intervention:   Biological: AAV2/8-HLP-FVIII-V3
Sponsors:   University College, London;   Medical Research Council
Recruiting - verified June 2017
Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products
Condition:   Hemophilia A
Interventions:   Drug: efmoroctocog alfa;   Drug: Factor VIII
Sponsor:   Swedish Orphan Biovitrum
Recruiting - verified February 2017
Study With Atezolizumab Plus Bevacizumab in Patients With Chemotherapy Resistant, MSI-like, Colorectal Cancer
Conditions:   MSI;   ColoRectal Cancer;   Chemotherapy;   Resistance, APC
Interventions:   Drug: Atezolizumab;   Drug: Bevacizumab
Sponsors:   Vall d'Hebron Institute of Oncology;   European Organisation for Research and Treatment of Cancer - EORTC
Not yet recruiting - verified May 2017
rVWF IN PROPHYLAXIS
Condition:   Von Willebrand Disease
Interventions:   Biological: von Willebrand factor (Recombinant);   Biological: Antihemophilic Factor (Recombinant)
Sponsor:   Baxalta US Inc.
Not yet recruiting - verified November 2016
PF-06741086 Multiple Dose Study in Severe Hemophilia
Condition:   Hemophilia A or B
Interventions:   Biological: PF-06741086;   Biological: PF-06741086;   Biological: PF-06741086;   Biological: PF-06741086
Sponsor:   Pfizer
Recruiting - verified June 2017
Long-Term Safety, Tolerability, and Efficacy of DTX101 (AAVrh10FIX) in Adults With Moderate/Severe to Severe Hemophilia B
Condition:   Hemophilia B
Intervention:  
Sponsor:   Dimension Therapeutics
Recruiting - verified January 2017
Efficacy and Safety of Turoctocog Alfa for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A
Conditions:   Congenital Bleeding Disorder;   Haemophilia A
Interventions:   Drug: turoctocog alfa;   Drug: turoctocog alfa
Sponsor:   Novo Nordisk A/S
Recruiting - verified May 2017
Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
Condition:   Hemophilia A
Intervention:  
Sponsor:   Octapharma
Recruiting - verified November 2016
BAX 111 rVWF in Pediatrics
Condition:   Von Willebrand Disease
Interventions:   Biological: von Willebrand factor (Recombinant);   Biological: Antihemophilic Factor (Recombinant)
Sponsor:   Baxalta US Inc.
Not yet recruiting - verified October 2016
Graded TTCE for Post-Embolization PAVM Monitoring
Condition:   Hereditary Hemorrhagic Telangiectasia (HHT)
Intervention:   Other: Transthoracic Contrast Echocardiogram
Sponsor:   University of Pennsylvania
Recruiting - verified October 2016
Low VW Activity in Adolescent HMB
Condition:   Von Willebrand Factor Deficiency
Interventions:   Other: Genetic Analysis;   Other: Medical Record Data Abstraction;   Other: Pictorial Blood Assessment Chart (PBAC) score;   Other: Complete Bleeding Symptom ISTH Bleeding Assessment Tool
Sponsors:   Baylor College of Medicine;   Texas Children's Hospital;   Baxalta US Inc.
Not yet recruiting - verified October 2016
Clinical Study to Investigate the PK, Efficacy, and Safety of Wilate in Patients With Severe Hemophilia A
Condition:   Severe Hemophilia A
Intervention:   Drug: Wilate
Sponsor:   Octapharma
Recruiting - verified March 2017
Exit Interviews to Assess Impact of Infusion Frequency in Hemophilia A
Condition:   Hemophilia A
Intervention:   Biological: BAY94-9027
Sponsor:   Bayer
Not yet recruiting - verified June 2017
Drug Use Investigation of Kovaltry in Hemophilia A Patients
Condition:   Hemophilia A
Intervention:   Drug: Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973)
Sponsor:   Bayer
Recruiting - verified June 2017
RIXUBIS Drug Use-Result Survey (Japan)
Condition:   Hemophilia B
Intervention:   Biological: RIXUBIS
Sponsor:   Baxalta US Inc.
Recruiting - verified May 2017
Efficacy and Safety Study of SCT800 for On-demand Treatment With in Patients With Hemophilia A
Condition:   Hemophilia A
Intervention:   Biological: SCT800
Sponsor:   Sinocelltech Ltd.
Not yet recruiting - verified September 2016
Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea
Condition:   Hemophilia B
Intervention:   Biological: RIXUBIS
Sponsor:   Baxalta US Inc.
Recruiting - verified December 2016
German Pediatric Hemophilia Research Database
Conditions:   Hemophilia A;   Hemophilia B;   Children;   Drug Specific Antibodies
Intervention:   Other: documentation only
Sponsors:   Ludwig-Maximilians - University of Munich;   Pediatric Committee of the German Thrombosis and Hemostasis Research Society
Recruiting - verified January 2017
Evaluate Efficacy and Safety of Recombinant Factor VIII (rFVIII)Treatment of Severe or Moderately Severe Hemophilia A
Condition:   Hemophilia A
Interventions:   Drug: Recombinant Factor VIII (50 IU/kg);   Drug: Recombinant Factor VIII (On-demand treatment)
Sponsor:   Jiangsu Chia-tai Tianqing Pharmaceutical Co.,Ltd
Recruiting - verified October 2016
A Clinical Phenotype Based Individualized Prophylaxis in Chinese Hemophilia A Children
Condition:   Haemophilia
Intervention:   Other: Observe the patient's condition and then change the regimen
Sponsor:   Beijing Children's Hospital
Recruiting - verified December 2016
Treatment of Nasal Staphylococcus Aureus Colonization in Patients With HHT
Conditions:   Hereditary Hemorrhagic Telangiectasia;   Epistaxis
Interventions:   Drug: Mupirocin;   Other: Placebo
Sponsor:   Hospital Italiano de Buenos Aires
Not yet recruiting - verified November 2016
Octreotide in Patients With GI Bleeding Due to Rendu-Osler-Weber
Conditions:   Hereditary Hemorrhagic Telangiectasia;   Gastrointestinal Hemorrhage;   Anemia
Intervention:   Drug: Octreotide LAR
Sponsors:   Radboud University;   St. Antonius Hospital
Recruiting - verified August 2016
Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A
Condition:   Hemophilia A
Interventions:   Biological: SCT800;   Biological: Xyntha
Sponsor:   Sinocelltech Ltd.
