Clinical trials: Factor VIII Deficiency
Hemophilia, Hemophilia A
ATHN 8: Previously Untreated Patients (PUPs) Matter Study
This is a multi-center cohort study of approximately 250 previously untreated patients (PUPs) with congenital moderate to severe hemophilia A or B in a network of up to 50 US Hemophilia Treatment Centers (HTCs).
- Conditions
- Hemophilia, Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor
- Trial name
- NCT03818529
- Where
- 27 sites in United States
- Volunteers
- 237 people
- Sponsor
- American Thrombosis and Hemostasis Network
Hemophilia A, Severe
A Study Evaluating Physical Activity and Joint Health in Severe Haemophilia A Patients ≥12 Years Treated Once Weekly With Efanesoctocog Alfa
FREEDOM is a multicentre, open-label, single-arm, phase 3b study in Europe that aims to enrol approximately 90 previously treated severe haemophilia A patients aged ≥12 years, currently on prophylaxis.
- Trial name
- FREEDOM (NCT05817812)
- Testing
- Efanesoctocog alfa
- Where
- 30 sites in Austria, Croatia and 12 more
- Volunteers
- 93 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A, Severe
An Open-label Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Immunogenicity of SKP-0141 for the Treatment and Prophylaxis in Severe Hemophilia a Patients
This is a prospective, multicenter, open-label study to assess efficacy, safety, pharmacokinetics (PK), and immunogenicity of human plasma-derived Factor VIII (FVIII) in previously treated patients (PTPs) with severe hemophilia…
- Trial name
- NCT06738901
- Testing
- SKP-0141
- Volunteers
- 55 people
- Sponsor
- SK Plasma Co., Ltd.
Congenital Bleeding Disorder, Haemophilia A
A Single Dose Trial of NNC 0155-0000-0004 in Patients With Haemophilia A
This trial is conducted in Asia and Europe. The aim of the trial is to investigate the pharmacokinetics (the rate at which the trial drug is eliminated from the body) of a single dose of turoctocog alfa (NNC 0155-0000-0004 (N8))…
- Trial name
- NCT01365520
- Testing
- turoctocog alfa
- Where
- 3 sites in Malaysia, United Kingdom
- Volunteers
- 4 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
PHYSICAL ACTIVITY AND FVIII CLEARANCE: RELEVANCE FOR PERSONALIZED THERAPY IN SEVERE HAEMOPHILIA A (PHYSEMO)
In persons with severe haemophilia A (HA) infused factor VIII (FVIII) half-life and other pharmacokinetic parameters can vary according to determinants such as blood group, von Willebrand factor (VWF) level or age.
- Trial name
- PHYSEMO (NCT04303936)
- Testing
- SenseWear® armband device
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Catholic University of the Sacred Heart
Congenital Bleeding Disorder, Haemophilia A
A Single Dose Trial of Recombinant Factor VIII (N8) in Japanese Subjects With Haemophilia A: An Extension to Trial NN7008-3543
This trial is conducted in Japan. The aim of this clinical trial is to investigate the pharmacokinetics (the effect of the investigated drug on the body) and safety of turoctocog alfa (recombinant factor VIII (N8)) in Japanese…
- Trial name
- NCT01238367
- Testing
- turoctocog alfa
- Where
- 6 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Novo Nordisk A/S
Factor VIII Deficiency, Factor IX Deficiency
The European Paediatric Network for Haemophilia Management ( PedNet Registry)
Rationale: Haemophilia is a rare disease; to improve knowledge international collaboration is needed. Well-defined clinical data will be collected from complete cohorts in order to prevent selection bias.
- Trial name
- PedNet (NCT02979119)
- Where
- 39 sites in Austria, Belgium and 17 more
- Volunteers
- 4,000 people
- Sponsor
- PedNet Haemophilia Research Foundation
Congenital Bleeding Disorder, Haemophilia A
Epidemiological Study on Haemophilia Care and Orthopaedic Status in Developing Countries
This study is conducted in Africa and Asia. The aim of this study is to evaluate in the participating countries the orthopaedic status and the degree of arthropathy of severe haemophilia patients in general.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia A With Inhibitors, Haemophilia B
- Trial name
- HAEMOcare (NCT01503567)
- Testing
- No treatment given
- Where
- 4 sites in India, Morocco and 2 more
- Volunteers
- 282 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
BAX 802 in CHA With Inhibitors
The purpose of this study is to evaluate the efficacy and safety of BAX 802 in males with congenital hemophilia A (CHA) with inhibitors who are undergoing major or minor elective surgical, dental, or other invasive procedures.
- Trial name
- CHAWI (NCT02895945)
- Testing
- Antihemophilic Factor (Recombinant), Porcine Sequence (BAX 802)
- Where
- 24 sites in United States, Bulgaria and 10 more
- Volunteers
- 8 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
Study of Biostate® in Children With Hemophilia A
The objective of this study is to assess the efficacy and safety of a Von Willebrand Factor/Factor VIII (VWF/FVIII), Biostate, and to investigate the pharmacokinetics of Biostate in children with haemophilia A.
