Clinical trials: Lymphatic Abnormalities
Von Willebrand Diseases
Minimize Menorrhagia in Women With Von Willebrand Disease
This is an outpatient, 24-week Phase III prospective, randomized, crossover trial comparing recombinant von Willebrand factor (rVWF) and tranexamic acid (TA, Lysteda®) to minimize menorrhagia in women with von Willebrand disease…
- Trial name
- VWDMin (NCT02606045)
- Testing
- recombinant von Willebrand factor, tranexamic acid
- Where
- 19 sites in United States
- Volunteers
- 39 people
- Sponsor
- Margaret Ragni
Hemophilia A
An Expanded Access Program of Emicizumab in Participants With Hemophilia A With Inhibitors
This open-label, multicenter expanded access program (EAP) is designed to provide emicizumab to eligible participants with hemophilia A with factor VIII (FVIII) inhibitors before it is commercially available in the United States…
- Trial name
- NCT03154437
- Testing
- Emicizumab
- Where
- 14 sites in United States
- Sponsor
- Genentech, Inc.
Blood Coagulation, Disorders, Surgical Procedures, Minimally Invasive
Is Plasma Transfusion Beneficial Prior to Low-Risk Procedures in Hospitalized Patients With Blood Clotting Abnormalities?
Blood clotting abnormalities or problems that happen during surgery ? even minor surgery ? are serious because of the possibility of serious bleeding that cannot be stopped.
- Trial name
- NCT00953901
- Testing
- Fresh Frozen Plasma Transfusion
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Mayo Clinic
Pre-Eclampsia, Intrauterine Growth Restriction
PEPPI Study: Identification of Women at Risk for Placental Dysfunction
The main purpose of this study is to evaluate Fetal Medicine Foundation's pre-eclampsia risk calculator using maternal characteristics, first trimester serum placental growth factor (PlGF) and mean arterial pressure (MAP) in a…
- Conditions
- Pre-Eclampsia, Intrauterine Growth Restriction, Polycystic Ovary Syndrome, Iron-deficiency
- Trial name
- NCT06115122
- Testing
- Pre-eclampsia screening program
- Where
- Finland
- Volunteers
- 3,000 people, healthy volunteers welcome
- Sponsor
- Oulu University Hospital
Anemia (Iron-Loading), Beta-Thalassemia
Evaluation of Subcutaneous Desferrioxamine as Treatment for Transfusional Hemochromatosis
To determine whether deferoxamine prevented the complications of transfusional iron overload.
- Conditions
- Anemia (Iron-Loading), Beta-Thalassemia, Hematologic Diseases, Hemoglobinopathies
- Trial name
- NCT00000595
- Testing
- deferoxamine
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
DiGeorge Syndrome, Congenital Heart Defects
Infection in DiGeorge Following CHD Surgery
We propose a retrospective review of patients with DiGeorge syndrome having undergone cardiac surgery to evaluate the incidence of blood stream and/or surgical site infection.
- Trial name
- NCT00278005
- Where
- United States
- Volunteers
- 400 people
- Sponsor
- Children's Healthcare of Atlanta
Sickle Cell Disease
Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease
This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member…
- Trial name
- HaploSCD (NCT01461837)
- Testing
- CD34 selected T-cell depleted allogeneic SCT
- Where
- 6 sites in United States
- Volunteers
- 21 people
- Sponsor
- New York Medical College
Sickle Cell Disease
A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease
Majority of patients who are eligible for allogeneic HSCT for cure of severe sickle cell disease lack a matched family donor. This study aims for cure of sickle cell disease by performing unrelated donor (outside family)…
- Trial name
- NCT01279616
- Testing
- Fludarabine monophosphate, Rituximab, Busulfan
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Nationwide Children's Hospital
Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
ScreenPlus is a consented, multi-disorder pilot newborn screening program implemented in conjunction with the New York State Newborn Screening Program that provides families the option to have their newborn(s) screened for a…
- Conditions
- Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease
- Trial name
- ScreenPlus (NCT05368038)
- Testing
- Confirmatory Testing
- Where
- 9 sites in United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Albert Einstein College of Medicine
Congenital Bleeding Disorder, Haemophilia A
Study Investigating Novoeight®/NovoEight® (Turoctocog Alfa) in Mexican Haemophilia A Patients
The trial is conducted in North America. The aim of the trial is to assess the safety of turoctocog alfa under conditions of routine clinical care in patients with haemophilia A in Mexico
- Trial name
- NCT03179748
- Testing
- turoctocog alfa
- Where
- Mexico
- Volunteers
- 10 people
- Sponsor
- Novo Nordisk A/S
Hemophilia
Outcomes of Prophylaxis and On-demand Treatment for Severe Hemophiliacs and Collection of Baseline Data in Hemophiliacs in Taiwan
Primary prophylaxis in severe hemophiliacs is defined that prophylaxis therapy starts before 2 years of age and prior to any clinically evident joint bleeding or after first joint bleeding and prior to the onset of joint damage…
- Trial name
- NCT02908243
- Testing
- collection of baseline data
- Volunteers
- 350 people
- Sponsor
- Taiwan Society of Thrombosis and Hemostasis
Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome
Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls
Background: Neurocognitive disorders affect how the brain uses oxygen. They may affect mental development in children. These disorders can be studied with imaging scans that use radiation; however, these methods are not ideal for…
- Conditions
- Juvenile Neuronal Ceroid Lipofuscinosis, Smith-Lemli-Opitz Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type C1
- Trial name
- NCT05642221
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Brain Tumor, Neurofibromatoses
Quality Improvement Project - "My Logbook! - I Know my Way Around!"; ("Mein Logbuch - Ich Kenne Mich Aus!")
