Clinical trials: Factor VIII Deficiency
Hemophilia A, Hemophilia A, Congenital
Searching Patterns In the Robustness of Immunological FVIII Tolerance
Children with hemophilia A lack clotting factor VIII (FVIII) due to a genetic mutation. It is well known that administration of FVIII concentrate leads to immunological tolerance for the FVIII protein in the majority of children.
- Trial name
- SPIRIT (NCT07539402)
- Where
- Netherlands
- Volunteers
- 500 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Congenital Bleeding Disorder, Haemophilia A
Post-marketing Safety Surveillance of NovoSeven® in Patients With Haemophilia and Inhibitors by Means of the UK Haemophilia Database
This study is conducted in Europe. The purpose of this retrospective study is to collect additional safety information of patients with haemophilia and inhibitors who are treated with rFVIIa.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT00853086
- Testing
- activated recombinant human factor VII
- Where
- United Kingdom
- Volunteers
- 139 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Congenital
Study Evaluating "Real World" Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog Alfa) for Routine Prophylaxis
The primary objective of this study is to investigate weekly prophylaxis dosing regimens used in standard clinical practice. In addition the study will capture reported bleed rate, pattern of change in KOVALTRY prophylaxis dose…
- Trial name
- TAURUS (NCT02830477)
- Testing
- Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973
- Where
- 25 sites in United States, Belgium and 11 more
- Volunteers
- 313 people
- Sponsor
- Bayer
Congenital Bleeding Disorder, Haemophilia A
Study for Turoctocog Alfa Treatment Regimen in Iraqi Haemophilia A Patients
The study has descriptive purposes, with aim of assessing how turoctocog alfa is used in the everyday practice and to provide a baseline for the management of haemophilia A and does not involve any change in the clinical…
- Trial name
- NCT06574984
- Testing
- Turoctocog alfa
- Where
- 7 sites in Iraq
- Volunteers
- 329 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
Study Evaluating Approach to Treatment of Haemophilia A and B in Spain
Analyze the clinical and therapeutic approach to treatment of haemophilia A and B in habitual clinical practice conditions in Spain.
- Trial name
- NCT00581438
- Volunteers
- 52 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Factor VIII Deficiency, Congenital, Factor 8 Deficiency, Congenital
Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe
This study aims to observe the safety and efficacy of the Xyntha Solofuse prefilled syringe in the setting of routine practice. The primary objective is to detect medically significant events (factor VIII inhibitor).
- Conditions
- Factor VIII Deficiency, Congenital, Factor 8 Deficiency, Congenital, Autosomal Hemophilia A, Classic Hemophilia
- Trial name
- NCT03034044
- Volunteers
- 106 people
- Sponsor
- Pfizer
Hemophilia A, Hemophilia
Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®
The purpose of this study is to describe the pharmacokinetic profile of patients with hemophilia A in prophylaxis in Spain using myPKFit®
- Conditions
- Hemophilia A, Hemophilia, Factor VIII Deficiency
- Trial name
- NCT03006965
- Testing
- octocog alfa, rurioctocog alfa pegol
- Where
- 14 sites in Spain
- Volunteers
- 50 people
- Sponsor
- Spanish Society of Thrombosis and Haemostasis
Haemophilia A, Haemophilia B
Post-marketing Surveillance (Special Use-results Surveillance) on Treatment With Alhemo
The purpose of the study is to investigate the safety and effectiveness of Alhemo in participants under real-world clinical practice in Japan. Total duration of this study is about 6 years.
- Trial name
- NCT06831734
- Testing
- Concizumab
- Where
- 23 sites in Japan
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Observational Study on Safety of Room Temperature Stable NovoSeven® in Patients With Haemophilia A or B
This study is conducted in Europe and Asia. The aim of this observational study is to monitor antibody formation towards the room temperature stable formulation of NovoSeven® (activated recombinant human factor VII).
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01220141
- Testing
- activated recombinant human factor VII
- Where
- 14 sites in Argentina, Austria and 12 more
- Volunteers
- 51 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Comparison of the Action of Drugs in the Body and Safety of N8 and Advate® in Haemophilia A Subjects
This trial is conducted in Europe and Asia. The aim of this clinical trial is to compare two recombinant factor VIII drugs, turoctocog alfa (recombinant factor VIII (N8)) with Advate®, in haemophilia A subjects, investigating the…
- Trial name
- NCT00837356
- Testing
- Advate®, turoctocog alfa
- Where
- 7 sites in Germany, Israel and 3 more
- Volunteers
- 23 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A
Register of Patients With haEmophilia A tReated With Afstyla®
Record real life data of patients with Hemophilia A and treated with Afstyla® to assess the effectiveness and the safety of the treatment used as prophylaxis, prevention of bleeding (e.g.
