Clinical trials: Coagulation Abnormalities
Myelofibrosis, Gaucher Disease
Clinical and Pathophysiological Investigations Into Erdheim Chester Disease
Background: Erdheim Chester Disease (ECD) is a very rare disease in which abnormal white blood cells start growing and affect the bones, kidneys, skin, and brain.
- Conditions
- Myelofibrosis, Gaucher Disease, Pulmonary Fibrosis, Hermansky-Pudlak Syndrome (HPS)
- Trial name
- NCT01417520
- Where
- United States
- Volunteers
- 88 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Hemophilia A
China ADVATE PTP Study
The purpose of this study is to assess efficacy, safety and pharmacokinetics of ADVATE in the treatment and prevention of bleeding episodes (BEs)
- Trial name
- NCT02170402
- Testing
- Octocog alfa (recombinant human coagulation factor VIII)
- Where
- 11 sites in China
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Hemophilia, Hemophilia A
Viscoelastic Properties of Lower Extremity Muscles in Patients With Hemophilia
The aim of our study is to evaluate the viscoelastic properties of lower extremity muscles in patients with hemophilia A and hemophilia B secondarily to compare them with their healthy peers.
- Conditions
- Hemophilia, Hemophilia A, Hemophilia B
- Trial name
- NCT05981313
- Where
- Turkey (Türkiye)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Hasan Kalyoncu University
Immune Thrombocytopenia, Autoimmune Hemolytic Anemia
Severe Immune Cytopenia Registry Www.Sic-reg.Org
Prospective registry study for children and young adults with severe immune cytopenias (persisting/chronic immune thrombocytopenia, autoimmune hemolytic anemia, and Evans syndrome) to improve the management, facilitate the…
- Conditions
- Immune Thrombocytopenia, Autoimmune Hemolytic Anemia, Evans Syndrome, Primary Immunodeficiency
- Trial name
- sic-reg (NCT03576742)
- Testing
- potential biomarkers
- Where
- Austria
- Volunteers
- 80 people
- Sponsor
- Medical University of Graz
Hemophilia A, Anticoagulant Therapy
Home Monitoring in Hemophilia A
Rationale: A novel point-of-care device capable of measuring factor VIII (FVIII) activity and thrombin generation (TG) is currently under development.
- Conditions
- Hemophilia A, Anticoagulant Therapy, Monitoring, Point of Care Testing
- Trial name
- NCT06702579
- Where
- Netherlands
- Volunteers
- 19 people
- Sponsor
- Radboud University Medical Center
Hemophilia A
Evaluation of Patient and Physician Reported Reasons for Switching Factor VIII Replacement Therapies
This US study will assess hemophilia A patient characteristics, health history and reasons for switching or not switching from both patient/caregiver and physician perspectives.
- Trial name
- PARkER (NCT03405337)
- Testing
- FVIII products, Conventional FVIII replacement therapies
- Where
- United States
- Volunteers
- 160 people
- Sponsor
- Bayer
Hemophilia
Phase III Clinical Trial of STSP-0601 for Injection in Hemophilia Patients
This study will assess the efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitor
- Trial name
- NCT06922045
- Testing
- STSP-0601 for Injection
- Where
- 18 sites in China
- Volunteers
- 40 people
- Sponsor
- Jiangsu BioJeTay Biotechnology Co., Ltd.
Hemophilia
Are There Differences Between Carriers of Haemophilia A and B?
This study aims to develop a systematic genetic screening strategy for (potential) female carriers of haemophilia by identifying as many female carriers as possible within the families of haemophilia patients regularly followed…
- Trial name
- NCT05217992
- Testing
- Genetic assessment (hemophilia testing)
- Where
- Belgium
- Volunteers
- 900 people, healthy volunteers welcome
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Hemophilia A Without Inhibitor
The Hemophilia Inhibitor Prevention Trial
This is a multi-center randomized phase III clinical trial, the Inhibitor Prevention Trial, in which Eloctate will be compared with Emicizumab, using adaptive design, to prevent inhibitors in patients with severe hemophilia A.