Not yet recruiting - verified August 2016
BAX 802 in CHA With Inhibitors
Condition:   Hemophilia A
Intervention:   Biological: Antihemophilic Factor (Recombinant), Porcine Sequence (BAX802)
Sponsor:   Baxalta US Inc.
Recruiting - verified April 2017
Performance Evaluation of Von Willebrand:Collagen-Binding Assays to Diagnose Von Willebrand Factor Deficiency in Patients With Increased Risk of Bleeding
Condition:   Von Willebrand Disease
Intervention:   Other: Non interventional study
Sponsor:   Nantes University Hospital
Not yet recruiting - verified June 2016
A Single-dose, Dose-escalation Study of a Long-acting MOD-5014 in Healthy Adult Male
Condition:   Hemophilia A or B With Inhibitors
Interventions:   Biological: MOD-5014;   Other: MOD-5014 Placebo
Sponsor:   Opko Biologics
Not yet recruiting - verified August 2016
Effectiveness of Psychological Interventions in Haemophilia
Condition:   Haemophilia
Interventions:   Behavioral: Hypnosis;   Behavioral: Cognitive-Behavioral Therapy
Sponsors:   University of Minho;   Hospital Sao Joao
Not yet recruiting - verified August 2016
Determinants and Consequences of the Transition to Adulthood Among Adolescents With Haemophilia
Condition:   Haemophilia
Intervention:   Other: Patient questionnaire
Sponsor:   Assistance Publique Hopitaux De Marseille
Recruiting - verified August 2016
The Comorbidity of Benign Hypermobility Joint Syndrome and Functional Constipation in Children
Conditions:   Benign Hypermobility Syndrome;   Functional Constipation
Intervention:  
Sponsor:   Medical University of Warsaw
Recruiting - verified March 2017
Molecular and Clinical Profile of Von Willebrand Disease in Spain
Condition:   Von Willebrand Disease
Intervention:   Genetic: VWF gene analysis
Sponsors:   Spanish Society of Thrombosis and Haemostasis;   Francisco Vidal Pérez;   Maria Fernanda López Fernández;   Almudena Pérez Rodríguez;   Irene Corrales Insa;   Ana Rosa Cid Haro
Not yet recruiting - verified August 2016
Human Fibrinogen Concentrate in Pediatric Cardiac Surgery
Conditions:   Hypofibrinogenemia;   Afibrinogenemia;   Bleeding Disorders
Interventions:   Drug: RiaStAP;   Drug: Saline
Sponsor:   Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Recruiting - verified April 2017
Effectiveness of the Myofascial Therapy in the Hemophilic Arthropathy of Ankle
Condition:   Haemophilia
Intervention:   Other: Physiotherapy sessions
Sponsor:   Real Fundación Victoria Eugenia
Not yet recruiting - verified July 2016
Study Evaluating "Real World" Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog Alfa) for Routine Prophylaxis
Condition:   Hemophilia A, Congenital
Intervention:   Biological: Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973
Sponsor:   Bayer
Recruiting - verified May 2017
Does the Thrombin Generation Test Performed During the Pharmacokinetic Profile of the Substitutive Factor VIII Bring Benefits to the Personalized Treatment of Pediatric Patients and Adult Hemophilia A Patients Under Prophylaxis ?
Condition:   Hemophilia
Interventions:   Device: Chronometric method;   Device: Chromogenic method;   Device: Thrombin generation test (TGT)
Sponsor:   Brugmann University Hospital
Not yet recruiting - verified June 2016
World Bleeding Disorders Registry
Condition:   Hemophilia A and B
Intervention:  
Sponsor:   World Federation of Hemophilia
Not yet recruiting - verified May 2016
TGA (Thrombin Generation Assay) and Prophylaxis in Haemophilia
Condition:   Haemophilia A
Intervention:   Other: Thrombin generation measurement.
Sponsor:   Hospices Civils de Lyon
Recruiting - verified February 2017
Immunosuppression During Penetrating Keratoplasty, Using a Subconjunctival Implant Releasing Dexamethasone : Tolerance and Safety Pilot Study
Conditions:   Keratoconus;   Fuchs' Endothelial Dystrophy;   Congenital Hereditary Stromal Dystrophy of the Cornea
Intervention:   Drug: Dexamethasone implant OZURDEX
Sponsor:   Centre Hospitalier Universitaire de Saint Etienne
Recruiting - verified January 2017
The Hemophilia Ultrasound Project
Conditions:   Hemophilia A;   Hemophilia B
Interventions:   Other: Ankle and Knee Ultrasound Joint Assessment;   Other: Ankle and Knee Magnetic Resonance Imaging
Sponsors:   University of Miami;   Baxalta US Inc.
Recruiting - verified August 2016
The Proteins of the Contact Activation System
Conditions:   Factor XI Deficiency;   Factor XII Deficiency
Intervention:   Other: Global Haemostasis assays
Sponsor:   Hospices Civils de Lyon
Recruiting - verified May 2016
Factor Product Utilization and Health Outcomes in Patients With Hemophilia
Conditions:   Hemophilia A, Congenital;   Hemophilia B, Congenital
Intervention:  
Sponsors:   University of British Columbia;   Biogen
Recruiting - verified June 2016
Topical Anesthesia for Closed PKP vs Retrobulbar Anesthesia for Open-sky PKP
Conditions:   Corneal Opacity;   Keratitis, Herpetic;   Corneal Ulcer;   Corneal Dystrophies, Hereditary
Interventions:   Procedure: closed PKP under topical anesthesia;   Procedure: open-sky PKP under retrobulbar anesthesia;   Drug: Anti-Rejection Agents;   Drug: Anti-Inflammatory Agents
Sponsor:   Wenzhou Medical University
Recruiting - verified July 2016
A Progressive Resistance Training Program in Patients With Haemophilia
Condition:   Haemophilia
Interventions:   Other: Progressive resistance training;   Other: Normal daily activities
Sponsors:   University of Valencia;   Baxalta US Inc.
