Clinical trials: Coagulation Abnormalities
Hemophilia A
Hematopoietic Stem Cell Transplantation Gene Therapy for Treatment of Severe Hemophilia A
This is a first-in-human, non-randomized, open label, single treatment, Phase 1 study in approximately 7 patients with severe hemophilia A.
- Trial name
- NCT04418414
- Testing
- Gene therapy, Biological
- Volunteers
- 7 people
- Sponsor
- Expression Therapeutics, LLC
Chronic Myeloproliferative Disorders, Leukemia
Fludarabine, Cyclophosphamide, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Bone Marrow Transplant for Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy, such as fludarabine and cyclophosphamide, and radiation therapy before a donor bone marrow transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00134004
- Testing
- cyclophosphamide, fludarabine phosphate, mycophenolate mofetil
- Where
- 3 sites in United States
- Volunteers
- 210 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Von Willebrand Disease (VWD), Type 2
Efficacy and Safety of BT200 (Rondaptivon Pegol) in Patients With Type 2B Von Willebrand Disease
This randomized clinical trial with a cross-over design is being conducted at the Department of Clinical Pharmacology at the Medical University of Vienna, and a total of 4-6 patients with type 2B von Willebrand disease (VWD) will…
- Trial name
- BT200-VWD2B (NCT07273721)
- Testing
- BT200
- Where
- Austria
- Volunteers
- 6 people
- Sponsor
- Medical University of Vienna
Hemophilia A
A Clinical Study to Evaluate the Effects of NXT007 Compared to Emicizumab Prophylaxis in People With Hemophilia A
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of NXT007 prophylaxis compared with emicizumab prophylaxis in people age 12 years and older with severe or moderate congenital…
- Trial name
- ZEBRHA 2 (NCT07416604)
- Testing
- NXT007, Emicizumab
- Where
- 63 sites in United States, Argentina and 20 more
- Volunteers
- 360 people
- Sponsor
- Hoffmann-La Roche
Von Willebrand Disease
Efficacy of Alphanate FVIII/VWF Concentrate in Type 3 Von Willebrand Patients
To assess the efficacy of FVIII/VWF Complex (Human), Alphanate® as replacement therapy in preventing excessive bleeding in subjects with congenital Type 3 von Willebrand Disease (VWD) who undergo surgical procedures.
- Trial name
- NCT00555555
- Testing
- Alphanate SD/HT
- Volunteers
- 4 people
- Sponsor
- Grifols Biologicals, LLC
Severe Hemophilia A
Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale
The primary objective of the study is to compare the pharmacokinetic (PK) of recombinant coagulation factor VIII Fc fusion protein (rFVIIIFc) manufactured at the current scale of 2000 L (2K) to the PK of rFVIIIFc manufactured at…
- Trial name
- Elevate (NCT02502149)
- Testing
- rFVIIIFc
- Where
- 15 sites in United States, Australia and 1 more
- Volunteers
- 24 people
- Sponsor
- Bioverativ Therapeutics Inc.
Hemophilia
Safety and Efficacy in a ReAl-Life Study in Patients With Haemophilia Treated wIth NovoEight® for Surgery
the aim of the SERAPHINE study is to gather data stemming from the French research database BERHLINGO (= Base d'Etude et de Recherche en Hémostase pour Les Investigateurs du Grand-Ouest, i.e.
- Trial name
- SERAPHINE (NCT04808349)
- Testing
- no intervention
- Where
- 3 sites in France
- Volunteers
- 60 people
- Sponsor
- Nantes University Hospital
Hemophilia B
A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B
This study will examine the safety and efficacy of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) for the control and prevention of bleeding episodes in subjects who have previously received factor…
- Trial name
- NCT01361126
- Testing
- Recombinant Coagulation Factor IX Albumin Fusion Protein
- Where
- 2 sites in Bulgaria, Israel
- Volunteers
- 17 people
- Sponsor
- CSL Behring
Hemophilia A
A Trial of the Safety and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A
The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-010 in subjects with hemophilia A.
- Trial name
- EDELWEISS (NCT06185335)
- Testing
- ANB-010, dose 1, ANB-010, dose 2, ANB-010, dose 3
- Where
- 17 sites in Russia
- Volunteers
- 50 people
- Sponsor
- Biocad
Noonan Syndrome
Link Between Abnormal Bleeding and Coagulation Disorders in Noonan Syndromes
Noonan syndrome is a relatively rare genetic disorder, affecting around 1 in every 1,000 to 2,500 children born. Patients often have a tendency to bleed more easily, particularly from the skin or mucocutaneous tissue (such as…
- Trial name
- PlatNoon (NCT07259135)
- Testing
- Reuse of routine clinical and biological data
- Where
- France
- Volunteers
- 100 people
- Sponsor
- University Hospital, Bordeaux
Hemophilia A
A Trial to Compare Prophylaxis Therapy to On-demand Therapy With a New Full Length Recombinant FVIII in Patients With Severe Hemophilia A
The objective of the trial is to demonstrate that 2-3 times per week prophylaxis therapy with BAY81-8973 is superior to on-demand therapy with BAY81-8973 in patients with severe Hemophilia A.
