Clinical trials: Inherited Abnormalities of Platelets
Von Willebrand Disease
A Canadian Study to Assess the Safety of Humate-P® Ivr (Infusion Volume Reduced)
As part of CSL Behring Canada's continued commitment to ensuring the safety of the new low volume preparation of Humate-P®, CSL Behring Canada proposes to conduct a prospective, multi-center structured data collection of routine…
- Trial name
- NCT00701545
- Where
- Canada
- Volunteers
- 21 people
- Sponsor
- CSL Behring
Von Willebrand Disease (VWD), Heavy Menstrual Bleeding (HMB)
HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
The purpose of this study is to evaluate the safety, tolerability, efficacy, and pharmacodynamics (PD) of multiple doses of ALN-6400 in adult and adolescent patients with VWD and HMB
- Trial name
- HMBeacon (NCT07575308)
- Testing
- ALN-6400
- Where
- 21 sites in United States, Netherlands and 3 more
- Volunteers
- 24 people
- Sponsor
- Alnylam Pharmaceuticals
Hemophilia A
Platelet Function in Patients With Hemophilia A
Abnormalities in the gene encoding Factor VIII (FVIII) results in hemophilia A, an X-linked recessive bleeding disorder with a prevalence of 1 in 5000 males.
- Trial name
- NCT02093065
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Boston Children's Hospital
Thrombocytopenia, Neonatal Thrombocytopenia
Neonatal Thrombocytopenia in the NICU
This prospective cohort study aims to determine the frequency, severity, and clinical outcome of thrombocytopenia among neonates admitted to the neonatal intensive care unit.
- Trial name
- NCT07882342
- Volunteers
- 120 people
- Sponsor
- Assiut University
Hemophilia A
Phenotypic Heterogeneity in Hemophilia A: An Investigation of the Role of Platelet Function
The is study will examine whether variation in clinical bleeding frequency and severity among boys with severe Hemophilia A (Factor VIII deficiency) is associated with variations in laboratory measurements of platelet activity.
- Trial name
- NCT02225483
- Where
- 2 sites in Canada
- Volunteers
- 42 people
- Sponsor
- CancerCare Manitoba
Von Willebrand Disease
Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease
This is an observational study, hence there is no study hypothesis
- Trial name
- NCT01602419
- Testing
- Patients using Wilate as standard of care
- Where
- 24 sites in United States, Argentina and 8 more
- Volunteers
- 120 people
- Sponsor
- Octapharma
Von Willebrand Disease
Optivate in People With Von Willebrand Disease Undergoing Surgery
An open, multi-centre study in patients with von Willebrand Disease (VWD) undergoing surgery.
- Trial name
- NCT00404300
- Testing
- Optivate
- Where
- 4 sites in Israel, United Kingdom
- Volunteers
- 25 people
- Sponsor
- Bio Products Laboratory
Von Willebrand Diseases
Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy…
- Trial name
- NCT04052698
- Testing
- Wilate
- Where
- 14 sites in United States, Belarus and 6 more
- Volunteers
- 43 people
- Sponsor
- Octapharma
Congenital Diaphragmatic Hernias
Contribution of PRF in CDH in Children With Prothetic Patch Closure
Improved management of giant congenital diaphragmatic hernias (CDH) in neonates : decreased risk of morbidity and mortality due to prosthesis release.
- Trial name
- HECODIAP (NCT03861182)
- Testing
- Biological functionalization of the prosthetic materials by PRF
- Where
- France
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- University Hospital, Strasbourg, France
Primary Immunodeficiency Diseases (PID), Immune Thrombocytopenic Purpura (ITP)
Safety and Efficacy Study of a 10% Intravenous Immune Globulin Solution in Subjects With Primary Immunodeficiency Disorders
The purpose of this study is to assess the safety and efficacy of Immune Globulin Intravenous (Human), 10% (IGIV 10%) in subjects with primary immunodeficiency disorders.
- Conditions
- Primary Immunodeficiency Diseases (PID), Immune Thrombocytopenic Purpura (ITP), Kawasaki Syndrome
- Trial name
- NCT00157079
- Testing
- Immune Globulin Intravenous (Human), 10%
- Where
- 11 sites in United States
- Volunteers
- 61 people
- Sponsor
- Baxalta now part of Shire
Patent Ductus Arteriosus
Platelet Transfusion for Treatment of Patent Ductus Arteriosus in Thrombocytopenic Preterm Neonates
Patent ductus arteriosus (PDA) is a common problem in preterm babies. Recently there have been various studies for and against an association between thrombocytopenia and PDA.
- Trial name
- NCT03022253
- Testing
- Liberal platelet transfusion, Restrictive platelet transfusion, Paracetamol
- Where
- India
- Volunteers
- 44 people
- Sponsor
- Post Graduate Institute of Medical Education and Research, Chandigarh
Quality of Life (QOL), Patient-Reported Outcome Measures (PROMs)
Validation of the EQ-5D-Y-3L and EQ-5D-Y-5L for Paediatric Patients in China
The goal of this observational study is to validate the EQ-5D-Y-3L and EQ-5D-Y-5L in measuring health-related quality of life (HRQoL) for paediatric patients with different health conditions in China.
- Conditions
- Quality of Life (QOL), Patient-Reported Outcome Measures (PROMs), Pneumonia Childhood, Neuroblastoma (NB)
- Trial name
- NCT06873672
- Where
- China
- Volunteers
- 360 people
- Sponsor
- RenJi Hospital
Congenital Heart Disease, Tetralogy of Fallot
Prognostic Value of NLR, TLR, and ALC in Predicting ToF Primary Repair Outcome
Tetralogy of Fallot (ToF) were cyanotic congenital heart disease with chronic hypoxia which increases the risk of exacerbated inflammatory response in ToF primary repair.
- Conditions
- Congenital Heart Disease, Tetralogy of Fallot, Neutrophil-lymphocyte Ratio, Absolute Lymphocyte Count
- Trial name
- NCT05976204
- Testing
- ToF primary repair
- Where
- Indonesia
- Volunteers
- 501 people
- Sponsor
- National Cardiovascular Center Harapan Kita Hospital Indonesia
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
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
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
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
Von Willebrand Disease (VWD)
Study to Evaluate Subcutaneous (SC) VGA039 in Patients With Von Willebrand Disease (VWD)
This is a phase 3 study that will evaluate subcutaneous (SC) VGA039 in patients with von Willebrand Disease (VWD)
- Trial name
- VIVID-6 (NCT07115004)
- Testing
- VGA039
- Where
- 32 sites in United States, Australia and 8 more
- Volunteers
- 60 people
- Sponsor
- Vega Therapeutics, Inc
Von Willebrand Disease
A Study With OPTIVATE® in People With Von Willebrand Disease
The main objective of the study is to assess the pharmacokinetics of OPTIVATE® after a single dose of 80 IU/kg VWF:RCo. The secondary objectives of the study are to assess efficacy and safety of OPTIVATE® in long-term use over at…
- Trial name
- NCT00387192
- Testing
- Optivate
- Where
- 4 sites in Israel, United Kingdom
- Volunteers
- 26 people
- Sponsor
- Bio Products Laboratory