- Trial name
- NCT01229007
- Testing
- Biostate
- Where
- 8 sites in Belarus, Georgia and 4 more
- Volunteers
- 35 people
- Sponsor
- CSL Behring
Severe Hemophilia A Without Inhibitor
Clinical Outcomes of Low Dose PK-guided EHL FVIII Concentrates Versus Standard Prophylaxis in Severe Haemophilia A
Individualised pharmacokinetic (PK)-guided dosing of extended half-life (EHL) FVIII concentrates prophylaxis may reduce hemophilia A bleeding events than previous prophylactic regimen.
- Trial name
- NCT05281185
- Testing
- Extended half-life FVIII concentrates with PK-guided dosing, standard half-life FVIII concentrates with weight-based dosing
- Where
- Thailand
- Volunteers
- 15 people
- Sponsor
- Chulalongkorn University
Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor
An Observational Study to Evaluate Physical Activity, Bleeding Incidence and Health Related Quality of Life, in Participants With Haemophilia A Without Inhibitors Receiving Standard of Care Treatment
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia…
- Conditions
- Severe Hereditary Factor VIII Deficiency Disease Without Inhibitor, Moderate Hereditary Factor VIII Deficiency Disease Without Inhibitor, Haemophilia A
- Trial name
- POWER (NCT04165135)
- Testing
- Standard of Care for Haemophilia A
- Where
- 17 sites in Italy
- Volunteers
- 103 people
- Sponsor
- Hoffmann-La Roche
Haemophilia A
Efficacy and Safety of Turoctocog Alfa Pegol (N8-GP) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Chinese Patients With Haemophilia A (pathfinder10)
The study investigates how well the medicine called turoctocog alfa pegol (N8-GP) works in previously treated Chinese patients with severe haemophilia A. Participants will be treated with N8-GP.
- Trial name
- Pathfinder10 (NCT05082116)
- Testing
- turoctocog alfa pegol (N8-GP)
- Where
- 11 sites in China
- Volunteers
- 36 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Observational Study Describing the Usual Clinical Practice Use of NovoSeven® in the Home Treatment of Joint Bleeds in Patients With Haemophilia A or B and Inhibitors
This trial is conducted in Africa and Asia. The aim of this study is to evaluate the efficacy of home treatment of joint bleeds (haemarthrosis) with NovoSeven® (activated recombinant human factor VII) in patients with haemophilia…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01234545
- Testing
- activated recombinant human factor VII
- Where
- 5 sites in Algeria, Morocco and 3 more
- Volunteers
- 35 people
- Sponsor
- Novo Nordisk A/S
Haemophilia A, Haemophilia B
A Study of the Impact of Hemophilia and Its Treatment on Brain Development, Thinking and Behaviour in Children With Hemophilia
The Hemophilia Growth and Development Study (HGDS) nearly 25 years ago showed haemophilia and HIV impacted brain development, thinking, and behaviour in children and adolescents.
- Trial name
- eTHINK (NCT03660774)
- Testing
- No treatment given
- Where
- 22 sites in United States
- Volunteers
- 563 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A With Inhibitors
A Long-term Assessment of Physical Activity, Range of Motion, and Functional Status Following Elective Orthopedic Surgery in Hemophilia Patients With Inhibitors
This study is conducted in the United States of America (USA). The aim of this study is to assess the long-term (5+ years) postoperative functional outcomes of elective orthopaedic surgery (EOS) patients from previously reported…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
- Trial name
- NCT01830712
- Testing
- eptacog alfa (activated)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Novo Nordisk A/S
Congenital Bleeding Disorder, Haemophilia A
Intra-subject Variability Following Administrations of Activated Recombinant Human Factor VII in Haemophilia Patients in a Non-bleeding State
This trial is conducted in Asia and Europe. The aim of this trial is to evaluate the intra-subject variability of thromboelastographic parameters (TEG® and ROTEM®) following two administrations of activated recombinant human…
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- NCT01562457
- Testing
- activated recombinant human factor VII
- Where
- 3 sites in France, Germany and 1 more
- Volunteers
- 30 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A
Safety and Efficacy of Long-term Treatment With SCT800 in Previously Treated Hemophilia A Patients.
This study is a multi-center, open-label, extension trial to evaluate the safety, efficacy of SCT800 in long term prophylaxis and on-demand treatment in patients with severe hemophilia A who have been previously treated with…
- Trial name
- NCT03947567
- Testing
- Recombinant Human Coagulation FVIII
- Where
- China
- Volunteers
- 240 people
- Sponsor
- Sinocelltech Ltd.
Hematologic Disorder, Bleeding Disorder
ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
In parallel with the growth of ATHN's clinical studies, the number of new therapies for all blood disorders is increasing significantly.
- Conditions
- Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia
- Trial name
- NCT04398628
- Where
- 76 sites in United States
- Volunteers
- 3,000 people
- Sponsor
- American Thrombosis and Hemostasis Network
Hemophilia A
Efficacy and Safety Study of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Pediatric Patients in Canada With Hemophilia A - A Continuation of Baxter Study 060101
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of children with hemophilia A. The study is open to pediatric patients in Canada who completed Baxter Study 060101.
- Trial name
- NCT00189982
- Testing
- Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- Canada
- Volunteers
- 4 people
- Sponsor
- Baxalta now part of Shire