Clinical trials: Lymphatic Malformation
Hypofibrinogenemia, Afibrinogenemia
Human Fibrinogen Concentrate in Pediatric Cardiac Surgery
The goal of the study is to determine whether the use of Human Fibrinogen Concentrate (RiaSTAP) will decrease blood loss and the need for component blood therapy in neonates and infants undergoing cardiopulmonary bypass.
- Conditions
- Hypofibrinogenemia, Afibrinogenemia, Bleeding Disorders
- Trial name
- RiaSTAP (NCT02822599)
- Testing
- RiaStAP, Saline
- Where
- 2 sites in United States
- Volunteers
- 30 people
- Sponsor
- Nicklaus Children's Hospital f/k/a Miami Children's Hospital
Hemophilia A, Hemophilia B
Recombinant Factor VIIa BI (rFVIIa BI) Treatment of Acute Bleeding Episodes Per an On-demand Regimen
The purpose of the study is to determine the efficacy and safety of rFVIIa BI as part of a six-month on-demand treatment regimen in hemophilia A or B subjects with inhibitors.
- Trial name
- NCT01757405
- Testing
- Recombinant Factor VIIa BI (rFVIIa BI)
- Where
- 16 sites in United States, Japan and 7 more
- Volunteers
- 40 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A
BAX 826 Dose-Escalation Safety Study
1. To assess tolerability and safety of BAX 826 after a single infusion in previously treated patients (PTPs) with severe hemophilia A 2. To determine the pharmacokinetic (PK) parameters of BAX 826 compared to ADVATE 3.
- Trial name
- NCT02716194
- Testing
- BAX 826, Octocog alfa
- Where
- 28 sites in Bulgaria, Germany and 7 more
- Volunteers
- 40 people
- Sponsor
- Baxalta now part of Shire
Bilirubin Encephalopathy
Bilirubin Neurotoxicity (BN) and Neurodevelopmental Impairment (NDI) in Extremely Preterm (EP) Infants: Avoidable by Reducing the Usual Intravenous Lipid (UL) Administration
The purpose of this study is to evaluate the effect of usual versus reduced lipid intake on unbound bilirubin levels, brainstem auditory evoked responses, and neurodevelopmental outcome at 2 years in extremely preterm infants.
- Trial name
- NCT04584983
- Testing
- usual prescribed intralipid (UL) regimen, restricted prescribed intralipid (RL) regimen
- Where
- United States
- Volunteers
- 134 people
- Sponsor
- The University of Texas Health Science Center, Houston
Iron Overload, Excessive Body Iron Burden
Massive Iron Deposit Assessment
Iron overload is a severe complication of multiple blood transfusions. As the body has no physiologic mechanism for clearing iron, repeated transfusions cause iron accumulation in organs and lead to iron toxicity.
- Trial name
- NCT01572922
- Testing
- R2*-UTE, R2*-GRE, Liver biopsy
- Where
- United States
- Volunteers
- 142 people
- Sponsor
- St. Jude Children's Research Hospital
Hemophilia A
Evaluation Of Acceptability And Satisfaction Of Hemophilia Patients Treated With FusENGO
This study aims to investigate prospectively throughout a period of 3- 6 months and not to exceed 12 months (according to local therapeutic plans) patients experiences of treatment with Factor VIII in the new device named…
- Trial name
- NCT01959919
- Testing
- Refacto FusENGO
- Where
- 19 sites in Italy
- Volunteers
- 86 people
- Sponsor
- Pfizer
Hyperbilirubinemia, Erythroblastosis, Fetal
Efficacy of High-dose Intravenous Immunoglobulin Therapy for Hyperbilirubinemia Due Rh Hemolytic Disease
The use of intravenous immunoglobulin G (IVIG) therapy has been reported in hyperbilirubinemia of Rh hemolytic disease but we don't have enough evidences for it.
- Trial name
- NCT00288600
- Testing
- Intravenous Immunoglobulin, Normal saline solution
- Where
- Brazil
- Volunteers
- 92 people
- Sponsor
- Oswaldo Cruz Foundation
Sickle Cell Disease
Intravenous Magnesium for Sickle Cell Vasoocclusive Crisis
The purpose of this study is to determine the safety and efficacy of intravenous magnesium in shortening the duration of a pain crisis and to determine the health-related quality of life and short term outcomes of children…
- Trial name
- MAGiC (NCT01197417)
- Testing
- Intravenous Magnesium Sulfate
- Where
- 8 sites in United States
- Volunteers
- 208 people
- Sponsor
- Medical College of Wisconsin
Niemann-Pick Disease, Type C
Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence.