Not yet recruiting - verified October 2016
Low Von Willebrand in Ireland Cohort Study
Condition:   Von Willebrand Factor, Deficiency
Intervention:  
Sponsor:   St. James's Hospital, Ireland
Recruiting - verified May 2017
Investigation of the Genetics of Hematologic Diseases
Conditions:   Bone Marrow Failure Syndromes;   Erythrocyte Disorder;   Leukocyte Disorder;   Hemostasis;   Blood Coagulation Disorder;   Sickle Cell Disease;   Dyskeratosis Congenita;   Diamond-Blackfan Anemia;   Congenital Thrombocytopenia;   Severe Congenital Neutropenia;   Fanconi Anemia
Intervention:  
Sponsors:   St. Jude Children's Research Hospital;   Boston Children’s Hospital
Recruiting - verified September 2016
FEIBA Reconstitution Volume Reduction and Faster Infusion Study
Condition:   Hemophilia A or B With Inhibitors
Intervention:   Biological: Factor VIII Inhibitor Bypassing Activity (nanofiltered, vapor heat-treated)
Sponsor:   Baxalta US Inc.
Not yet recruiting - verified October 2016
Improved Factor VIII Inhibitor Evaluation
Conditions:   Hemophilia;   Hemophilia A
Intervention:  
Sponsors:   Boston VA Research Institute, Inc.;   Baxalta US Inc.;   Emory University;   University of Michigan
Not yet recruiting - verified March 2016
Symptoms and Treatment Results in Hereditary Hemorrhagic Telangiectasia
Condition:   Hereditary Haemorrhagic Telangiectasia (HHT)
Intervention:   Other: questionnaire
Sponsor:   University Hospital, Essen
Recruiting - verified December 2016
Efficacy & Safety of REFACTO AF in Prophylaxis Treatment
Condition:   Severe Hemophilia A
Intervention:  
Sponsor:   Pfizer
Not yet recruiting - verified June 2017
Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B
Condition:   Hemophilia B
Intervention:   Biological: SB-FIX
Sponsor:   Sangamo Therapeutics
Recruiting - verified January 2017
Personalized Prophylactic Treatment With Advate® in Severe or Moderate Haemophilia A Patients
Conditions:   Hemophilia A;   Factor VIII
Intervention:   Drug: Recombinant VIII factor
Sponsor:   Hospital Universitario La Fe
Recruiting - verified August 2016
BAX 855 Previously Untreated Patient (PUP)
Condition:   Hemophilia A
Intervention:   Biological: PEGylated Recombinant Factor VIII
Sponsor:   Baxalta US Inc.
Recruiting - verified April 2017
Post-Marketing Non-Interventional Safety Evaluation of Obizur in the Treatment of Bleeding Episodes for Patients With Acquired Hemophilia A
Condition:   Acquired Hemophilia A
Intervention:   Biological: OBIZUR
Sponsor:   Baxalta US Inc.
Recruiting - verified August 2016
Weight-based Dosing in Hemophilia A
Condition:   Hemophilia
Intervention:   Drug: rFVIII
Sponsor:   University of Pittsburgh
Recruiting - verified May 2017
Minimize Menorrhagia in Women With Type 1 Von Willebrand Disease
Condition:   Type 1 Von Willebrand Disease
Interventions:   Drug: recombinant von Willebrand factor;   Drug: tranexamic acid
Sponsors:   University of Pittsburgh;   University of North Carolina;   Carnegie Mellon University;   Duke University
Not yet recruiting - verified June 2017
Biomarker for Patient With Alport Disease or Highly Suspected for Alport Disease
Conditions:   Nephritis, Hereditary;   Alport Syndrome;   Alport Syndrome, Autosomal Dominant;   Alport Syndrome, Autosomal Recessive;   Hematuria-Nephropathy-Deafness Syndrome
Intervention:  
Sponsors:   University of Rostock;   Centogene AG Rostock
Recruiting - verified September 2016
Prospective Pilot Study of Floseal for the Treatment of Anterior Epistaxis in Patients With (HHT)
Conditions:   Hereditary Hemorrhagic Telangiectasia (HHT);   Epistaxis
Interventions:   Drug: Floseal;   Other: Packing
Sponsors:   St. Michael's Hospital, Toronto;   The Ottawa Hospital
Recruiting - verified January 2017
Ultrasonography in Hemophilic Joint Disease and Serum Markers
Conditions:   Hemophilia A;   Hemophilia B;   Hemarthroses
Intervention:   Other: ultrasound
Sponsors:   Northwell Health;   National Cancer Institute (NCI);   University of California, San Diego
Recruiting - verified July 2016
Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)
Conditions:   Lymphoma;   Childhood Lymphoma;   Solid Tumor;   Childhood Solid Tumor;   Neuroblastoma;   Ewing Sarcoma;   Hodgkin Lymphoma;   Non-Hodgkin Lymphoma;   Rhabdomyosarcoma;   Soft Tissue Sarcoma;   Medulloblastoma;   Germ Cell Tumor;   Wilms Tumor;   Brain Neoplasms;   Medulloblastoma, Childhood;   Neuroectodermal Tumors, Primitive
Interventions:   Drug: Genistein;   Drug: Placebo
Sponsor:   University of Virginia
Recruiting - verified June 2017
Extension at 10 Years of the: "Observational Study Evaluating Efficacy and Costs of Secondary Prophylaxis vs On-demand Therapy With Kogenate Bayer in Patients With Severe Haemophilia A."
Condition:   Hemophilia A
Interventions:   Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222);   Drug: Recombinant Factor VIII (Kogenate FS, BAY14-2222)
Sponsor:   Bayer
Recruiting - verified June 2017
Vasculitis Pregnancy Registry
Conditions:   Vasculitis;   Behcet's Disease;   CNS Vasculitis;   Cryoglobulinemic Vasculitis;   Eosinophilic Granulomatosis With Polyangiitis (EGPA);   Churg-Strauss Syndrome (CSS);   Granulomatosis With Polyangiitis (GPA);   Wegener's Granulomatosis;   IgA Vasculitis;   Henoch-Schoenlein Purpura (HSP);   Microscopic Polyangiitis (MPA);   Polyarteritis Nodosa (PAN);   Takayasu Arteritis (TAK);   Urticarial Vasculitis;   Systemic Vasculitis
Intervention:   Other: Online questionnaires
Sponsors:   University of Pennsylvania;   Duke University;   University of South Florida
Recruiting - verified April 2017
Validation of a Specific Measure for Assessment of Compliance With Treatment in Patients With Hemophilia
Condition:   Haemophilia
Intervention:   Other: Patients with haemophilia
Sponsors:   Real Fundación Victoria Eugenia;   Medtep Inc.
