Clinical trials: Lymphatic Abnormalities
Thalassemia Major
Perceptions of Thalassemia Major in Singapore: An Exploratory Study of Stigma
Background: Thalassemia major (TM) is a chronic disorder that affects a person s ability to produce hemoglobin, resulting in anemia.
- Trial name
- NCT01129076
- Where
- 2 sites in Singapore
- Volunteers
- 30 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
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
Von Willebrand Disease
Study of Voncento® in Subjects With Von Willebrand Disease
This is a multi-centre, open-label, single-arm, phase 4, post-marketing study to further investigate the efficacy and safety of Voncento in subjects with Von Willebrand Disease (VWD) in whom treatment with a Von Willebrand Factor…
- Trial name
- NCT02552576
- Testing
- Voncento
- Where
- 12 sites in Austria, Germany and 3 more
- Volunteers
- 26 people
- Sponsor
- CSL Behring
Hemophilia A
Study Evaluating ReFacto® in Hemophilia A Undergoing Major Surgery
The primary objective of this clinical research study is to evaluate the safety and efficacy of ReFacto in subjects with hemophilia A undergoing major surgery monitored using the chromogenic substrate assay at the local…
- Trial name
- NCT00092976
- Testing
- ReFacto
- Where
- 4 sites in United States
- Volunteers
- 15 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Hemophilia
Musculoskeletal Function in Hemophilia
Hemophilia, which results from deficiency of factor VIII or IX, is a common hereditary X-linked bleeding disorder affecting up to 10/100,000 population. About 60-70% of them have severe disease (factor level <1%).
- Trial name
- NCT00324493
- Where
- 10 sites in Argentina, Brazil and 7 more
- Volunteers
- 250 people
- Sponsor
- Christian Medical College, Vellore, India
Quality of Life (QOL), Patient-Reported Outcome Measures (PROMs)
Validation of the EQ-5D-Y-3L and EQ-5D-Y-5L for Paediatric Patients in China
The goal of this observational study is to validate the EQ-5D-Y-3L and EQ-5D-Y-5L in measuring health-related quality of life (HRQoL) for paediatric patients with different health conditions in China.
- Conditions
- Quality of Life (QOL), Patient-Reported Outcome Measures (PROMs), Pneumonia Childhood, Neuroblastoma (NB)
- Trial name
- NCT06873672
- Where
- China
- Volunteers
- 360 people
- Sponsor
- RenJi Hospital
Fanconi Anemia, Severe Aplastic Anemia
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine…
- Conditions
- Fanconi Anemia, Severe Aplastic Anemia, Myelodysplastic Syndromes, T Cell Receptor Alpha/Beta Depletion
- Trial name
- NCT03579875
- Testing
- Total Body Irradiation (TBI) (Plan 1), Cyclophosphamide (CY) (Plan 1), Fludarabine (FLU)
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Sickle Cell Disease
Iron Mediated Vascular Disease in Sickle Cell Anemia Patients
The purpose of this research study is to determine the frequency and severity of iron overload in patients with Sickle Cell Anemia and its relationship to blood vessel function.
- Trial name
- NCT01239901
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- Children's Hospital Los Angeles
Neonatal Thrombocytopenia, Intraventricular Hemorrhage
Monitoring Outcome in Neonatal Thrombocytopenia
Rationale: Approximately 10% of neonates admitted to neonatal intensive care units develop a major hemorrhage. In an attempt to avert this severe complication various preventive measures have been implemented.
- Conditions
- Neonatal Thrombocytopenia, Intraventricular Hemorrhage, Platelet Transfusion
- Trial name
- MONET (NCT03110887)
- Volunteers
- 700 people
- Sponsor
- Sanquin-LUMC J.J van Rood Center for Clinical Transfusion Research
Congenital Bleeding Disorder, Haemophilia B
Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B
This trial is conducted in Africa, Asia, Europe and the United States of America (USA). The aim of the trial is to evaluate the safety and efficacy of NNC-0156-0000-0009 (nonacog beta pegol) during surgical procedures in patients…
- Trial name
- paradigm™ 3 (NCT01386528)
- Testing
- nonacog beta pegol
- Where
- 41 sites in United States, Austria and 15 more
- Volunteers
- 13 people
- Sponsor
- Novo Nordisk A/S
Osteonecrosis
Functional Outcomes Following Hip Core Decompression in Younger Participants With Osteonecrosis
Although uncommon in the general pediatric population, osteonecrosis (ON) is prevalent in children and adolescents with cancer, particularly among those that have had chronic exposure to glucocorticoids and among those having…
- Trial name
- NCT02458937
- Testing
- Functional Mobility Assessment, GAITRite® System, Range of Motion
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- St. Jude Children's Research Hospital
Haemophilia
Evaluation of Xylitol Wipes as an Oral Hygiene Protocol for Haemophilic Children
The purpose of this study is to evaluate xylitol wipes as an oral hygiene protocol for haemophilic children
- Trial name
- NCT05899075
- Testing
- xylitol wipes
- Where
- Egypt
- Volunteers
- 30 people
- Sponsor
- Al-Azhar University
Lymphatic Abnormality
Injection Sclerotherapy With Bleomycin VS Doxycycline in Patients With Lymphatic Malformations
This is a randomized clinical trial comparing the safety and effectiveness of injection sclerotherapy using bleomycin versus doxycycline in children under 12 years with lymphatic malformations.
