Clinical trials: Coagulation Protein Disorders
Haemostasis, Congenital Bleeding Disorder
Bridging Hemophilia B Experiences, Results and Opportunities Into Solutions (B-HERO-S)
This study is conducted in the United States of America (USA). Tha aim of this study is bridging Hemophilia B Experiences, Results and Opportunities into Solutions (B-HERO-S).
- Conditions
- Haemostasis, Congenital Bleeding Disorder, Haemophilia B
- Trial name
- NCT02568202
- Testing
- No treatment given
- Where
- United States
- Volunteers
- 449 people
- Sponsor
- Novo Nordisk A/S
Von Willebrand Diseases
A Pilot Crossover Trial of Prophylactic Wilate Compared to Placebo for Heavy Menstrual Bleeding in Patients With VWD
The EMPOWER trial is a pilot multi-center, placebo-controlled (normal saline), double-blind (patient and outcome assessor), crossover, 2-year randomized trial in female outpatients with von Willebrand disease (VWD) and heavy…
- Trial name
- EMPOWER (NCT06205095)
- Testing
- Lyophilized concentrate of human coagulation von Willebrand Factor and factor VIII
- Where
- 2 sites in Canada
- Volunteers
- 20 people
- Sponsor
- Unity Health Toronto
Hemophilia A
Phase 3 Study for Efficacy and Safety Outcomes Data in Japanese Patients With Severe Hemophilia A
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in Japanese patients with severe hemophilia A.
- Trial name
- GENEr8-JPN (NCT06224907)
- Testing
- Valoctocogene roxaparvovec
- Where
- 4 sites in Japan
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- BioMarin Pharmaceutical
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
Safety, Efficacy and PK of BIVV001 in Pediatric Patients With Hemophilia A
Primary Objective: To evaluate the safety of BIVV001 in previously treated pediatric participants with hemophilia A. Secondary Objectives: To evaluate the efficacy of BIVV001 as a prophylaxis treatment.
- Trial name
- XTEND-Kids (NCT04759131)
- Testing
- efanesoctocog alfa (BIVV001)
- Where
- 40 sites in United States, Australia and 13 more
- Volunteers
- 74 people
- Sponsor
- Bioverativ, a Sanofi company
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
Hemophilia A
Moroctocog Alfa (AF-CC) for Prophylaxis and Treatment of Bleeding Episodes in Previously Treated Hemophilia A Patients
Moroctocog-alfa (AF-CC) is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia A (congenital factor VIII deficiency or classic hemophilia).
- Trial name
- NCT04396639
- Testing
- Moroctocog-alfa (AF-CC)
- Where
- 5 sites in India
- Volunteers
- 50 people
- Sponsor
- Pfizer
Hemophilia
Resistance Training With Blood Flow Restriction in Hemophilia
The main objectives of this study are to evaluate safety and chronic effects of a low-load resistance exercise intervention (elbow flexions and extensions, and knee extensions) with blood flow restriction in people with…
- Trial name
- HemoBFR (NCT05568524)
- Testing
- BFRT, Control
- Where
- Spain
- Volunteers
- 20 people
- Sponsor
- University of Valencia
Telemedicine, Exercise
Feasibility of a Blended Therapy Approach
Exercise is an important part of therapy guidelines in the rehabilitation of rare diseases (RDs) as Haemophilia and Myositis. The aim of this study is not to evaluate a new therapy intervention, but to evaluate the delivery of…
- Conditions
- Telemedicine, Exercise, Feasibility, Myositis
- Trial name
- bletheRD (NCT03713151)
- Testing
- Dividat FIT
- Where
- Switzerland
- Volunteers
- 30 people
- Sponsor
- University of Zurich
Chronic Myeloproliferative Disorders, Leukemia
Allogeneic Hematopoietic Cell Transplantation for Patients With Busulfex-based Regimen
RATIONALE: Giving chemotherapy, such as fludarabine and busulfan, before a donor peripheral stem cell transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- LCCC0510 (NCT00448357)
- Testing
- rabbit anti-thymocyte globulin (ATG), therapeutic allogeneic lymphocytes, busulfan
- Where
- United States
- Volunteers
- 54 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Hemophilia
Impact of Moderate Intensity Physical Activities on PK-guided EHL FVIII Concentrates Prophylaxis Severe HA Patients
To compare clinical outcomes before and after using combination between moderate to vigorous-intensity physical activities and pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis in moderate to severe…
- Trial name
- NCT05728528
- Testing
- combination between moderate to vigorous-intensity physical activities and pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis, pharmacokinetic-guided extended half-life FVIII concentrates prophylaxis alone
- Where
- Thailand
- Volunteers
- 13 people
- Sponsor
- Chulalongkorn University
Chronic Myeloproliferative Disorders, Leukemia
506U78 in Treating Patients With Hematologic Cancer and Kidney or Liver Impairment
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: Phase I trial to study the effectiveness of 506U78 in treating patients who have hematologic cancer…
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00004239
- Testing
- Compound 506U78
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- Alliance for Clinical Trials in Oncology
Hemophilia A, Hemophilia B
Psychometric Validation of the Hemophilia Functional Ability Scoring Tool (Hemo-FAST)
The purpose of this study is to validate the newly developed Hemophilia Functional Ability Scoring Tool (Hemo-FAST), which is a fast and simple scoring in haemophilia able to assess patient-reported functional mobility.
- Trial name
- Hemo-FAST (NCT04731701)
- Testing
- Hemophilia Joint Health Score (HJHS)
- Where
- 16 sites in France
- Volunteers
- 180 people
- Sponsor
- Swedish Orphan Biovitrum
Hemophilia A
Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A
Most transient inhibitor formation, if any, will develop within the first 4 weeks. The study is to further monitor whether participants with severe Hemophilia A will develop inhibitors or antibodies at the later stage when…
- Trial name
- NCT00621673
- Testing
- Kogenate (BAY 14-2222)
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Bayer
Hemophilia A
Impact of Emicizumab on Activated Clotting Time Using the i-STAT Alinity Analyzer
Emicizumab (Hemlibra®) is a subcutaneous hemostatic treatment approved in 2019 for the prophylaxis in severe hemophilia A. Emicizumab is a bispecific monoclonal antibody mimicking factor VIIIa that provides a constant level of…
- Trial name
- EMISTAT (NCT05856903)
- Testing
- ACT
- Where
- France
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- University Hospital, Grenoble
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
Hemophilia
Tissue Factor Pathway Inhibitor (TFPI) and Haemorrhagic Manifestations in Haemophilia A and B Patients
Haemophilia is a rare and serious congenital defect of blood coagulation due to a genetic mutation on a sexual chromosome. It affects quasi-essentially the men and it is responsible for bleeding.
- Trial name
- NCT02540187
- Testing
- blood specimen
- Where
- 7 sites in France
- Volunteers
- 164 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Factor X Deficiency
A Database For The Collection Of Clinical, Laboratory, And Treatment Data From Patients With Factor X Deficiency To Define The Natural History Of The Disease And Improve Patient Management
Factor X deficiency is one of the most severe rare bleeding disorders and may lead to serious spontaneous hemorrhages, particularly in patients with severe congenital forms.
- Trial name
- PRO-RBDD-FX (NCT07875478)
- Where
- 2 sites in Italy
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Fondazione Luigi Villa
Haemophilia B
A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France
Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic…
- Trial name
- B-SURE (NCT03655340)
- Testing
- Alprolix
- Where
- 18 sites in France
- Volunteers
- 91 people
- Sponsor
- Swedish Orphan Biovitrum
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
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