Clinical trials: Thalassaemia
Sickle Cell Disease
Sickle Cell Anemia in an Arab Bedouin Village in the Northern Israel
Sickle cell anemia and sickle cell thalassemia are frequent diseases among the israeli arab population. The purpose of this study is to assess the clinical characteristics of the patients in one arab village and the laboratory…
- Trial name
- NCT00481039
- Testing
- Medical history and basic laboratory analysis
- Where
- Israel
- Volunteers
- 300 people
- Sponsor
- HaEmek Medical Center, Israel
Anemia, Sickle Cell
Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease
The purpose of the study is to determine the maximum tolerated dose, safety and effect on induction of fetal hemoglobin of pomalidomide in patients with Sickle Cell Disease.
- Trial name
- SCD-001 (NCT01522547)
- Testing
- pomalidomide
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Celgene
Sickle Cell Disease, Thalassemia
Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling
Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of healthy cells.
- Conditions
- Sickle Cell Disease, Thalassemia, Hemoglobinopathy
- Trial name
- NCT06313398
- Testing
- Biotin-labeled red blood cells
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Iron, Abnormal Blood Level, Other Abnormal Blood Chemistry
Fresh Versus Old Red Blood Cells for Transfusion
Packed red blood cell units destined for transfusion can be stored for up to 42 days prior to transfusion based on Food and Drug Administration (FDA) guidelines.
- Trial name
- NCT01319552
- Testing
- Fresh transfusion, Old transfusion
- Where
- United States
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Columbia University
Hemoglobin Mutations, Erythrocyte Variants
Collection of Blood From Persons With Hemoglobin and Erythrocyte Polymorphisms for Laboratory Malaria Research
This study will collect blood samples for use in laboratory studies of malaria. The World Health Organization reports that 40 percent of the world's population is at risk for malaria, mostly in the poorest countries.
- Trial name
- NCT00277849
- Where
- United States
- Volunteers
- 130 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Atrial Fibrillation, Thalassemia
Atrial Fibrillation in Beta-Thalassemia
The study aims to evaluate the clinical, laboratory and instrumental differences that exist between beta-thalassemia patients with atrial fibrillation and those not affected by arrhythmia.
- Trial name
- NCT05508932
- Where
- Italy
- Volunteers
- 350 people
- Sponsor
- University Hospital of Ferrara
Thalassemia, Iron Overload
Evaluation of the Quality of Life in Patients With Chronic Iron Overload Due to Hemoglobinopathies in Greece.
This observational clinical study aims to evaluate the HRQoL of thalassemia patients with iron overload in Greece, who are under treatment with deferasirox based on standard clinical practice.
- Trial name
- FEROUSA (NCT07352878)
- Volunteers
- 150 people
- Sponsor
- Elpen Pharmaceutical Co. Inc.
Sickle Cell Disease
Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease
A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own…
- Trial name
- GRASP-EA (NCT07640815)
- Testing
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Where
- United States
- Sponsor
- David Williams
Iron Overload After Hematopoietic Stem Cell Transplantation (HSCT) in Patients With Beta-thalassemia Major
Post Hematopoietic Stem Cell Transplantation
This was a prospective, single-arm, multicenter, national, phase II clinical study. The purpose of this Phase II study was to examine the safety and efficacy of deferasirox to decrease iron overload (IOL) in the posttransplant…
- Trial name
- NCT01610297
- Testing
- ICL670
- Where
- 6 sites in Turkey (Türkiye)
- Volunteers
- 27 people
- Sponsor
- Novartis Pharmaceuticals
Sickle Cell Disease & Thalassemia
Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies
Allogeneic Stem Cell Transplantation of NiCord®, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
- Trial name
- NCT01590628
- Testing
- NiCord
- Where
- 3 sites in United States
- Volunteers
- 16 people
- Sponsor
- Gamida Cell ltd
Beta-Thalassemia
Detection Of β-thalassemia Carriers In Assiut
Thalassemia is different in kids with microcytic hypochromic anemia than general population because there is a confusion between symptoms of thalassemia and iron deficiency anemia in kids and both of them differ in management and…
- Trial name
- NCT03822585
- Testing
- CBC, Iron Study, Serum Ferrittin, HPLC,Genitic Study
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Assiut University
Hurler Syndrome, Fanconi Anemia
Bone Marrow Transplant With Abatacept for Non-Malignant Diseases
This is a single arm, phase I study to assess the tolerability of abatacept when combined with cyclosporine and mycophenolate mofetil as graft versus host disease prophylaxis in children undergoing unrelated hematopoietic stem…
- Conditions
- Hurler Syndrome, Fanconi Anemia, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome
- Trial name
- NCT01917708
- Testing
- Abatacept
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Emory University
MRI Scans
Quantitative Susceptibility Mapping (QSM) to Guide Iron Chelating Therapy
The overall goal of this research is to help develop a new magnetic resonance (MR) method, Quantitative Susceptibility Mapping (QSM), to improve the measurement of liver iron concentrations without the need for a liver biopsy.