- Trial name
- OPERA (NCT04675541)
- Testing
- Afstyla®
- Where
- 17 sites in France
- Volunteers
- 62 people
- Sponsor
- CSL Behring
Hemophilia A
Safety,Efficacy and Pharmacokinetics Evaluation of SCT800 in Previously Treated Paediatric Patients With Severe Haemophilia A.
This study is a multicenter phase III uncontrolled open-label trial to evaluate the efficacy,safety and pharmacokinetics of SCT800 in regular prophylaxis and perioperative treatment in patients (<12 years old) with severe…
- Trial name
- NCT03947320
- Testing
- Recombinant Human Coagulation FVIII
- Volunteers
- 70 people
- Sponsor
- Sinocelltech Ltd.
Hemophilia A
Non Neutralizing Antibodies: Prevalence and Characterization
Antibodies (Abs) directed against factorVIII (FVIII) remain the main iatrogenic complication in haemophilia A (HA) patients. Anti-FVIII Abs inhibiting pro-coagulant properties of the molecule are named inhibitors whereas Abs…
- Trial name
- NCT01541527
- Testing
- blood test
- Where
- France
- Sponsor
- University Hospital, Montpellier
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Factor VII Deficiency, Glanzmann Thrombasthenia
Study of Coagulation Factor VIIa Marzeptacog Alfa (Activated) in Subjects With Inherited Bleeding Disorders
The purpose of the trial is to evaluate the PK, bioavailability, PD, efficacy and safety of MarzAA for on demand treatment and control of bleeding episodes in adult subjects with inherited bleeding disorders.
- Conditions
- Factor VII Deficiency, Glanzmann Thrombasthenia, Hemophilia A With Inhibitor
- Trial name
- NCT04548791
- Testing
- Coagulation Factor VIIa variant
- Where
- 20 sites in United States, India and 3 more
- Volunteers
- 19 people
- Sponsor
- Catalyst Biosciences
Hemophilia A
SAFE Study: Safety of aPCC Following Emicizumab Prophylaxis
The purpose of the aPCC-emicizumab safety study is to investigate the hemostatic efficacy as measured by thrombin generation, of a low personalized dose of aPCC (FEIBA) in children and adults with hemophilia A and inhibitors on…
- Trial name
- SAFE (NCT04563520)
- Testing
- Emicizumab, FEIBA, rFVIIa
- Where
- 2 sites in United States
- Volunteers
- 5 people
- Sponsor
- Emory University
Severe Hemophilia A
Rescue Immunotolerance Study in Induction of Immune Tolerance (ITI)-Experienced Patients (RES.I.S.T. Experienced)
The purpose of this study is to evaluate whether a concentrate containing both FVIII and von Willebrand Factor (VWF) given at a high dose will induce immune tolerance in subjects who have already experienced and failed ITI with…
- Trial name
- RESIST EXP (NCT01051076)
- Testing
- VWF/FVIII concentrates
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- City of Hope Medical Center
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa (N8) in Prevention and On-demand Treatment of Bleeding Episodes in Subjects With Haemophilia A: An Extension to Trials NN7008-3543, NN7008-3545, NN7008-3600, NN7008-3893 and NN7008-4015
This trial is conducted in Asia, Europe, Japan, Oceania, North America and South America. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa (N8) in Haemophilia A patients.
- Trial name
- NCT00984126
- Testing
- turoctocog alfa
- Where
- 57 sites in United States, Brazil and 18 more
- Volunteers
- 214 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A, Haemophilia A With Inhibitors
A Study Following People With Haemophilia A and B, With or Without Inhibitors, When on Usual Treatment (Explorer™6)
This study will collect data on bleeds and data related to quality of life in people with severe congenital (a disease existing from birth) haemophilia A and B, with or without inhibitors.
- Conditions
- Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B, Haemophilia B With Inhibitors
- Trial name
- explorer™6 (NCT03741881)
- Testing
- No treatment given
- Where
- 143 sites in United States, Algeria and 34 more
- Volunteers
- 231 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A
A Study to Investigate the Safety and Efficacy of Optivate® in Children Under 6 Years of Age With Haemophilia A.
The main objectives of this study are: to assess Optivate® consumption (IU/kg consumed per month for prophylactic and on-demand therapy and dose at each bleed). to assess clinical outcome when treating a bleed with Optivate®.
- Trial name
- NCT02246894
- Testing
- Optivate® (Human Coagulation Factor VIII)
- Where
- 5 sites in Poland
- Sponsor
- Bio Products Laboratory