Clinical trials: Coagulation Protein Disorders
Hemophilia
Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX
Primary Objective: To characterize the long-term safety and tolerability of fitusiran Secondary Objectives: To characterize the efficacy and long-term efficacy of fitusiran as assessed by the frequency of: Bleeding episodes…
- Trial name
- ATLAS-OLE (NCT03754790)
- Testing
- Fitusiran
- Where
- 79 sites in United States, Australia and 18 more
- Volunteers
- 281 people
- Sponsor
- Genzyme, a Sanofi Company
Hemophilia A With Inhibitor, Hemophilia A With Anti Factor VIII
Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec in Hemophilia A With Active or Prior Inhibitors
This Phase I/II clinical study will evaluate the safety and efficacy of valoctocogene roxaparvovec in patients with severe haemophilia A and inhibitors to FVIII.
- Trial name
- GENEr8-INH (NCT04684940)
- Testing
- Valoctocogene roxaparvovec
- Where
- 9 sites in United States, Brazil and 5 more
- Volunteers
- 10 people
- Sponsor
- BioMarin Pharmaceutical
Von Willebrand Diseases
Impact of Von Willebrand Factor and Its Multimers on Angiogenesis
The study aims to study the expression of numerous proteins involved in angiogenesis in 70 patients with von Willebrand disease in order to try to identify markers of interest.
- Trial name
- WILLANGIO (NCT04810702)
- Testing
- no intervention
- Where
- France
- Volunteers
- 109 people, healthy volunteers welcome
- Sponsor
- Nantes University Hospital
Hemophilia A
Study Comparing Blood Levels of ReFacto and Advante in Hemophilia A
The purpose of the study is to compare the pharmacokinetic parameters of ReFacto and Advate, using the chromogenetic substrate assay to measure plasma Factor VIII activity in plasma.
- Trial name
- NCT00168051
- Testing
- ReFacto, Advante
- Where
- 13 sites in United States, Belgium and 6 more
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia B
Phase 1/2 Dose Confirmation Study of FLT180a in Hemophilia B
Study of FLT180a gene therapy in adults with Hemophilia B. Up to 9 patients will be enrolled to receive a single dose of FLT180a and be followed for 52 weeks. Results will confirm the dose for a future Phase 3 study.
- Trial name
- NCT05164471
- Testing
- verbrinacogene setparvovec
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- Spur Therapeutics
Von Willebrand Disease
Study of a pd VWF/FVIII Concentrate, Biostate®, in Subjects With Von Willebrand Disease
The aim of this study is to assess the pharmacokinetics (PK), efficacy, and safety of Biostate® in subjects with Von Willebrand Disease (VWD).
- Trial name
- NCT00941616
- Testing
- Biostate®
- Where
- 5 sites in Bulgaria, Poland and 2 more
- Volunteers
- 22 people
- Sponsor
- CSL Behring
Von Willebrands Disease
Investigating a Von Willebrand Factor (VWF) Functional Screening Assay for Assigning the Phenotypic Variants of Von Willebrand Disease (VWD)
The purpose of this study is to improve the investigators ability to diagnose von Willebrand Disease (VWD), a common inherited bleeding disorder.
- Trial name
- VWF-phV (NCT02466789)
- Where
- United States
- Volunteers
- 134 people
- Sponsor
- Jonathan Roberts
Severe Hemophilia A
Efficacy and Safety Study of Human-cl rhFVIII in PTPs With Severe Hemophilia A
This study will determine the efficacy of human-cl rhFVIII in previously treated patients with severe hemophilia A during prophylactic treatment, treatment of bleeding episodes and in surgical prophylaxis.
- Trial name
- NCT01125813
- Testing
- recombinant Factor VIII
- Where
- 11 sites in Austria, Bulgaria and 2 more
- Volunteers
- 32 people
- Sponsor
- Octapharma
Hemophilia A
Pharmacokinetic Assessment of Standard Half-Life (SHL) FVIII, Extended Half-Life (EHL) FVIII, and Efanesoctocog Alfa (BIVV001) in Severe Hemophilia A
Primary objective To assess the half-life of BIVV001, Standard Half-Life (SHL) rFVIII and Extended Half-Life (EHL) rFVIII after a single intravenous (IV) injection Secondary objectives To characterize additional pharmacokinetic…
- Trial name
- NCT05042440
- Testing
- Efanesoctocog alfa, Octocog alfa, Rurioctocog alfa pegol
- Where
- Bulgaria
- Volunteers
- 13 people
- Sponsor
- Sanofi
Hemophilia
Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B
Primary Objective: To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B Secondary Objectives: To characterize the safety and…
- Trial name
- ATLAS-PEDS (NCT03974113)
- Testing
- Fitusiran
- Where
- 16 sites in United States, Canada and 4 more
- Volunteers
- 32 people
- Sponsor
- Genzyme, a Sanofi Company
Thrombosis
Reversibility of Apixaban Anticoagulation With the Four Factor Prothrombin Complex Concentrate Kcentra
Apixaban is an anticoagulant (also known as blood thinner) approved by the Food and Drug Administration (FDA) for reducing the risk of stroke and systemic embolism in patients with non-valvular atrial fibrillation.
