Clinical trials: Lymphatic Abnormalities
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa Pegol (N8-GP) in Previously Untreated Patients With Haemophilia A
This trial is conducted globally. The aim of the trial is to investigate the safety and efficacy of turoctocog alfa pegol (N8-GP) in previously untreated patients (PUPs) with haemophilia A.
- Trial name
- pathfinder™6 (NCT02137850)
- Testing
- turoctocog alfa pegol
- Where
- 96 sites in United States, Algeria and 21 more
- Volunteers
- 124 people
- Sponsor
- Novo Nordisk A/S
Sickle Cell Disease
Dense Red Blood Cells in Sickle Cell Children
Quantitative and prognostic evaluation of dense red blood cells in sickle cell children: preliminary single center study from the Creteil pediatric cohort.
- Trial name
- DREPADENSE (NCT02887118)
- Where
- France
- Volunteers
- 82 people
- Sponsor
- Centre Hospitalier Intercommunal Creteil
Brain and Central Nervous System Tumors, Chordoma
Polyvinylpyrrolidone-Sodium Hyaluronate Gel in Reducing Pain From Oral Mucositis in Young Patients With Cancer
RATIONALE: Polyvinylpyrrolidone-sodium hyaluronate gel may lessen the pain of oral mucositis, or mouth sores, in patients undergoing treatment for cancer.
- Conditions
- Brain and Central Nervous System Tumors, Chordoma, Kidney Cancer, Leukemia
- Trial name
- NCT00349024
- Testing
- polyvinylpyrrolidone-sodium hyaluronate gel
- Where
- 21 sites in Ireland, United Kingdom
- Volunteers
- 50 people
- Sponsor
- Children's Cancer and Leukaemia Group
Hemophilia
The Effects of Exercise Training in Children With Hemophilia
This study aims to investigate the effects of an individually planned exercise program, counseling and family education on the followings in patients with hemophilia (PWH); 1.
- Trial name
- NCT04703062
- Testing
- Experimental group 1, Experimental group 2
- Where
- Turkey (Türkiye)
- Volunteers
- 30 people
- Sponsor
- Istanbul University - Cerrahpasa
Hemophilia A
Pharmacokinetic Study of SCT800 in Previously Treated Patients With Hemophilia A
Participants will be assigned to A or B groups with a scale of 1:1 based on a prospectively randomized treatment-sequence assignment, i.e. infuse SCT800 followed by Xyntha (group A), or the alternate sequence (group B).
- Trial name
- NCT02888223
- Testing
- SCT800, Xyntha
- Where
- China
- Volunteers
- 16 people
- Sponsor
- Sinocelltech Ltd.
Hemophilia A, Hemophilia B
Treatment Patterns And Outcomes In Patients Treated With Benefix Or Refacto/Refacto AF - A Swedish Cohort Study
The overall aim of the study is to describe demographic and clinical characteristics, treatment patterns and outcomes, in the populations of hemophilia patients treated with BeneFIX and ReFacto/ReFacto AF in Sweden
- Trial name
- NCT02740413
- Volunteers
- 86 people
- Sponsor
- Pfizer
Hemophilia A, Hemophilia B
Data Registry in Chinese Hemophilia A and B Patients
This is an open-label, prospective, multi-center, patient registry in Chinese Hemophilia A and B patients treated with anti-hemophilic coagulation factor replacement therapy.
- Trial name
- NCT07101224
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Institute of Hematology & Blood Diseases Hospital, China
Sickle Cell Disease
Ibuprofen and Opioid (Morphine or Diamorphine) for Acute Pain in Sickle Cell Disease - Sickle With Ibuprofen & Morphine
The use of oral ibuprofen combined with Opioid (Morphine or Diamorphine) administered through patient controlled analgesia (PCA) will be clinically effective for acute pain crisis in adults with sickle cell disease (SCD).
- Trial name
- SWIM (NCT00880373)
- Testing
- Ibuprofen, Diamorphine or Morphine
- Where
- 2 sites in United Kingdom
- Volunteers
- 320 people
- Sponsor
- London North West Healthcare NHS Trust
Hematologic Malignancies, Nonmalignant Diseases
Study of Melphalan Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients
Melphalan is a chemotherapy drug used extensively in bone marrow transplantation. The goal of this study is to determine what causes some children to have different drug concentrations of melphalan in their bodies and if drug…
- Conditions
- Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- NCT03609827
- Testing
- Melphalan
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of California, San Francisco
Hemophilia A
Non Neutralizing Antibodies: Prevalence and Characterization
Antibodies (Abs) directed against factorVIII (FVIII) remain the main iatrogenic complication in haemophilia A (HA) patients. Anti-FVIII Abs inhibiting pro-coagulant properties of the molecule are named inhibitors whereas Abs…
- Trial name
- NCT01541527
- Testing
- blood test
- Where
- France
- Sponsor
- University Hospital, Montpellier
Sickle Cell Disease
Evaluation of Knowledge Among Adolescents With Sickle Cell Disease.