Recruiting - verified October 2015
Individualizing Hemophilia Prophylaxis Using Thromboelastography
Condition:   Hemophilia A
Intervention:   Other: Thromboelastography/Thromboelastometry-Guided Treatment Regimen
Sponsor:   Children's Hospital Los Angeles
Recruiting - verified July 2016
A Single Escalating Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors
Conditions:   Hemophilia A;   Hemophilia B
Intervention:   Drug: BAY1093884
Sponsor:   Bayer
Recruiting - verified June 2017
Study of Voncento® in Subjects With Von Willebrand Disease
Condition:   Von Willebrand Disease
Intervention:   Biological: Voncento
Sponsor:   CSL Behring
Recruiting - verified June 2017
ATHN 2: Factor Switching Study
Condition:   Hemophilia
Interventions:   Biological: Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013;   Biological: Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013
Sponsors:   American Thrombosis and Hemostasis Network;   Bioverativ (Biogen);   Shire (Baxalta);   CSL Behring
Recruiting - verified June 2017
Basic Study of Combination Therapy Based on APCC-induced FVllI Activation in Hemophilia A Patients With Inhibitors
Condition:   Hemophilia A With Inhibitor
Intervention:  
Sponsors:   Nara Medical University;   Baxalta US Inc.
Not yet recruiting - verified September 2015
An Open-label Extension Study of an Investigational Drug, ALN-AT3SC, in Patients With Moderate or Severe Hemophilia A or B
Conditions:   Hemophilia A;   Hemophilia B
Intervention:   Drug: ALN-AT3SC
Sponsor:   Alnylam Pharmaceuticals
Recruiting - verified July 2016
A Study of the Safety and Efficacy of Coagulation Factor VIIa (Recombinant) LR769 for the Prevention of Excessive Bleeding in Congenital Hemophilia A or B Patients With Inhibitors to Factor VIII or IX Undergoing Elective Surgery or Other Invasive Procedure
Condition:   Hemophilia
Intervention:   Biological: Coagulation Factor VIIa (Recombinant)
Sponsors:   LFB USA, Inc.;   Laboratoire français de Fractionnement et de Biotechnologies
Recruiting - verified January 2017
Swiss Hemophilia Registry
Condition:   Hemophilia and Other Severe Bleeding Disorders
Intervention:   Other: Registry
Sponsor:   Swiss Hemophilia Network
Recruiting - verified March 2017
Investigating a Von Willebrand Factor (VWF) Functional Screening Assay for Assigning the Phenotypic Variants of Von Willebrand Disease (VWD)
Condition:   Von Willebrands Disease
Intervention:  
Sponsors:   Jonathan Roberts;   Blood Center of Wisconsin
Recruiting - verified August 2016
Natural History Study of Factor IX Treatment and Complications
Condition:   Factor IX Deficiency
Intervention:   Other: Standard care with blood and urine sample collection
Sponsors:   Skane University Hospital;   Indiana Hemophilia and Thrombosis Center;   University of Bonn;   Biogen;   Swedish Orphan Biovitrum
Recruiting - verified May 2017
Efficacy of a Timolol Nasal Spray as a Treatment for Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT) - (TEMPO)
Condition:   Hemorrhagic Hereditary Telangiectasia (HHT)
Interventions:   Drug: Timolol nasal spray;   Drug: Placebo nasal spray
Sponsor:   Hospices Civils de Lyon
Recruiting - verified November 2016
"Incidence and Consequences of Disorders of Glycosylation in Patients With Conotruncal and Septal Heart Defects"
Conditions:   Congenital Heart Diseases;   Conotruncal Defects;   Congenital Disorder of Glycosylation;   Antithrombin III Deficiency
Intervention:   Other: none intervention
Sponsors:   Hospital Universitari Vall d'Hebron Research Institute;   Universidad de Murcia
Recruiting - verified March 2017
A Gene Therapy Study for Hemophilia B
Condition:   Hemophilia B
Intervention:   Genetic: SPK-9001
Sponsors:   Spark Therapeutics;   Pfizer
Recruiting - verified February 2017
Study of Recombinant Factor VIIa Fusion Protein (rVIIa-FP, CSL689) for On-demand Treatment of Bleeding Episodes in Patients With Hemophilia A or B With Inhibitors
Conditions:   Hemophilia A With Inhibitors;   Hemophilia B With Inhibitors
Interventions:   Drug: CSL689;   Drug: Eptacog alfa (activated)
Sponsor:   CSL Behring
Recruiting - verified June 2017
Safety/Efficacy Study to Assess Whether FVIII/VWF Concentrate Can Induce Immune Tolerance in Haemophilia A Patients
Condition:   Hemophilia A
Intervention:   Drug: Plasma-derived FVIII/VWF concentrate
Sponsors:   Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico;   Sintesi Research Srl
Recruiting - verified June 2015
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 Fanhdi
Sponsor:   Instituto Grifols, S.A.