In this study, the main goal is to implement and evaluate a novel, evidence-based psycho-educative program for children in oncological care. Patients are provided with booklets tailored to each specific stage of their treatment.
- Conditions
- Brain Tumor, Neurofibromatoses, Sarcoma, Leukemia
- Trial name
- NCT04474678
- Testing
- "My Logbook! - I know my way around!"
- Where
- 22 sites in Austria, Germany and 2 more
- Volunteers
- 240 people
- Sponsor
- Medical University of Vienna
DiGeorge Syndrome, DiGeorge Anomaly
Thymus Transplantation With Immunosuppression
The research purpose is to determine if thymus transplantation with immunosuppression is a safe and effective treatment for complete DiGeorge anomaly.
- Conditions
- DiGeorge Syndrome, DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- 884 (NCT00579709)
- Testing
- Thymus Tissue for Transplantation
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Hemophilia A and B
World Bleeding Disorders Registry Pilot Study
The World Federation of Hemophilia (WFH) is conducting the pilot phase of an international, observational, World Bleeding Disorders Registry (WBDR).
- Trial name
- WBDR (NCT02776826)
- Volunteers
- 356 people
- Sponsor
- World Federation of Hemophilia
22q.11.2 Deletion/Duplication
Non-Invasive Chromosomal Evaluation of 22q11.2
This study is being conducted to develop and evaluate a cell-free fetal DNA test (Harmony) for non-invasive prenatal detection of 22q11.2 chromosomal deletion or duplication.
- Trial name
- 22Q (NCT02541058)
- Where
- Belgium
- Volunteers
- 420 people, healthy volunteers welcome
- Sponsor
- Cindy Cisneros
Erythroblastosis, Fetal, Erythroblastosis Fetalis, Rh Disease
EPO-4-Rhesus Study
Up to 80% of infants with hemolytic disease due to maternal alloimmunization, treated with IUT, require at least one top-up transfusion for late anemia during the first 3 months of life.
- Conditions
- Erythroblastosis, Fetal, Erythroblastosis Fetalis, Rh Disease, Erythroblastosis Fetalis Due to RH Antibodies, Erythroblastosis Fetalis Due to Isoimmunization
- Trial name
- EPO-4-Rhesus (NCT03104426)
- Testing
- Darbepoetin Alfa
- Where
- Netherlands
- Volunteers
- 42 people
- Sponsor
- Sanquin-LUMC J.J van Rood Center for Clinical Transfusion Research
Hemophilia A
Prophylaxis Versus On-demand Therapy Through Economic Report
The project is a controlled observational, multicenter, prospective data collection on secondary prophylaxis with Kogenate Bayer in adolescents and adults with severe haemophilia A (FVIII < 1%).
- Trial name
- POTTER (NCT01159587)
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- Italy
- Volunteers
- 58 people
- Sponsor
- Bayer
Neonatal Jaundice
Reliability of Transcutaneous Bilirubin Measurement According to the Skin Colour of Newborns
Neonatal jaundice is a physiological process characterized by a yellow coloration of the skin and mucous membranes linked to an increase in a pigment: bilirubin.
- Trial name
- BILICOLOR (NCT07315126)
- Testing
- Skin color assessment
- Where
- France
- Volunteers
- 510 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Leukemia
Biomarkers in Blood Samples From Patients With Acute Myeloid Leukemia
RATIONALE: Studying samples of blood from patients with cancer in the laboratory may help doctors identify and lean more about biomarkers related to cancer.
- Trial name
- NCT01385150
- Testing
- mutation analysis, protein analysis, laboratory biomarker analysis
- Volunteers
- 220 people
- Sponsor
- Eastern Cooperative Oncology Group
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