Evaluation of knowledge about contraception in sickle cell adolescents.
- Trial name
- SEXODREP (NCT03235011)
- Where
- France
- Volunteers
- 99 people
- Sponsor
- Adele CARLIER-GONOD
Kaposiform Hemangioendotheliomas, Tufted Angioma
Safety and Efficacy Study of Sirolimus in Complicated Vascular Anomalies
The purpose of this study is to determine if the use of sirolimus in the treatment of children and young adults with complicated vascular anomalies will prove to be safe and provide objective response resulting in improved…
- Conditions
- Kaposiform Hemangioendotheliomas, Tufted Angioma, Capillary Venous Lymphatic Malformation, Venous Lymphatic Malformation
- Trial name
- NCT00975819
- Testing
- sirolimus
- Where
- 2 sites in United States
- Volunteers
- 61 people
- Sponsor
- Denise Martin Adams
Hemophilia A
A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A
This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in…
- Trial name
- NCT05911763
- Testing
- Efanesoctocog Alfa BIVV001
- Where
- 28 sites in United States
- Volunteers
- 200 people
- Sponsor
- Sanofi
Sickle Cell Disease
Prevalence and Pathophysiology of Systemic Arterial Pressure Abnormalities in Childhood Sickle Cell Disease
It is usually found that the blood pressure of adults with sickle cell disease is lower than in non-sickle cell patients. On the other hand, three recent prospective studies in children with sickle cell disease show prevalence of…
- Trial name
- DrépaPA (NCT04911049)
- Testing
- Blood Pressure measurement
- Where
- France
- Volunteers
- 200 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Congenital Bleeding Disorder, Haemophilia A
Project to Update the Study of Congenital Haemophilia in Spain
This study is conducted in Europe. The aim of the study is to update the knowledge about the prevalence and severity of congenital haemophilia (A and B) in Spain.
- Conditions
- Congenital Bleeding Disorder, Haemophilia A, Haemophilia B
- Trial name
- PUCHS (NCT01959555)
- Testing
- No treatment given
- Where
- Spain
- Volunteers
- 1,157 people
- Sponsor
- Novo Nordisk A/S
Glanzmann Thrombasthenia
Anti-αIIbβ3 Immunization in Glanzmann Thrombasthenia: Prevalence and Associated Risk Factors: Thrombasthenia Anti-αIIbβ3 Antibodies Study (TAAS)
This project aim to correlate risk factors (genetic, therapeutic and socio-demographic factors) to anti-αIIbβ3 antibodies formation following blood products transfusion (platelets or packed red cells) or pregnancy in a national…
- Trial name
- TAAS (NCT04595617)
- Testing
- Antibodies screening
- Where
- 7 sites in France
- Volunteers
- 28 people
- Sponsor
- University Hospital, Bordeaux
Beta-Thalassemia Major, Sickle Cell Disease
A Study Evaluating the Safety and Efficacy of LentiGlobin BB305 Drug Product in β-Thalassemia Major (Also Referred to as Transfusion-dependent β-Thalassemia [TDT]) and Sickle Cell Disease
This is a Phase 1/2, open label, safety, and efficacy study of the administration of LentiGlobin BB305 Drug Product to participants with either transfusion dependent beta-thalassemia (TDT) or sickle cell disease (SCD).
- Trial name
- NCT02151526
- Testing
- LentiGlobin BB305 Drug Product
- Where
- France
- Volunteers
- 7 people
- Sponsor
- Genetix Biotherapeutics Inc.
Hereditary Spherocytosis
Hereditary Spherocytosis and Vascular Function
Background : Patients with hereditary spherocytosis (HS) are characterized by increased red blood cell (RBC) fragility and a loss of RBC deformability.
- Trial name
- VASCUSPHERO (NCT04451785)
- Testing
- blood sample, Pulse wave velocity
- Where
- 2 sites in France
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- Hospices Civils de Lyon
Factor XIII Deficiency
An Open Enrollment Study of Factor XIII Concentrate in Subjects With Congenital Factor XIII Deficiency
Congenital deficiency of factor XIII is an extremely rare inherited disorder associated with potentially life-threatening bleeding. Factor XIII Concentrate is given to patients whose blood is lacking factor XIII.
- Trial name
- NCT00945906
- Testing
- FXIII Concentrate (Human) (FXIII)
- Where
- 25 sites in United States
- Volunteers
- 61 people
- Sponsor
- CSL Behring
Sickle Cell Disease, Sickle Cell Anemia
Stroke Prevention in Young Adults With Sickle Cell Anemia
Sickle cell disease (SCD) is the most common genetic disease, affecting about 25 million people worldwide. Approximately 150,000 Nigerian children are born each year with sickle cell disease (SCD), making it the country with the…
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia, Stroke, Ischemic, Silent Stroke
- Trial name
- SPIYA (NCT04808778)
- Testing
- Hydroxyurea therapy per standard care
- Where
- 2 sites in United States, Nigeria
- Volunteers
- 250 people
- Sponsor
- Vanderbilt University Medical Center
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