Clinical trials: Congenital Bleeding Disorder
Telangiectasia, Hereditary Hemorrhagic, Osler-Rendu Disease
ATERO : A Randomised Study With Tranexamic Acid in Epistaxis of Rendu Osler Syndrome
Scientific Objectives Primary objective : Demonstrate that oral intake of tranexamic acid significantly reduces the risk of epistaxis occurrence, estimated by the average monthly duration of episodes of epistaxis.
- Trial name
- NCT00355108
- Testing
- tranexamic acid
- Where
- France
- Volunteers
- 170 people
- Sponsor
- Hospices Civils de Lyon
Hemophilia
ATHN 2: Factor Switching Study
This is a longitudinal, observational study of patients with Hemophilia A or B who are planning to switch to a newly approved coagulation factor replacement product, or who have recently switched factor products.
- Trial name
- NCT02546622
- Testing
- Factor VIII Replacement Products for Hemophilia which were FDA approved after January 1, 2013, Factor IX Replacement Products for Hemophilia which were FDA approved after January 1, 2013
- Where
- 27 sites in United States
- Volunteers
- 310 people
- Sponsor
- American Thrombosis and Hemostasis Network
Craniosynostoses, Hypovolemia
Hemodynamic Monitoring During Craniosynostosis Surgery: Comparing Traditional and Newer Technology Monitors (CRASY-PRAM)
Hemodynamic evaluation during pediatric anesthesia is essential to care management. Intraoperative cardiovascular instability is frequent in major surgeries, and appropriate monitoring is necessary to ensure safe anesthetic…
- Conditions
- Craniosynostoses, Hypovolemia, Hemorrhage, Hemodynamic Instability
- Trial name
- CRASY-PRAM (NCT06263075)
- Testing
- Intraoperative monitoring
- Where
- Italy
- Volunteers
- 30 people
- Sponsor
- IRCCS Azienda Ospedaliero-Universitaria di Bologna
Wiskott-Aldrich Syndrome
Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome
A protocol named as "CIP-2015" for patients with Wiskott-Aldrich Syndrome may reduce the rate of GvHD. The details of the protocal followed with: 1. Conditioning regimen Busulfan 16 mg/kg in total, Fludarabine 160 mg/m2 in total.
- Trial name
- NCT03198195
- Testing
- cyclophosphamide
- Volunteers
- 5 people
- Sponsor
- Capital Research Institute of Pediatrics
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
Haemophilia A
Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial
This trial is conducted in Asia, Europe and the United States of America (USA). The aim of the trial is to evaluate safety of immune tolerance induction (ITI) treatment with turoctocog alfa (a recombinant factor VIII) in patients…
- Trial name
- NCT03588741
- Testing
- Turoctocog alfa
- Where
- 14 sites in Austria, Bulgaria and 5 more
- Volunteers
- 1 people
- Sponsor
- Novo Nordisk A/S
Antepartum Hemorrhage, Postpartum Hemorrhage
Strengthening Maternal Neonatal and Child Health Services in a Rural District of Pakistan
The Maternal Neonatal and Child health indicators in District Dadu of Pakistan portrays a dismal pictures and after the floods of 2010-2011 the health infrastructure of this district was badly affected.
- Conditions
- Antepartum Hemorrhage, Postpartum Hemorrhage, Anemia, Eclampsia
- Trial name
- SRC (NCT02797353)
- Testing
- Maternal Neonatal and Child Health Interventions
- Volunteers
- 15,000 people, healthy volunteers welcome
- Sponsor
- Aga Khan University
Haemophilia B
Observatory of Patients With Haemophilia B Treated by IdElvion®
OrPHEe is a non-interventional, prospective and national study which aim is to record real life data in haemophilia B French patients treated with Idelvion® to confirm the efficacy and safety of this product established in…
- Trial name
- OrPHEe (NCT05086575)
- Where
- 28 sites in France
- Volunteers
- 222 people
- Sponsor
- CSL Behring
Von Willebrand Disease (VWD)
A Survey to Describe the Experience and Unmet Needs of Persons Living With Von Willebrand Disease (VWD) and Their Caregivers
The main aim of this study is to describe the experience and unmet needs of persons living with VWD and their caregivers in Canada.
- Trial name
- NCT05695560
- Testing
- No Intervention
- Where
- Canada
- Volunteers
- 12 people
- Sponsor
- Takeda
Hemophilia B
The Efficacy and Safety of ZS801 in Chinese Hemophilia B Patients.
A non-randomized, open-label study to evaluate the safety, kinetics and efficacy of a single intravenous infusion of ZS801 in hemophilia B subjects with endogenous FIX ≤2%.