- Trial name
- 4PAR (NCT02270918)
- Testing
- Kcentra, Apixaban
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Thomas Jefferson University
Haemophilia B
A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN
This study will collect information on side effects and how well Refixia/REBINYN works during long-term treatment (prophylaxis) in males with haemophilia B.
- Trial name
- NCT03745924
- Testing
- Nonacog beta pegol
- Where
- 30 sites in Austria, Belgium and 11 more
- Volunteers
- 60 people
- Sponsor
- Novo Nordisk A/S
Von Willebrand Disease (VWD), Acquired Von Willebrand Disease
Endothelial Colony-Forming Cells in Patients With VWD, AVWS and Healthy Subjects
The goal of this observational study is to learn how endothelial colony-forming cells (ECFCs) behave in people with von Willebrand disease (VWD), acquired von Willebrand syndrome (AVWS), and in healthy individuals.
- Trial name
- ECFCs/2022 (NCT07358013)
- Testing
- Blood sample collection for VWF measurements in plasma, Blood sample collection for ECFC isolation and characterization, Genetic testing of VWF
- Where
- Italy
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Haemophilia
Self-myofascial Release With Foam Roller in Patients With Hemophilic Knee Arthropathy
Introduction: Hemophilic ankle arthropathy is manifested by degenerative functional alterations (deficit of muscular strength, mobility and proprioception) (intra-articular alterations) and chronic pain.
- Trial name
- NCT05108480
- Testing
- Experimental group
- Where
- Spain
- Volunteers
- 56 people
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Hemophilia B
Mutation p.Ile112Thr : Discrepancy Between Factor IX Level and Bleeding Phenotype
It appears that the mutation p.Ile112Thr in the factor IX gene confers a discrepancy between mild factor IX level and severe bleeding phenotype. Databases and litterature analysis are poor on this matter.
- Trial name
- Hémophilie B (NCT03946384)
- Testing
- data collection
- Where
- France
- Volunteers
- 12 people
- Sponsor
- Centre Hospitalier Universitaire Dijon
Von Willebrand Diseases
The Lived Experience of People With Von Willebrand Disease
von Willebrand disease (vWD) is reported to be the most common bleeding disorder, with prevalence estimated at 1% of the general population.
- Trial name
- VWD360 (NCT06064643)
- Testing
- Online Survey, Qualitative Interview, Bleed Diary
- Where
- United Kingdom
- Volunteers
- 611 people
- Sponsor
- Haemnet
Hemophilia
Blood Flow Restriction in Improving Muscle Strength of Patients With Hemophilic Ankle Arthropathy
Background. The main physical sequela of patients with hemophilia is the development of a progressive, degenerative intra-articular lesion, known as hemophilic arthropathy).
- Trial name
- NCT06261593
- Testing
- Blood flow restriction
- Where
- Spain
- Volunteers
- 31 people
- Sponsor
- Investigación en Hemofilia y Fisioterapia
Hemophilia
A Study of Fitusiran in Severe Hemophilia A and B Patients Previously Receiving Factor or Bypassing Agent Prophylaxis
Primary Objective: To characterize the frequency of bleeding episodes (BE) while receiving fitusiran treatment, relative to the frequency of bleeding episodes while receiving factor concentrate or bypassing agent (BPA)…
- Trial name
- ATLAS-PPX (NCT03549871)
- Testing
- Fitusiran, BPA prophylaxis, Factor (FVIII or FIX) prophylaxis
- Where
- 35 sites in United States, Australia and 13 more
- Volunteers
- 80 people
- Sponsor
- Genzyme, a Sanofi Company
Anemia, Chronic Myeloproliferative Disorders
Busulfan and Fludarabine Before Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer
RATIONALE: Drugs used in chemotherapy, such as busulfan and fludarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Conditions
- Anemia, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00301912
- Testing
- filgrastim, busulfan, fludarabine phosphate
- Where
- United States
- Sponsor
- University of California, San Francisco
Hemophilia
A Feasibility Study to Collect Data in Patients With Haemophilia
The purpose of this study is to collect reference data in patients with haemophilia. The study will also collect and store blood samples for potential future exploratory research in the disease area.
- Trial name
- NCT00798499
- Testing
- Laboratory variables
- Where
- 2 sites in Sweden
- Volunteers
- 50 people
- Sponsor
- AstraZeneca
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