Clinical trials: Lymphatic Abnormalities
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease (SCD)
The main purpose of the study is to evaluate the efficacy and safety of the study drug known as prasugrel for the reduction of Vaso-Occlusive Crisis events in pediatric participants with sickle cell disease.
- Trial name
- NCT01794000
- Testing
- Prasugrel
- Where
- 50 sites in United States, Belgium and 12 more
- Volunteers
- 341 people
- Sponsor
- Eli Lilly and Company
Sickle Cell Disease
A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease
This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle…
- Trial name
- Hibiscus 2 (NCT06612268)
- Testing
- Etavopivat
- Where
- 175 sites in United States, Australia and 19 more
- Volunteers
- 510 people
- Sponsor
- Novo Nordisk A/S
Hemophilia A, Hemophilia B
World Bleeding Disorders Registry
The WBDR is an international observational disease registry of patients with hemophilia. It will provide a platform for a network of hemophilia treatment centres (HTCs) around the world to collect uniform and standardized patient…
- Conditions
- Hemophilia A, Hemophilia B, Von Willebrand Diseases
- Trial name
- WBDR (NCT03327779)
- Where
- Canada
- Volunteers
- 20,000 people
- Sponsor
- World Federation of Hemophilia
Sphingomyelin Lipidosis
Compassionate Use Program for Olipudase Alfa Enzyme Replacement Therapy for Patients With Chronic Acid Sphingomyelinase Deficiency (ASMD)
The objective of this program is to provide access to enzyme replacement therapy (ERT) with olipudase alfa for certain patients with ASMD, a severe, life threatening disease, that could not participate in the olipudase clinical…
- Trial name
- NCT04877132
- Testing
- olipudase alfa (GZ402665)
- Sponsor
- Sanofi
Sickle Cell Anemia
Carbon Monoxide Monitor for the Measurement of End-Tidal Carbon Monoxide Levels in Children With or Without Hemolysis
People who have Sickle Cell Anemia (HbSS) produce red blood cells with shorter lifespans. These red blood cells breakdown faster, and this is called hemolysis.
- Trial name
- NCT01848691
- Testing
- Measurement of carbon monoxide level in exhaled breath
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Ashutosh Lal
Sickle Cell Disease, Vaso-occlusive Crisis
A Study of Varespladib Infusion in Subjects With Sickle Cell Disease.
The purpose of this study is to determine the effectiveness of A 001 infusion in preventing acute chest syndrome in sickle cell disease (SCD) subjects with vaso-occlusive crisis, fever, and elevated serum C-reactive protein (CRP).
- Trial name
- IMPACTS-2 (NCT01522196)
- Testing
- Varespladib
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Anthera Pharmaceuticals
Velocardiofacial Syndrome
Computerized Cognitive Skills Training for Adolescents With Velocardiofacial Syndrome
This study will test a computer-based treatment for youth with the genetic disorder velocardiofacial syndrome (VCFS) to help them improve skills in memory, attention, and executive functioning.
- Trial name
- NCT00917189
- Testing
- Challenging Our Minds Program
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- State University of New York - Upstate Medical University
Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders
Collecting and Storing Blood Samples From Patients With Cancer
RATIONALE: Collecting and storing samples of blood from patients with cancer to test in the laboratory may help the study of cancer in the future.