- Trial name
- NCT07320001
- Testing
- Bleomycin, doxycycline (tetracycline)
- Volunteers
- 92 people
- Sponsor
- Basharat Hussain
Leukemia, Myelodysplastic Syndromes
Evaluating Cell Damage in Patients With Acute Myeloid Leukemia, Myelodysplastic Syndromes, or Fanconi Anemia; in Patients Who Were Exposed to Alkylating Agents; and in Healthy Volunteers
RATIONALE: Studying samples of bone marrow from patients with cancer and from healthy volunteers in the laboratory may help doctors learn more about changes that occur in bone marrow stromal (connective tissue) cells.
- Trial name
- NCT00899795
- Testing
- cytogenetic analysis, fluorescence in situ hybridization, flow cytometry
- Where
- United States
- Volunteers
- 35 people, healthy volunteers welcome
- Sponsor
- OHSU Knight Cancer Institute
Marfan Syndrome, Turner Syndrome
National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions
The National Registry of Genetically Triggered Thoracic Aortic Aneurysms and Cardiovascular Conditions (GenTAC) was initiated in 2006 by the National Heart, Lung, and Blood Institute (NHLBI) and the National Institute of…
- Conditions
- Marfan Syndrome, Turner Syndrome, Ehlers-Danlos Syndrome, Loeys-Dietz Syndrome
- Trial name
- GenTAC (NCT01322165)
- Where
- 7 sites in United States
- Volunteers
- 3,706 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Bone Marrow Transplant (BMT)
HPC Offered for PRESERVE Expansion
Ossium recognizes that not all patients who could benefit from Ossium's HPC, Marrow can access the PRESERVE trial for logistical or eligibility reasons.
- Trial name
- HOPE (NCT07145411)
- Testing
- Bone Marrow Transplant with Ossium's HPC, Marrow (bone marrow recovered from deceased organ and tissue donors)
- Where
- 5 sites in United States
- Volunteers
- 10 people
- Sponsor
- Ossium Health, Inc.
Hemophilia B
Post Marketing Study in Haemophilia B Patients Using Nonafact® (Human Coagulation Factor IX)
In this postmarketing study, the safety of Nonafact® (human coagulation factor IX) is evaluated in previous treated and untreated patients with severe, moderate or mild haemophilia B.
- Trial name
- NCT00139828
- Testing
- human coagulation Factor IX
- Where
- 7 sites in Netherlands
- Volunteers
- 12 people
- Sponsor
- Prothya Biosolutions
Niemann-Pick Disease, Type C
Establishment of Genomic and Phenotypic Database for Niemann-Pick Disease, Type C
Background: Niemann-Pick type C (NPC) disease is a rare, progressive neurodegenerative disease that affects mainly the brain, liver, and spleen but also other parts of the body.
- Trial name
- NCT05588167
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Hemophilia B
A Safety and Efficacy Extension Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Patients With Hemophilia B
This study will examine the long-term safety and efficacy of rIX-FP for the control and prevention of bleeding episodes in children and adults with severe hemophilia B.
- Trial name
- NCT02053792
- Testing
- rIX-FP
- Where
- 42 sites in United States, Australia and 13 more
- Volunteers
- 97 people
- Sponsor
- CSL Behring
Von Willebrand Diseases
Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy…
- Trial name
- NCT04052698
- Testing
- Wilate
- Where
- 14 sites in United States, Belarus and 6 more
- Volunteers
- 43 people
- Sponsor
- Octapharma
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