Recruiting - verified September 2015
Cardiopulmonary Exercise Testing to Evaluate Pulmonary AVMs With and Without Airflow Obstruction
Conditions:   Pulmonary Arteriovenous Malformations;   Hereditary Hemorrhagic Telangiectasia;   Asthma;   COPD
Intervention:   Other: Cardiopulmonary exercise test
Sponsor:   Imperial College London
Not yet recruiting - verified April 2015
Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
Condition:   Hemophilia
Intervention:  
Sponsor:   Karolinska Institutet
Recruiting - verified March 2017
A Phase III Study on the Safety, Pharmacokinetics and Efficacy of Coagulation Factor VIIa
Conditions:   Hemophilia A With Inhibitors;   Hemophilia B With Inhibitors
Intervention:   Biological: Coagulation FVIIa (Recombinant)
Sponsors:   LFB USA, Inc.;   Laboratoire français de Fractionnement et de Biotechnologies
Recruiting - verified January 2017
Myofascial Therapy in Patients With Hemophilic Arthropathy
Condition:   Hemophilia
Intervention:   Other: Myofascial therapy
Sponsor:   Universidad Católica San Antonio de Murcia
Recruiting - verified September 2015
A Study of a Long-Acting r-Factor 7a (Factor VIIa) in Adult Men With Hemophilia A or B
Conditions:   Hemophilia A;   Hemophilia B
Intervention:   Drug: MOD-5014
Sponsor:   Opko Biologics
Recruiting - verified September 2016
Study to Assess the Efficacy, Safety and Pharmacokinetic of Octafibrin in Paediatric Subjects With Fibrinogen Deficiency
Condition:   Congenital Fibrinogen Deficiency
Intervention:   Biological: Octafibrin
Sponsor:   Octapharma
Recruiting - verified May 2016
Intranasal Bevacizumab for HHT-Related Epistaxis
Conditions:   HHT;   Hereditary Hemorrhagic Telangiectasia;   Epistaxis;   Nose Bleeds;   Nasal Bleeding
Interventions:   Drug: Bevacizumab;   Drug: Placebo (Saline)
Sponsor:   Stanford University
Recruiting - verified April 2017
Patient Functioning and Well-being, Economic, and Clinical Impact of Hemophilia A and Its Treatment
Condition:   Hemophilia A
Intervention:   Drug: Available Recombinant, and Human Factor VIII products incl. Kogenate FS (Recombinant Factor VIII, BAY14-2222)
Sponsor:   Bayer
Recruiting - verified June 2017
European Alport Therapy Registry - European Initiative Towards Delaying Renal Failure in Alport Syndrome
Conditions:   Alport Syndrome;   Hereditary Kidney Disease;   Pediatric Kidney Disease;   Thin Basement Membrane Disease;   Familial Benign Hematuria
Interventions:   Drug: ACE-inhibitor;   Drug: AT1-inhibitor;   Drug: HMG-Coenzyme inhibitor (statin);   Drug: Spironolactone;   Drug: Paricalcitol
Sponsors:   University Hospital Goettingen;   Gesellschaft für Pädiatrische Nephrologie;   Deutsche Gesellschaft für Nephrologie;   Alport Selbsthilfe e.V.;   Association pour l'Information et la Recherche sur les Maladies Rénales Génétiques (AIRG);   KfH Foundation Preventive Medicine
Recruiting - verified October 2016
A Longitudinal Study of Hermansky-Pudlak Syndrome Pulmonary Fibrosis
Condition:   Hermansky Pudlak Syndrome
Interventions:   Other: Pulmonary function test;   Other: Chest CT;   Other: Sample collection
Sponsors:   Vanderbilt University;   University of South Florida;   National Institutes of Health (NIH);   National Heart, Lung, and Blood Institute (NHLBI)
Recruiting - verified January 2017
Prospective Biomarkers of Bone Metabolism in Hemophilia A
Conditions:   Hemophilia;   Bone Disease
Intervention:   Drug: Advate
Sponsors:   Oregon Health and Science University;   Baxter Healthcare Corporation
Recruiting - verified September 2016
Atenativ Effect on Uterine Blood Flow and Preeclampsia
Condition:   Antithrombin III Deficiency
Intervention:   Drug: Atenativ
Sponsors:   Vastra Gotaland Region;   Octapharma
Recruiting - verified March 2016
Pharmacokinetics, Efficacy, and Safety of Human Plasma-Derived Fibrinogen (FIB Grifols) in Patients With Congenital Afibrinogenaemia
Condition:   Congenital Afibrinogenaemia
Intervention:   Biological: Human Plasma-Derived Fibrinogen Concentrate
Sponsor:   Instituto Grifols, S.A.
Recruiting - verified July 2016
Gene Therapy for WAS Follow-up
Condition:   Wiskott-Aldrich Syndrome
Intervention:   Genetic: Autologous CD34+ cells transduced with WASP lentiviral vector
Sponsor:   Genethon
Recruiting - verified August 2016
The European Paediatric Network for Haemophilia Management ( PedNet Registry)
Conditions:   Factor VIII Deficiency;   Factor IX Deficiency
Intervention:   Drug: Coagulation proteins
Sponsors:   UMC Utrecht;   Lund University Hospital
Recruiting - verified November 2016
Randomized Controlled Feasibility Trial of the Fearon Algorithm to Improve Management of Unstable Warfarin
Condition:   Hypoprothrombinemia
Interventions:   Other: Warfarin adjustment using the Fearon algorithm;   Other: Warfarin adjustment using standard dosing
Sponsors:   McMaster University;   Epitome Pharmaceuticals
Recruiting - verified March 2016
Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia A
Condition:   Severe Haemophilia A
Intervention:   Biological: Human cl rhFVIII
Sponsor:   Octapharma
Recruiting - verified October 2016
An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia A
Condition:   Hemophilia A
Intervention:   Biological: rFVIIIFc
Sponsors:   Bioverativ Therapeutics Inc.;   Swedish Orphan Biovitrum
Recruiting - verified May 2017
To Investigate Safety and Efficacy of NovoEight® (rFVIII) During Long-term Treatment of Haemophilia A in Japan
Conditions:   Congenital Bleeding Disorder;   Haemophilia A
Intervention:   Drug: turoctocog alfa
Sponsor:   Novo Nordisk A/S
Recruiting - verified June 2017
Efficacy and Safety Study of Octafibrin for On-demand Treatment of Acute Bleeding and to Prevent Bleeding During and After Surgery
Condition:   Congenital Fibrinogen Deficiency
Intervention:   Drug: Octafibrin
Sponsor:   Octapharma
Recruiting - verified May 2016
Hemophilia Inhibitor Prevention (INHIBIT) Trial
Condition:   Severe Hemophilia A
Intervention:   Drug: Long-acting recombinant factor VIII Fc fusion protein
Sponsor:   University of Pittsburgh
Not yet recruiting - verified April 2017
Development of a Specific Measure for Assessing Adherence in Adult Patients With Hemophilia.
