Clinical trials: Lymphatic Abnormalities
Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor
Combination Chemotherapy Followed by Bone Marrow Transplantation in Treating Patients With Rare Cancer
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Bone marrow transplantation may allow doctors to give higher doses of chemotherapy and kill more tumor cells.
- Conditions
- Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Head and Neck Cancer, Kidney Cancer
- Trial name
- NCT00002515
- Testing
- filgrastim, carboplatin, thiotepa
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Sickle Cell Disease, Hemoglobinopathies
Non-Myeloablative Conditioning and Bone Marrow Transplantation
Allogeneic blood or marrow transplantation (alloBMT) is a curative therapy for a variety of hematologic disorders, including sickle cell disease and thalassemia.
- Trial name
- NCT01850108
- Testing
- Thymoglobulin, Fludarabine, Cyclophosphamide (CTX)
- Where
- 3 sites in United States, France and 1 more
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Vanderbilt-Ingram Cancer Center
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Ehler's Danlos Syndrome, Obstructive Sleep Apnea
Obstructive Sleep Apnoea in Children and Adolescents With Ehlers-Danlos Syndrome
Ehlers-Danlos Syndrome (EDS) is a clinically and genetically heterogeneous group of inherited connective tissue disorders characterized by joint hypermobility, skin hyperextensibility, and tissue fragility.
- Trial name
- NCT02712060
- Testing
- No intervention
- Where
- Switzerland
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Zurich
Beta-Thalassemia
Adherence of Beta Thalssemia Patients to Oral Chelation Therapy
The β-thalassemias are a group of inherited disorders of hemoglobin (Hb) synthesis characterized by chronic anemia of varying severity.
- Trial name
- NCT06568926
- Testing
- Serum Ferritin level
- Where
- Egypt
- Volunteers
- 200 people
- Sponsor
- Sohag University
Hemophilia B
Safety and Pharmacokinetic Study of a Recombinant Coagulation Factor IX Albumin Fusion Protein in Subjects With Hemophilia B
The primary objective of the study is to assess the safety of IV administration of rIX-FP. Safety will be evaluated by adverse events and laboratory changes over time.
- Trial name
- NCT01233440
- Testing
- Recombinant Coagulation Factor IX Albumin Fusion Protein, Plasma derived FIX [pdFIX]
- Where
- 20 sites in Austria, France and 4 more
- Volunteers
- 25 people
- Sponsor
- CSL Behring
Sickle Cell Disease (SCD)
Pharmacokinetics and Pharmacodynamics Study of SEG101 (Crizanlizumab) in Sickle Cell Disease (SCD) Patients With Vaso- Occlusive Crisis (VOC)
The purpose of the CSEG101A2202 study was to characterize the Pharmacokinetic (PK) and Pharmacodynamic (PD) of SEG101/crizanlizumab and to evaluate the safety and efficacy of SEG101/crizanlizumab in sickle cell disease (SCD)…
- Trial name
- NCT03264989
- Testing
- crizanlizumab
- Where
- 12 sites in United States
- Volunteers
- 57 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease
Sickle Cell Pro-Inflammatory Response to Interval Training Study
Recommendations for exercise prescription currently do not exist for individuals with sickle cell anemia (SCA) despite the known impact that SCA-related complications has on physical functioning and fitness.
- Trial name
- SPRINTS (NCT03653676)
- Testing
- Exercise Test (CIIT)
- Where
- 3 sites in United States
- Volunteers
- 110 people, healthy volunteers welcome
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Anemia
Iron Prophylaxis for Anemia in Infants With Cyanotic Congenital Heart Disease
Anemia is a common disorder in infants with one working chamber of the heart that pumps blood. Anemia is when the level of healthy blood cells becomes too low.
- Trial name
- NCT00459225
- Testing
- Iron (ferrous sulfate)
- Where
- United States
- Sponsor
- Emory University
Severe Hemophilia A Without Inhibitor
Clinical Outcomes of Low Dose PK-guided EHL FVIII Concentrates Versus Standard Prophylaxis in Severe Haemophilia A
Individualised pharmacokinetic (PK)-guided dosing of extended half-life (EHL) FVIII concentrates prophylaxis may reduce hemophilia A bleeding events than previous prophylactic regimen.