- Trial name
- NCT04303559
- Testing
- Eloctate Injectable Product, Emicizumab Injection [Hemlibra]
- Where
- 3 sites in United States
- Volunteers
- 1 people
- Sponsor
- Margaret Ragni
Von Willebrand Disease, Type 3
A Study to Assess the Efficacy and Safety of Emicizumab in Participants With Type 3 Von Willebrand Disease
This is a Phase III, multicenter, open-label clinical study designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of emicizumab prophylaxis in participants aged 1 month and above, who have been…
- Trial name
- WILL-EMI (NCT06998524)
- Testing
- Emicizumab, von Willebrand Factor (VWF) Concentrates, Factor VIII (FVIII) Concentrates
- Where
- 29 sites in United States, Belgium and 12 more
- Volunteers
- 75 people
- Sponsor
- Hoffmann-La Roche
Haemophilia
National Survey of People With Haemophilia in Portugal
The purpose of this study is to implement a national survey to collect information regarding Portuguese people with haemophilia (PWH) on socio-demographic, clinical and psychosocial factors.
- Trial name
- NCT02870114
- Where
- Portugal
- Volunteers
- 146 people
- Sponsor
- University of Minho
Von Willebrand Disease
The Von Willebrand Disease (VWD) International Prophylaxis Study
The von Willebrand Disease Prophylaxis Network (VWD PN) is an international study group formed with the goal of investigating the role of prophylaxis in clinically severe VWD that is non-responsive to other treatment(s).
- Trial name
- VIP (NCT00557908)
- Testing
- VWF/FVIII products
- Where
- 3 sites in United States, Sweden
- Volunteers
- 105 people
- Sponsor
- Skane University Hospital
Hemophilia A
An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A
The primary objective of the study was to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants (PUPs) with severe hemophilia A.
- Trial name
- NCT02234323
- Testing
- rFVIIIFc
- Where
- 71 sites in United States, Australia and 12 more
- Volunteers
- 108 people
- Sponsor
- Bioverativ, a Sanofi company
Hemophilia A
A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health
This is a 48-month observational, prospective, multicentre study. The overall aim of the study is to evaluate the long-term effectiveness of Elocta treatment on joint health in patients treated prophylactically with Elocta in a…
- Trial name
- A-MORE (NCT04293523)
- Testing
- ELOCTA
- Where
- 53 sites in Czechia, Estonia and 12 more
- Volunteers
- 427 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire
Hemophilia B
RIXUBIS Drug Use-Result Survey (Japan)
The purpose of this survey is to understand the following items observed in the actual clinical use of RIXUBIS. 1. Unexpected adverse drug reactions 2. Occurrence of adverse drug reactions in the actual clinical use 3.
- Trial name
- NCT02937831
- Testing
- RIXUBIS
- Where
- 5 sites in Japan
- Volunteers
- 6 people
- Sponsor
- Baxalta now part of Shire
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Hemophilia B
Post-Marketing Surveillance (Usage Results Study) of RIXUBIS in Adult and Pediatric Patients With Haemophilia B in South Korea
Primary: To characterize the safety of RIXUBIS when used under normal clinical care in South Korea. Secondary: To describe hemostatic effectiveness in subjects receiving RIXUBIS under normal clinical care in South Korea.
- Trial name
- NCT02922231
- Testing
- RIXUBIS
- Where
- 5 sites in South Korea
- Volunteers
- 57 people
- Sponsor
- Baxalta now part of Shire
Hypofibrinogenemia, Congenital, Afibrinogenemia, Congenital
Human Fibrinogen Concentrate (FGTW) in Pediatric Patients With Congenital Fibrinogen Deficiency
The aim of the study is to evaluate clinical pharmacology, efficacy and safety of FGTW in pediatric patients with congenital fibrinogen deficiency.
- Trial name
- NCT02094430
- Testing
- biological: human fibrinogen concentrate
- Where
- 4 sites in France, Lebanon and 2 more
- Volunteers
- 16 people
- Sponsor
- Laboratoire français de Fractionnement et de Biotechnologies
Congenital Bleeding Disorder, Haemophilia A
A Multinational, Open-Label, Non-Controlled Trial on Safety, Efficacy and Pharmacokinetics of NNC 0129-0000-1003 in Previously Treated Paediatric Patients With Severe Haemophilia A
This trial is conducted globally. The aim of the trial is to investigate safety, efficacy and pharmacokinetics (the exposure of the trial drug in the body) of NNC 0129-0000-1003 (N8-GP) in children with severe haemophilia A who…
- Trial name
- pathfinder™5 (NCT01731600)
- Testing
- turoctocog alfa pegol
- Where
- 51 sites in United States, Brazil and 14 more
- Volunteers
- 68 people
- Sponsor
- Novo Nordisk A/S