- Conditions
- Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma
- Trial name
- NCT00900120
- Sponsor
- University of Medicine and Dentistry of New Jersey
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
Biological Therapy in Treating Patients With Advanced Myelodysplastic Syndrome, Acute or Chronic Myeloid Leukemia, or Acute Lymphoblastic Leukemia Who Are Undergoing Stem Cell Transplantation
This phase I/II trial is studying the side effects of biological therapy and to see how well it works in treating patients with advanced myelodysplastic syndrome, chronic myeloid leukemia, acute myeloid leukemia, or acute…
- Conditions
- 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), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
- Trial name
- NCT00052520
- Testing
- therapeutic allogeneic lymphocytes, aldesleukin, peripheral blood stem cell transplantation
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Fred Hutchinson Cancer Center
Blood Coagulation Disorders, Hemophilia A
Effekt-2 - Efficacy and Safety of Long-term Treatment With KOGENATE® FS in Latin America
The aim of this international prospective post-marketing surveillance study is to obtain data on treatment procedures, long-term safety and efficacy and patient acceptance of KOGENATE Bayer/FS in treatment of patients with…
- Trial name
- NCT00969319
- Testing
- Recombinant Factor VIII (Kogenate FS, BAY14-2222)
- Where
- 2 sites in Mexico, Venezuela
- Volunteers
- 105 people
- Sponsor
- Bayer
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
Childhood Langerhans Cell Histiocytosis, Leukemia
Umbilical Cord Blood and Placental Blood Transplantation in Treating Patients With Hematologic Cancer or Aplastic Anemia
RATIONALE: Umbilical cord blood or placental blood transplantation may be able to replace immune cells that were destroyed by the chemotherapy or radiation therapy that was used to kill cancer cells.
- Conditions
- Childhood Langerhans Cell Histiocytosis, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00008164
- Testing
- umbilical cord blood transplantation
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Herbert Irving Comprehensive Cancer Center
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
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of…
- Trial name
- paradigm™ 2 (NCT01333111)
- Testing
- nonacog beta pegol
- Where
- 43 sites in United States, Canada and 13 more
- Volunteers
- 74 people
- Sponsor
- Novo Nordisk A/S
Hemophilia B
Study Evaluating rFIX; BeneFIX® in Hemophilia B
The primary objective of this clinical research study is to assess the safety and efficacy of rFIX for a minimum of 6 months in previously treated patients (PTPs) with hemophilia B (FIX:C ≤2%) during standard-of-care treatment…
- Trial name
- NCT00093171
- Testing
- rFIX
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia
Alemtuzumab, Fludarabine, and Busulfan Followed By Donor Stem Cell Transplant in Treating Young Patients With Hematologic Disorders
RATIONALE: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread.
- Conditions
- Congenital Amegakaryocytic Thrombocytopenia, Diamond-blackfan Anemia, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00301834
- Testing
- alemtuzumab, busulfan, cyclosporine
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- University of California, San Francisco
Sickle Cell Disease
The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of…
- Trial name
- LIBRA (NCT06975865)
- Testing
- Rilzabrutinib
- Where
- 63 sites in United States, Belgium and 12 more
- Volunteers
- 192 people
- Sponsor
- Sanofi
Severe Hemophilia A
rFVIII-Fc (Produced by AryoGen Pharmed Co.) Pharmacokinetic Study
The study is designed as a randomized, two-armed, double-blind, single-dose, crossover, two-sequence, active-controlled, multi-center, bioequivalence clinical trial with a primary endpoint of dose-normalized area under the curve…
- Trial name
- NCT06137092
- Testing
- Factor VIII, recombinant human with Fc fusion (rFVIII-Fc)
- Where
- 5 sites in Iran
- Volunteers
- 50 people
- Sponsor
- AryoGen Pharmed Co.
Lung Disease, Pneumothorax
Study of the Disease Process of Lymphangioleiomyomatosis
Pulmonary lymphangioleiomyomatosis (LAM) is a destructive lung disease typically affecting women of childbearing age. Currently, there is no effective therapy for the disease and the prognosis is poor.
- Conditions
- Lung Disease, Pneumothorax, Tuberous Sclerosis, Lymphangioleiomyomatosis
- Trial name
- NCT00001465
- Testing
- Toshibia Aquilion One CT
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Hemophilia A, Hemophilia B
Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study
This is a multi-center, international study designed to collect clinical, genetic and quality of life information on females with hemophilia, an inherited bleeding disorder.
- Trial name
- NCT00936312
- Where
- 11 sites in United States, Australia and 7 more
- Volunteers
- 200 people
- Sponsor
- Weill Medical College of Cornell University
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