- Trial name
- NCT05281185
- Testing
- Extended half-life FVIII concentrates with PK-guided dosing, standard half-life FVIII concentrates with weight-based dosing
- Where
- Thailand
- Volunteers
- 15 people
- Sponsor
- Chulalongkorn University
Thrombocytopenia, Metachromatic Leukodystrophy
Stem Cell Transplantation (SCT) for Genetic Diseases
OBJECTIVES: I. Ascertain whether stem cell transplantation (SCT) is an effective method by which missing or dysfunctional enzymes can be replaced in patients with various inborn errors of metabolism. II.
- Conditions
- Thrombocytopenia, Metachromatic Leukodystrophy, Fanconi's Anemia, Thalassemia Major
- Trial name
- NCT00004378
- Testing
- Stem Cell Transplantation
- Where
- United States
- Sponsor
- National Center for Research Resources (NCRR)
Hemophilia B
Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B
To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including: 1) Unknown…
- Trial name
- NCT00484185
- Testing
- BeneFIX (coagulation factor IX (recombinant))
- Where
- South Korea
- Volunteers
- 183 people
- Sponsor
- Pfizer
Von Willebrand Disease, Blood Coagulation Disorders
Study of Safety and Efficacy of Antihemophilic Factor/Von Willebrand Factor Complex in Surgical Subjects With Von Willebrand Disease (vWD)
The purpose of this study is to test the safety and effectiveness of Humate-P® to prevent bleeding in patients with von Willebrand Disease who are undergoing surgery.
- Conditions
- Von Willebrand Disease, Blood Coagulation Disorders, Blood Platelet Disorders, Hematologic Disease
- Trial name
- NCT00168090
- Testing
- Blood coagulation Factor VIII and vWF, human
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- CSL Behring
Hemophilia a, Hemophilia B
The Efficacy and Safety of SerpinPC in Participants with Severe Hemophilia a or Moderately Severe to Severe Hemophilia B
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of prophylactic SerpinPC administered subcutaneously (SC) to participants with severe hemophilia A (HemA) (with or without…
- Trial name
- PRESent-2 (NCT05789524)
- Testing
- SerpinPC
- Where
- 59 sites in United States, Armenia and 13 more
- Volunteers
- 60 people
- Sponsor
- ApcinteX Ltd
Sickle Cell Anemia in Children, Sickle Cell Disease
Promoting Utilization and Safety of Hydroxyurea Using Precision in Africa
Sickle cell anemia (SCA) is among the world's most common and devastating blood disorders, affecting more than 300,000 newborns per year.
- Trial name
- PUSHUP (NCT05285917)
- Testing
- Hydroxyurea
- Where
- Angola
- Volunteers
- 400 people
- Sponsor
- Brown University
Sickle Cell Disease
The Implementation of the Automated Erythrocytapheresis in Egyptian Sickle Cell Disease Center
Improvements of health infrastructure, preventive care and clinical treatment have reduced the morbidity and mortality of sickle cell disease (SCD).
- Trial name
- NCT03903289
- Testing
- Automated red cell exchange, Manual red cell exchange, Simple red cell transfusion
- Where
- Egypt
- Volunteers
- 20 people
- Sponsor
- Ain Shams University
Sickle Cell Disease
A Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Crovalimab as Adjunct Treatment in Prevention of Vaso-Occlusive Episodes (VOE) in Sickle Cell Disease (SCD)
This study is designed to evaluate the efficacy, safety and pharmacokinetics of crovalimab compared with placebo as adjunct therapy in the prevention of VOEs in participants with SCD.
- Trial name
- CROSSWALK-c (NCT05075824)
- Testing
- Crovalimab
- Where
- 27 sites in United States, Brazil and 9 more
- Volunteers
- 95 people
- Sponsor
- Hoffmann-La Roche
Sickle Cell Anemia, Malaria
Sulfadoxine- Pyrimethamine Versus Weekly Chloroquine for Malaria Prevention in Children With Sickle Cell Anemia
Malaria is fatal and increases the risk of death among children with sickle cell anemia. Chemoprophylaxis significantly improves quality of life in these children.
- Trial name
- NCT00399074
- Testing
- sulfadoxine pyrimethamine
- Where
- Uganda
- Volunteers
- 220 people
- Sponsor
- Makerere University
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
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