Condition:   Haemophilia
Intervention:  
Sponsor:   Universidad de Murcia
Recruiting - verified September 2015
Adherence to Treatment in Hemophilia
Condition:   Haemophilia
Intervention:  
Sponsors:   ANA TORRES-ORTUÑO;   Pfizer
Recruiting - verified September 2015
Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B
Condition:   Hemophilia B
Intervention:   Biological: rFIXFc
Sponsors:   Bioverativ Therapeutics Inc.;   Swedish Orphan Biovitrum
Recruiting - verified February 2017
An Open-label Safety and Efficacy Study of Recombinant FVIII in Patients With Severe Hemophilia A
Conditions:   Hemophilia A;   Severe Hemophilia A
Intervention:   Biological: rVIII‑SingleChain
Sponsor:   CSL Behring
Recruiting - verified May 2017
Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies
Conditions:   Sickle Cell Disease;   Transfusion Dependent Alpha- or Beta- Thalassemia;   Diamond Blackfan Anemia;   Paroxysmal Nocturnal Hemoglobinuria;   Glanzmann Thrombasthenia;   Severe Congenital Neutropenia;   Shwachman-Diamond Syndrome;   Non-Malignant Hematologic Disorders
Interventions:   Drug: Reduced Toxicity Ablative Regimen;   Drug: Reduced Intensity Preparative Regimen;   Drug: Myeloablative Preparative Regimen
Sponsor:   Masonic Cancer Center, University of Minnesota
Recruiting - verified February 2017
Assessment of Proprioceptive and Functional Characteristics in Patients With Hemophilia
Condition:   Hemophilia
Intervention:  
Sponsor:   Universidad Católica San Antonio de Murcia
Recruiting - verified September 2015
Use of Proteomics for the Diagnosis of a Platelet-related Bleeding Disorder
Condition:   Inherited Platelet Disorders
Intervention:   Other: Blood collection for platelet protein samples
Sponsors:   Hadassah Medical Organization;   Hebrew University of Jerusalem
Not yet recruiting - verified February 2016
A Survey on the Success of Inhibitor Elimination Using Individualized Concentrate Selection and Controlled ITI
Condition:   Haemophilia A
Intervention:  
Sponsors:   Haemophilia Centre Rhine Main;   University of Bonn;   Malmö University;   University Hospital, Paris;   Hospital Louis Pradel, Haematology Department, Lyon;   Department of Hematology, CHU Montpellier;   Octapharma Molecular Biochemistry, Berlin
Recruiting - verified May 2017
Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B
Conditions:   Congenital Bleeding Disorder;   Haemophilia B
Intervention:   Drug: nonacog beta pegol
Sponsor:   Novo Nordisk A/S
Recruiting - verified February 2017
ADVATE Hemophilia A Outcome Database (AHEAD)
Condition:   Hemophilia A
Intervention:   Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Sponsor:   Baxalta US Inc.
Recruiting - verified March 2017
Global Hemostatic Methods in Hemophilia and Von Willebrand's Disease
Conditions:   Hemophilia A;   Hemophilia B;   Von Willebrand's Disease
Intervention:  
Sponsors:   Karolinska University Hospital;   The Swedish Society of Medicine;   Karolinska Institutet
Recruiting - verified August 2016
Patients Treated for Wiskott-Aldrich Syndrome (WAS) Since 1990
Condition:   Wiskott-Aldrich Syndrome
Intervention:  
Sponsors:   National Institute of Allergy and Infectious Diseases (NIAID);   Primary Immune Deficiency Treatment Consortium;   Rare Diseases Clinical Research Network
Recruiting - verified September 2016
Pharmacokinetic, Efficacy and Safety of BT524 in Patients With Congenital Fibrinogen Deficiency
Conditions:   Congenital Afibrinogenemia;   Congenital Hypofibrinogenemia
Intervention:   Biological: BT524 (fibrinogen concentrate from human plasma)
Sponsors:   Biotest;   ICON plc;   Interlab;   Phoenix Clinical Research
Recruiting - verified June 2016
A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
Condition:   Hemophilia B
Intervention:   Biological: rIX-FP
Sponsor:   CSL Behring
Recruiting - verified May 2017
Web-based Application for the Population Pharmacokinetic Service - Phase 1
Conditions:   Hemophilia A;   Hemophilia B
Intervention:   Device: Population pharmacokinetic estimation
Sponsors:   McMaster University;   Canadian Hemophilia Society
Recruiting - verified March 2016
Integrated Cancer Repository for Cancer Research
Conditions:   Pancreatic Cancer;   Thyroid Cancer;   Lung Cancer;   Esophageal Cancer;   Thymus Cancer;   Colon Cancer;   Rectal Cancer;   GIST;   Anal Cancer;   Bile Duct Cancer;   Duodenal Cancer;   Gallbladder Cancer;   Gastric Cancer;   Liver Cancer;   Small Intestine Cancer;   Peritoneal Surface Malignancies;   Familial Adenomatous Polyposis;   Lynch Syndrome;   Bladder Cancer;   Kidney Cancer;   Penile Cancer;   Prostate Cancer;   Testicular Cancer;   Ureter Cancer;   Urethral Cancer;   Hypopharyngeal Cancer;   Laryngeal Cancer;   Lip Cancer;   Oral Cavity Cancer;   Nasopharyngeal Cancer;   Oropharyngeal Cancer;   Paranasal Sinus Cancer;   Nasal Cavity Cancer;   Salivary Gland Cancer;   Skin Cancer;   CNS Tumor;   CNS Cancer;   Mesothelioma;   Breastcancer;   Leukemia;   Melanoma;   Sarcoma;   Unknown Primary Tumor;   Multiple Myeloma;   Ovarian Cancer;   Endometrial Cancer;   Vaginal Cancer
Intervention:  
Sponsor:   University of Nebraska
Recruiting - verified May 2017
Study to Investigate Immunogenicity, Efficacy and Safety of Treatment With Human-cl rhFVIII
Condition:   Severe Hemophilia A
Intervention:   Biological: Human-cl rhFVIII
Sponsor:   Octapharma
Recruiting - verified October 2016
Willebrand International Non-interventional Global Surveillance
Condition:   Von Willebrand Disease
Intervention:  
Sponsor:   Laboratoire français de Fractionnement et de Biotechnologies
Recruiting - verified March 2016
Participation in a Research Registry for Immune Disorders