- Trial name
- Leopold II (NCT01233258)
- Testing
- rFVIII (BAY81-8973) on demand, rFVIII (BAY81-8973) prophylaxis low-dose, rFVIII (BAY81-8973) prophylaxis high-dose
- Where
- 47 sites in United States, Argentina and 16 more
- Volunteers
- 80 people
- Sponsor
- Bayer
Hemophilia A and B
Identification of New Biomarkers in Haemophilic Arthropathy Through Transcriptome Analysis
Chronic arthropathy is one of the most common complications of haemophilia, affecting up to 90 per cent of patients aged 4 and over in at least one joint.
- Trial name
- THEMA (NCT07867821)
- Testing
- Surgery
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Wiskott-Aldrich Syndrome
Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome
The Wiskott-Aldrich Syndrome (WAS) is an inherited disorder that results in defects of the blood and bone marrow. It affects boys because the genetic mistake is carried on the X chromosome.
- Trial name
- NCT01410825
- Testing
- Retrovirus-mediated gene transfer
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- David Williams
Glanzmann Thrombasthenia
Anti-αIIbβ3 Immunization in Glanzmann Thrombasthenia: Prevalence and Associated Risk Factors: Thrombasthenia Anti-αIIbβ3 Antibodies Study (TAAS)
This project aim to correlate risk factors (genetic, therapeutic and socio-demographic factors) to anti-αIIbβ3 antibodies formation following blood products transfusion (platelets or packed red cells) or pregnancy in a national…
- Trial name
- TAAS (NCT04595617)
- Testing
- Antibodies screening
- Where
- 7 sites in France
- Volunteers
- 28 people
- Sponsor
- University Hospital, Bordeaux
VWD - Von Willebrand's Disease
Update in VWD Laboratory Diagnosis
The present study will focus on the investigation of the global contribution and limitations of the multimeric analysis and mutation analysis in the VWD diagnostic process. 1.
- Trial name
- NCT03896997
- Volunteers
- 100 people
- Sponsor
- Assiut University
Hemophilia B
Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B
The purpose of the study is to evaluate the safety, tolerability and effect on FIX antigen and activity levels of ascending doses of SB-FIX.
- Trial name
- NCT02695160
- Testing
- SB-FIX
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Sangamo Therapeutics
Hemophilia B
Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
The primary objective of the study was to evaluate the safety of recombinant coagulation factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with severe hemophilia B.
- Trial name
- PUPs B-LONG (NCT02234310)
- Testing
- rFIXFc
- Where
- 27 sites in United States, Australia and 9 more
- Volunteers
- 33 people
- Sponsor
- Bioverativ, a Sanofi company
Haemostasis, Haemophilia A
A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors
This trial is conducted in Asia, Europe and the United States of America (USA). The aim of the trial is to assess the efficacy of concizumab administered s.c.
- Trial name
- explorer™5 (NCT03196297)
- Testing
- Concizumab, Turoctocog alfa
- Where
- 31 sites in United States, France and 9 more
- Volunteers
- 36 people
- Sponsor
- Novo Nordisk A/S
Acquired Fibrinogen Deficiency
Phase 3 Study of Fibrinogen Concentrate (CSL511) in Subjects With Pseudomyxoma Peritonei Undergoing Cytoreductive Surgery
This study is a phase 3, prospective, single center, randomized, open label, controlled, parallel arm, interventional study to investigate the efficacy and safety of CSL511, in participants undergoing cytoreductive surgery (CRS)…
- Trial name
- NCT06617897
- Testing
- CSL511 Fibrinogen concentrate (human), Cryoprecipitate
- Where
- United Kingdom
- Volunteers
- 90 people
- Sponsor
- CSL Behring
Von Willebrand Disease
A Study of Recombinant Von Willebrand Factor (rVWF) With or Without ADVATE in Children With Severe Von Willebrand Disease (VWD)
The main aim of the study is to check effectiveness, side effects, and tolerability of vonicog alfa (recombinant von Willebrand factor [rVWF]), with or without ADVATE, in the treatment and control of nonsurgical bleeding events…
- Trial name
- NCT02932618
- Testing
- Vonicog alfa, Antihemophilic Factor (Recombinant)
- Where
- 46 sites in United States, Austria and 11 more
- Volunteers
- 47 people
- Sponsor
- Baxalta now part of Shire
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