- Trial name
- NCT03471143
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Washington University School of Medicine
Hemophilia B
Study Evaluating On-Demand Treatment With BeneFIX In Chinese Subjects
This study will evaluate the safety and efficacy of on-demand treatment with BeneFIX in Chinese hemophilia B subjects.
- Trial name
- NCT00866606
- Testing
- Benefix
- Where
- 6 sites in China
- Volunteers
- 35 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Sickle Cell Anemia, Severe Acute Malnutrition
Management of Severe Acute Malnutrition in SCD, in Northern Nigeria
Except for children with HIV, all recommendations for treatment of childhood malnutrition are for children < 5 years of age. The overall goal of this randomized controlled nutrition feasibility trial is to identify whether…
- Trial name
- NCT03634488
- Testing
- hydroxyurea (20mg/kg/day), Ready-to-use therapeutic food
- Where
- 3 sites in United States, Nigeria
- Volunteers
- 132 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University Medical Center
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Choline Magnesium Trisalicylate and Combination Chemotherapy in Treating Patients With Acute Myeloid Leukemia
This randomized phase II trial studies how well choline magnesium trisalicylate with idarubicin and cytarabine works in treating patients with acute myeloid leukemia.
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT02144675
- Testing
- choline magnesium trisalicylate, idarubicin, cytarabine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Rutgers, The State University of New Jersey
Priapism
A Study to Evaluate the Safety and Efficacy of Crizanlizumab in Sickle Cell Disease Related Priapism
The goal of the study was to evaluate the efficacy and safety of crizanlizumab in sickle cell disease (SCD) patients with priapism.
- Trial name
- SPARTAN (NCT03938454)
- Testing
- Crizanlizumab
- Where
- 13 sites in United States
- Volunteers
- 36 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Adenosine 2A Agonist Lexiscan in Children and Adults With Sickle Cell Disease
Sickle cell disease (SCD) is an inherited blood disorder that causes the red blood cells to change their shape from a round shape to a half-moon/crescent or sickled shape.
- Trial name
- NCT01085201
- Testing
- Lexiscan
- Where
- 7 sites in United States
- Volunteers
- 39 people
- Sponsor
- Dana-Farber Cancer Institute
Chronic Iron Overload Due to Transfusion-dependant Anemias
Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)
Assessed the new film-coated tablet formulation to the currently approved dispersible tablet formulation with regards to overall safety, Gastrointestinal (GI) tolerability, palatability, satisfaction and compliance
- Trial name
- NCT02125877
- Testing
- Deferasirox dispersible tablet, Defearisox film-coated tablet
- Where
- 51 sites in United States, Argentina and 14 more
- Volunteers
- 173 people
- Sponsor
- Novartis Pharmaceuticals
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently…
- Trial name
- NCT03938792
- Testing
- PF-06741086
- Where
- 62 sites in United States, Bulgaria and 18 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
Accelerated Phase Chronic Myelogenous Leukemia, Blastic Phase Chronic Myelogenous Leukemia
Etanercept in Treating Young Patients With Idiopathic Pneumonia Syndrome After Undergoing a Donor Stem Cell Transplant
This phase II trial is studying how well etanercept works in treating young patients with idiopathic pneumonia syndrome after undergoing a donor stem cell transplant.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Blastic Phase Chronic Myelogenous Leukemia, Childhood Acute Lymphoblastic Leukemia in Remission, Childhood Acute Myeloid Leukemia in Remission
- Trial name
- NCT00309907
- Testing
- etanercept, methylprednisolone
- Where
- 26 sites in United States
- Volunteers
- 39 people
- Sponsor
- Children's Oncology Group
Congenital Bleeding Disorder, Haemophilia A
Evaluating the Haemostatic Effect of NNC 0129-0000-1003 During Surgical Procedures in Subjects With Haemophilia A.
This trial is conducted globally. The aim of this trial is to evaluate the haemostatic effect of NNC 0129-0000-1003 during surgical procedures in subjects with haemophilia A.
- Trial name
- pathfinder™3 (NCT01489111)
- Testing
- turoctocog alfa pegol
- Where
- 70 sites in United States, Australia and 19 more
- Volunteers
- 36 people
- Sponsor
- Novo Nordisk A/S
Antithrombin III Deficiency, Covid19
Antithrombin III in Infectious Disease Caused by COVID-19
The purpose of this research study is to see if participants who have SARS-CoV-2 and low levels of AT3 in the blood will benefit by being given AT3.
- Trial name
- NCT04899232
- Testing
- Antithrombin III
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Enrique Ginzburg
Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Clofarabine and Cytarabine in Treating Patients With Acute Myeloid Leukemia With Minimal Residual Disease
RATIONALE: Drugs used in chemotherapy, such as clofarabine and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Conditions
- Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12)
- Trial name
- NCT00863434
- Testing
- clofarabine, cytarabine, filgrastim
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- University of Washington