Conditions:   ADA-SCID;   Adenosine Deaminase Deficiency;   Wiskott- Aldrich Syndrome;   Primary Immunodeficiencies;   Severe Combined Immune Deficiency
Intervention:  
Sponsors:   National Human Genome Research Institute (NHGRI);   National Institute of Allergy and Infectious Diseases (NIAID);   National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Recruiting - verified March 6, 2017
Iron Deficiency and Hereditary Haemorrhagic Telangiectasia
Condition:   Hereditary Haemorrhagic Telangiectasia
Intervention:   Drug: Ferrous sulphate 200mg oral tablet
Sponsor:   Imperial College London
Recruiting - verified July 2013
BMT Abatacept for Non-Malignant Diseases
Conditions:   Hurler Syndrome;   Fanconi Anemia;   Glanzmann Thrombasthenia;   Wiskott-Aldrich Syndrome;   Chronic Granulomatous Disease;   Severe Congenital Neutropenia;   Leukocyte Adhesion Deficiency;   Shwachman-Diamond Syndrome;   Diamond-Blackfan Anemia;   Dyskeratosis-congenita;   Chediak-Higashi Syndrome;   Severe Aplastic Anemia;   Thalassemia Major;   Hemophagocytic Lymphohistiocytosis;   Sickle Cell Disease
Intervention:   Drug: Abatacept
Sponsor:   Emory University
Recruiting - verified August 2016
Use of a TGA and TEM in the Assessment of the Efficacy of Treatment With APCC or rFVIIa
Condition:   Haemophilia
Intervention:  
Sponsor:   Stowarzyszenie Pomocy Chorym na Zakrzepicę i Skazy Krwotoczne Thrombus
Recruiting - verified December 2015
Reperfusion of Pulmonary Arteriovenous Malformations After Embolotherapy
Condition:   Pulmonary Arteriovenous Malformations
Intervention:   Procedure: Angiography and embolotherapy
Sponsor:   St. Michael's Hospital, Toronto
Recruiting - verified May 2017
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases
Conditions:   Severe Combined Immunodeficiency (SCID);   Immunodeficiency With Predominant T-cell Defect, Unspecified;   Severe Chronic Neutropenia;   Chronic Granulomatous Disease (CGD);   Hyper IgE Syndromes;   Hyper IgM Deficiencies;   Wiskott-Aldrich Syndrome;   Mendelian Susceptibility to Mycobacterial Disease;   Common Variable Immune Deficiency (CVID)
Intervention:   Biological: CD3/CD19 negative allogeneic hematopoietic stem cells
Sponsor:   University of Pittsburgh
Recruiting - verified June 2017
Immune Disorder HSCT Protocol
Conditions:   Immune Deficiency Disorders;   Severe Combined Immunodeficiency;   Chronic Granulomatous Disease;   X-linked Agammaglobulinemia;   Wiskott-Aldrich Syndrome;   Hyper-IgM;   DiGeorge Syndrome;   Chediak-Higashi Syndrome;   Common Variable Immune Deficiency;   Immune Dysregulatory Disorders;   Hemophagocytic Lymphohistiocytosis;   IPEX;   Autoimmune Lymphoproliferative Syndrome;   X-linked Lymphoproliferative Syndrome
Intervention:   Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
Sponsor:   Washington University School of Medicine
Recruiting - verified November 2016
Multicentre, Non-controlled, Prospective, Post-Marketing Safety Study Following Long-Term Prophylactic OptivateTreatment in Subjects With Severe Haemophilia A
Condition:   Haemophilia A
Intervention:   Biological: Optivate 500IU
Sponsor:   Bio Products Laboratory
Recruiting - verified December 2016
Modifiers of Disease Severity in Cerebral Cavernous Malformations
Conditions:   Cavernous Angioma, Familial;   Cerebral Cavernous Malformations;   Cerebral Cavernous Hemangioma
Intervention:  
Sponsors:   University of New Mexico;   University of California, San Francisco;   Barrow Neurological Institute;   Angioma Alliance;   National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting - verified May 2016
Topical Anti-angiogenic Therapy for Telangiectasia in HHT: Proof of Concept
Condition:   Hereditary Hemorrhagic Telangiectasia
Interventions:   Drug: Topical timolol maleate;   Drug: placebo saline drops
Sponsors:   St. Michael's Hospital, Toronto;   University of California, San Francisco;   The Hospital for Sick Children;   University of Toronto;   Sunnybrook Health Sciences Centre;   Ryerson University;   National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting - verified May 2017
Cardiopulmonary Exercise Testing to Evaluate Pulmonary AVMs
Conditions:   Pulmonary Arteriovenous Malformations;   Hereditary Hemorrhagic Telangiectasia
Interventions:   Other: Cardiopulmonary exercise test;   Procedure: Blood test
Sponsor:   Imperial College London
Recruiting - verified October 2015
Outcome of Acquired Haemophilia With Steroid Combined With Cyclophosphamide Versus Steroid Combined With Rituximab (CREHA Study)
Condition:   Acquired Haemophilia
Interventions:   Drug: Steroid + cyclophosphamide;   Drug: Steroid + rituximab
Sponsor:   University Hospital, Rouen
Recruiting - verified November 2016
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
Conditions:   SCID;   Omenn's Syndrome;   Reticular Dysgenesis;   Wiskott-Aldrich Syndrome;   Bare Lymphocyte Syndrome;   Common Variable Immunodeficiency;   Chronic Granulomatous Disease;   CD40 Ligand Deficiency;   Hyper IgM Syndrome;   X-linked Lymphoproliferative Disease;   Hemophagocytic Lymphohistiocytosis;   Griscelli Syndrome;   Chediak-Higashi Syndrome;   Langerhan's Cell Histiocytosis
Interventions:   Drug: Alemtuzumab 0.3 mg;   Drug: Cyclophosphamide;   Drug: Busulfan;   Biological: Stem Cell Transplantation;   Drug: Fludarabine phosphate 40 mg;   Drug: Melphalan;   Drug: Alemtuzumab 0.2 mg;   Drug: Busulfan;   Drug: Fludarabine phosphate 30 mg;   Drug: MESNA
Sponsor:   Masonic Cancer Center, University of Minnesota
Recruiting - verified February 2017
Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A
Conditions:   Congenital Bleeding Disorder;   Haemophilia A
Intervention:   Drug: turoctocog alfa pegol