- Trial name
- NCT04171635
- Testing
- Quantitative Susceptibility Mapping (QSM) Magnetic Resonance Imaging (MRI), R2* Magnetic Resonance Imaging (MRI)
- Where
- 2 sites in United States
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- Weill Medical College of Cornell University
Sickle Red Blood Cell, Fetal Hemoglobin
Effect of Broccoli Sprouts Homogenate on SS RBC
The overall purpose of this study is to obtain a better understanding of the biological response of red blood cells to sulforaphane contained in fresh broccoli sprouts that have been put through a blending process.
- Conditions
- Sickle Red Blood Cell, Fetal Hemoglobin, Oxidative Stress
- Trial name
- BSH (NCT01715480)
- Testing
- Broccosprouts® (Brassica Protection Products LLC) homogenate
- Where
- United States
- Volunteers
- 21 people, healthy volunteers welcome
- Sponsor
- Duke University
Thalassemia, Beta, Thalassemia
Impact of Virtual Reality on Pain and Anxiety During Intravenous Cannulation Procedures Among Thalassemia Patients in UAE.
The goal of this trial is to evaluate the effects of therapeutic Virtual Reality (VR) on pain, anxiety, fatigue and boredom during intravenous (IV) cannulations in comparison to standard of care (SOC).The main question it aims to…
- Conditions
- Thalassemia, Beta, Thalassemia, Pain, Anxiety
- Trial name
- NCT07099196
- Testing
- Headset-based Virtual Reality Simulation
- Where
- United Arab Emirates
- Volunteers
- 115 people
- Sponsor
- Mohammed Bin Rashid University of Medicine and Health Sciences
Sickle Cell Disease, Thalassemia, Beta
Study of the Role of Genetic Modifiers in Hemoglobinopathies
This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).
- Conditions
- Sickle Cell Disease, Thalassemia, Beta, Thalassemia Alpha, Hemoglobinopathies
- Trial name
- INHERENT (NCT05799118)
- Testing
- GWAS
- Where
- 26 sites in United States, Angola and 14 more
- Volunteers
- 30,000 people
- Sponsor
- Cyprus Institute of Neurology and Genetics
Pain
The Effect of Acupressure on Pain in Children With Thalassemia
This randomized controlled trial evaluates the effect of acupressure on reducing the pain of blood collection in children with Thalassemia. The hypothesis of this study is that acupressure application reduces acute pain.
- Trial name
- acupressure (NCT06163196)
- Testing
- acupressure
- Where
- Turkey (Türkiye)
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- Mersin University
Transfusion-dependent Thalassemia, Non-transfusion-dependent Thalassemia
Open-label, Multicenter Study Assessing Preference for Deferasirox Film-coated Tablet Compared to Dispersible Tablet
Study to evaluate patient preference of deferasirox film-coated tablet (FCT) or deferasirox dispersible tablet (DT) in patient with transfusion - dependent thalassemia or non-transfusion -dependent thalassemia as measured by…
- Trial name
- Jupiter (NCT02993224)
- Testing
- Deferasirox dispersable tablet (DT), Deferasirox film coated tablet (FCT)
- Where
- 17 sites in Egypt, Lebanon and 5 more
- Volunteers
- 148 people
- Sponsor
- Novartis Pharmaceuticals
Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA)
Traditional Chinese Medicine in the Supportive Management of Anaemic and Cytopenic (Leukopenia, Thrombocytopenia) Haematological Disorders
The purpose of this study is to study the efficacy of Traditional Chinese Medicine (TCM) on anaemic and cytopenic haematological disorders including myelodysplastic syndrome (MDS), aplastic anaemia (AA), myelofibrosis (MF) and…
- Conditions
- Myelodysplastic Syndrome (MDS), Aplastic Anaemia (AA), Myelofibrosis (MF), Thalassemia Intermedia
- Trial name
- NCT01224496
- Testing
- Chinese herbal concoction twice a day for 6 months
- Where
- Singapore
- Volunteers
- 38 people
- Sponsor
- Singapore General Hospital