- Trial name
- NCT05630651
- Testing
- ZS801
- Where
- China
- Volunteers
- 6 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Hemophilia A
An Open-label Safety, Efficacy and Pharmacokinetic Study of a Recombinant FVIII Compared to Recombinant Human Antihemophilic FVIII in Patients With Severe Hemophilia A
This is an open-label, non-randomized, efficacy, safety and pharmacokinetic (PK) study comparing octocog alfa and rVIII-SingleChain.
- Trial name
- NCT01486927
- Testing
- rVIII-SingleChain, Octocog alfa
- Where
- 56 sites in United States, Australia and 18 more
- Volunteers
- 175 people
- Sponsor
- CSL Behring
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 B
Prospective Registry of European Hemophilia B Patients Receiving BeneFIX® for Usual Use
This is an open-label multi-center Registry in patients with hemophilia B receiving BeneFIX. All patients who begin treatment with BeneFIX in European Union countries, will be eligible for participation.
- Trial name
- NCT00167973
- Where
- 90 sites in Austria, Belgium and 8 more
- Volunteers
- 218 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Protein C Deficiency
Retrospective Study to Capture Dosing and Treatment Outcome Data in Participants With Severe Congenital Protein C Deficiency Who Were Treated With Protein C Concentrate Under an Emergency Use IND
This is a data collection study with the purpose of capturing dosing and treatment outcome data in participants with severe congenital protein C deficiency who were treated with protein C concentrate under an Emergency Use…
- Trial name
- NCT00161720
- Testing
- Protein C Concentrate (Human) Vapor Heated
- Where
- 10 sites in United States
- Volunteers
- 11 people
- Sponsor
- Baxalta now part of Shire
Hereditary Hemorrhagic Telangiectasia, Epistaxis
Efficacy of Thalidomide in the Treatment of Hereditary Hemorrhagic Telangiectasia
Hereditary hemorrhagic telangiectasia (HHT) (OMIM 187300 and 600376), also known as Rendu-Osler-Weber syndrome, is an autosomal dominant disease and has a prevalence between 1:5000 and 1:8000 in different populations.
- Trial name
- THALI-HHT (NCT01485224)
- Testing
- Thalidomide
- Where
- Italy
- Volunteers
- 31 people
- Sponsor
- Fondazione IRCCS Policlinico San Matteo di Pavia
Early-Onset Sepses, Neonatal, Periventricular Haemorrhage Neonatal
Zinc Supplementation on Very Low Birth Weight Infant
Premature birth is a major cause of neonatal death in addition to neonatal asphyxia and infections. Early in life, premature babies must get aggressive nutrition so that there is no extrauterine growth restriction (EUGR) in the…
- Conditions
- Early-Onset Sepses, Neonatal, Periventricular Haemorrhage Neonatal, Bronchopulmonary Dysplasia, Retinopathy of Prematurity
- Trial name
- NCT04050488
- Testing
- Zinc Sulfate
- Volunteers
- 364 people, healthy volunteers welcome
- Sponsor
- Fakultas Kedokteran Universitas Indonesia
Von Willebrand Diseases
Real-life Data of Constitutional Von Willebrand Disease in Western France
The objective of this observational study HOPSCOTcH-WILL II is to provide an accurate and detailed account of current Von Willebrand Disease (VWD) therapeutic management by collecting real-life data on hemorrhagic treated events…
- Trial name
- HOPSCOTCH-II (NCT04887324)
- Testing
- no intervention
- Where
- 5 sites in France
- Volunteers
- 922 people
- Sponsor
- Nantes University Hospital
Von Willebrand Disease
Change in Thrombin Generation Potential and Thromboelastography During the Menstrual Cycle
The primary objective of this study is to describe characteristics and trends for thrombin generation (TG) and thromboelastography (TEG) at 4 time points during the menstrual cycle.
- Trial name
- NCT00178542
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- The University of Texas Health Science Center, Houston
Hemophilia
Myofascial Therapy in Patients With Hemophilic Arthropathy
Designing a treatment protocol using myofascial therapy for the treatment of pain and restrictions on mobility in patients with hemophilic arthropathy of the knee and ankle.
- Trial name
- He-Fascial (NCT02433782)
- Testing
- Myofascial therapy
- Where
- Spain
- Volunteers
- 25 people
- Sponsor
- Universidad Católica San Antonio de Murcia
Chronic Myeloproliferative Disorders, Leukemia
12-O-Tetradecanoylphorbol-13-acetate in Treating Patients With Hematologic Cancer or Bone Marrow Disorder
RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. PURPOSE: This phase I trial is studying the side effects and best dose of…
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00004058
- Testing
- tetradecanoylphorbol acetate
- Where
- United States
- Sponsor
- University of Medicine and Dentistry of New Jersey