Sponsor:   Novo Nordisk A/S
Recruiting - verified September 2016
Clinical and Basic Investigations Into Erdheim Chester Disease
Conditions:   Myelofibrosis;   Gaucher Disease;   Pulmonary Fibrosis;   Hermansky-Pudlak Syndrome (HPS);   Cancer
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Recruiting - verified April 3, 2017
Hemophilia Inhibitor Previously Untreated Patient Study
Condition:   Hemophilia A
Intervention:   Drug: FVIII concentrate
Sponsors:   The University of Texas Health Science Center, Houston;   Rho, Inc.;   Baxter Healthcare Corporation
Recruiting - verified May 2016
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Conditions:   Hematologic Malignancies;   Inherited Disorders of Metabolism;   Inherited Abnormalities of Platelets;   Histiocytic Disorders;   Acute Myelogenous Leukemia (AML or ANLL);   Acute Lymphoblastic Leukemia (ALL);   Other Acute Leukemia;   Chronic Myelogenous Leukemia (CML);   Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases;   Other Leukemia;   Hodgkin Lymphoma;   Non-hodgkin Lymphoma;   Multiple Myeloma/ Plasma Cell Disorder (PCD);   Inherited Abnormalities of Erythrocyte Differentiation or Function;   Disorders of the Immune System;   Automimmune Diseases;   Severe Aplastic Anemia
Intervention:   Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
Sponsors:   Center for International Blood and Marrow Transplant Research;   National Marrow Donor Program
Recruiting - verified May 2017
Gene Therapy for Wiskott-Aldrich Syndrome (WAS)
Condition:   Wiskott-Aldrich Syndrome
Intervention:   Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
Sponsors:   Genethon;   Great Ormond Street Hospital for Children NHS Foundation Trust;   Institute of Child Health
Recruiting - verified February 2016
Gene Therapy for WAS
Condition:   Wiskott-Aldrich Syndrome
Intervention:   Genetic: Autologous CD34 positive cells transduced with a lentiviral vector containing human WAS gene
Sponsors:   Genethon;   Hôpital Necker-Enfants Malades
Recruiting - verified February 2016
Thrombocytopathy in Gaucher Disease Patients
Conditions:   Gaucher Disease;   Thrombocytopathy
Intervention:  
Sponsors:   Rabin Medical Center;   Genzyme, a Sanofi Company
Recruiting - verified October 2016
BAY81-8973 Pediatric Safety and Efficacy Trial
Condition:   Haemophilia A
Intervention:   Biological: Recombinant Factor VIII (BAY81-8973)
Sponsor:   Bayer
Recruiting - verified June 2017
Genotype and Phenotype Correlation in Hereditary Thrombotic Thrombocytopenic Purpura (Upshaw-Schulman Syndrome)
Conditions:   Thrombotic Thrombocytopenic Purpura;   Congenital Thrombotic Thrombocytopenic Purpura;   Familial Thrombotic Thrombocytopenic Purpura;   Thrombotic Thrombocytopenic Purpura, Congenital;   Upshaw-Schulman Syndrome
Intervention:   Other: Observation
Sponsors:   University Hospital Inselspital, Berne;   Swiss National Science Foundation;   Mach-Gaensslen Foundation Switzerland;   Baxalta Innovations GmbH, Wien, Austria
Recruiting - verified September 2016
Cerebral Hemorrhage Risk in Hereditary Hemorrhagic Telangiectasia
Condition:   Hereditary Hemorrhagic Telangiectasia
Intervention:  
Sponsors:   St. Michael's Hospital, Toronto;   National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting - verified May 2017
Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
Condition:   Hemophilia B
Interventions:   Genetic: Gene Transfer;   Drug: scAAV2/8-LP1-hFIXco
Sponsors:   St. Jude Children's Research Hospital;   National Heart, Lung, and Blood Institute (NHLBI);   Hemophilia of Georgia, Inc.;   Children's Hospital of Philadelphia
Recruiting - verified April 2017
Registry for Vascular Anomalies Associated With Coagulopathy
Conditions:   Multifocal Lymphangioendotheliomatosis With Thrombocytopenia;   Cutaneovisceral Angiomatosis With Thrombocytopenia;   Vascular Anomaly With Thrombocytopenia;   Hemangiomas
Intervention:   Other: no intervention
Sponsor:   Medical College of Wisconsin
Recruiting - verified March 2017
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
Condition:   Von Willebrand Disease
Intervention:   Biological: Alphanate SD/HT
Sponsor:   Grifols Biologicals Inc.
Recruiting - verified January 2017
Safety, Pharmacokinetics and Efficacy of an AT-III Concentrate.
Condition:   Antithrombin III Deficiency
Intervention:   Drug: Plasma-derived AT-III concentrate
Sponsor:   Grifols Biologicals Inc.
Recruiting - verified January 2017
Safety Study of Alphanate in Previously Treated Patients With Severe Hemophilia A
Condition:   Severe Hemophilia A
Intervention:   Drug: Alphanate SD/HT
Sponsor:   Grifols Biologicals Inc.
Recruiting - verified January 2017
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 - verified August 2016
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 - verified May 2016
Molecular Studies on Hereditary Haemorrhagic Telangiectasia Families
Condition:   Telangiectasia, Hereditary Hemorrhagic
Intervention:  
Sponsors:   Imperial College London;   British Heart Foundation
Recruiting - verified August 2008
Case Notes Review on Patients With Hereditary Haemorrhagic Telangiectasia
Condition:   Telangiectasia, Hereditary Hemorrhagic
Intervention:  
Sponsors:   Imperial College London;   The Margaret Hayton HHT Fund
Recruiting - verified August 2008
Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome
Conditions:   Albinism;   Intestinal Disease;   Kidney Disease;   Myocardial Disease;   Pulmonary Fibrosis
Intervention:  
Sponsor:   National Human Genome Research Institute (NHGRI)
Recruiting - verified March 9, 2017
http://www.clinicaltrials.gov/ct2/results/rss.xml?&term=Inherited+Bleeding+Disorder&recr=Open&show_rss=Y&count=10000