Clinical trials: Beta Thalassemia-Major
Acute Leukemia, Severe Aplastic Anemia
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion.
- Conditions
- Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma
- Trial name
- NCT04099966
- Testing
- alpha beta depletion
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Mitchell Cairo
Beta-thalassemia Major Complicated With Diabetes
Zinc Supplementation in Patients With β-Thalassemia Major Complicated With Diabetes Mellitus
Beta-thalassemia represents a group of recessive inherited hemoglobin disorders characterized by reduced synthesis of β-globin chain.
- Trial name
- NCT03851055
- Testing
- Zinc
- Where
- Egypt
- Volunteers
- 80 people
- Sponsor
- Ain Shams University
Incentive Spirometer, Pulmonary Function
Effect of Incentive Respiratory Training on Pulmonary Functions and Functional Capacity in Children With B-thalassemia Major
The study was done to: 1. Investigate the effect of respiratory training on functional lung capacity and 2. To detect the effect of respiratory training on pulmonary functions in children with β-thalassemia major. 3.
- Conditions
- Incentive Spirometer, Pulmonary Function, Functional Capacity, Child, Only
- Trial name
- NCT07112703
- Testing
- Incentive spirometer exercises, Diaphragmatic breathing, Pursed lip breathing:
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Rawan Mohammed Khairy Mostafa Elsawy
Beta Thalassemia
Genetic Factors Affecting the Severity of Beta Thalassemia
Patients with genetic diseases can have widely differing severities. We are looking for genetic factors which influence the severity of beta thalassemia.
- Trial name
- NCT00159042
- Where
- Israel
- Volunteers
- 50 people
- Sponsor
- Deborah Rund
Thalassemia Major
Long Term Outcomes in β Thalassemia Major
Beta thalassemia (β-thalassemia) is the most common genetic disease worldwide. Individuals with thalassemia are born with a defect in hemoglobin.
- Trial name
- NCT02307786
- Where
- United States
- Volunteers
- 176 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
Cognitive Change, Beta-Thalassemia
Assessment of Cognitive Function, Fatigue and Health Related Quality of Life in Children With Beta Thalassemia
This study aims to: Assess the cognitive function in children with beta thalassemia Evaluate the fatigue in beta thalassemic children Assess the health related quality of life measures in children with beta thalassemia.
- Conditions
- Cognitive Change, Beta-Thalassemia, Fatigue, Quality of Life
- Trial name
- NCT05469230
- Where
- Egypt
- Volunteers
- 168 people, healthy volunteers welcome
- Sponsor
- Cairo University
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.
Thalassemia Major
Thalassemic Iron Overload Cardiomyopathy is Ameliorated by Taurine Supplementation
Hypothesis: Taurine, in combination with standard iron chelation therapy, is more effective than chelation therapy alone in reducing cardiac iron overload, oxidative stress and cardiac damage in β-Thalassemia.
- Trial name
- TICATS (NCT04291352)
- Testing
- Taurine
- Where
- Canada
- Volunteers
- 60 people
- Sponsor
- University Health Network, Toronto
Osteoporosis, Thalassemia Major
Diagnostic Pilot Study of Dual Energy Absorptiometry in the Detection of Osteopenia or Osteoporosis in Patients With Thalassemia Major
OBJECTIVES: I. Determine the frequency and severity of osteopenia and osteoporosis in patients with thalassemia major who undergo dual energy x-ray absorptiometry, and correlate these findings with other relevant endocrinologic…
- Conditions
- Osteoporosis, Thalassemia Major, Osteopenia
- Trial name
- NCT00006138
- Volunteers
- 35 people
- Sponsor
- National Center for Research Resources (NCRR)
Transfusion-dependent Beta-Thalassemia
Gene-Modified Stem Cell Therapy for Subjects With Transfusion-dependent Beta-thalassemia
This is a prospective, dual-centre, single dose, Phase IIb, single arm, open label study. The proposed clinical trial involves a single infusion of autologous HSPCs genetically modified with the GLOBE lentiviral vector, using an…
- Trial name
- NCT07680803
- Testing
- FT007
- Where
- 2 sites in Italy
- Volunteers
- 9 people
- Sponsor
- Fondazione Telethon
Thalassemia in Children, Pulmonary Hypertension
Erythrocyte Glutamine Level Relation to Pulmonary Hypertension Risk in Beta Thalassemia Major Children
The study will investigate the relation between erythrocyte glutamine/glutamate ratio and pulmonary hypertension risk in Egyptian thalassemic children in Assiut University Children Hospital
- Conditions
- Thalassemia in Children, Pulmonary Hypertension, Hemolysis
- Trial name
- NCT03133169
- Testing
- blood sample, Tricuspid regurge velocity
- Where
- Egypt
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Assiut University
Beta-Thalassemia, Iron Overload
Amlodipine as Adjuvant Treatment to Iron Chelation for Prevention of Cardiac Iron Overload in Thalassemia Patients
This is a randomized, open label, two arms superiority trial of a representative population of patients with a primary diagnosis of transfusion dependent thalassemia with evidence of moderate cardiac iron overload, defined as an…
- Trial name
- CANALI (NCT02474420)
- Testing
- Amlodipine, Deferasirox
- Where
- Canada
- Volunteers
- 60 people
- Sponsor
- Kevin H.M. Kuo, MD, MSc, FRCPC
Transfusion Dependent Beta-Thalassaemia
A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects with Transfusion Dependent Β-Thalassaemia.
This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.
- Trial name
- NCT05752123
- Testing
- ET-01
- Where
- China
- Volunteers
- 2 people
- Sponsor
- EdiGene (GuangZhou) Inc.
Thalassemia Majors (Beta-Thalassemia Major)
Risk Factors Affecting Growth in Thalassemic Children at AUCH
Thalassemia major is a hereditary hemoglobinopathy characterized by ineffective erythropoiesis and severe anemia, necessitating lifelong blood transfusions(1,2).
- Trial name
- NCT07509996
- Testing
- Non-interventional assessment of growth and risk factors in thalassemic children
- Volunteers
- 85 people
- Sponsor
- Assiut University
Thalassemia Major, Hemosiderosis
Efficacy Study in Removing Excess Iron From the Heart
The purpose of this study is to determine whether deferiprone has superior efficacy in removing excess iron from the heart when compared with deferoxamine.
- Trial name
- NCT00105495
- Testing
- Ferriprox (deferiprone), Desferal (deferoxamine)
- Where
- 4 sites in Greece, Italy
- Volunteers
- 60 people
- Sponsor
- ApoPharma
Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy
Determine knowledge, attitudes, and beliefs among adult patients, and parents of pediatric patients, with transfusion dependent beta-thalassemia and sickle cell disease toward gene therapy to treat their or their child's illness…
- Trial name
- NCT06664541
- Where
- United States
- Volunteers
- 100 people
- Sponsor
- Georgetown University
Thalassemia Major
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT).
- Trial name
- NCT04009525
- Testing
- Busulfan, Cyclophosphamide, Fludarabine
- Where
- China
- Volunteers
- 823 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Beta-Thalassemia, Thalassemia Major
Efficacy Study of the Use of Sequential DFP-DFO Versus DFP
Changes in chelation treatment and transfusion practices, during the past two decades, have dramatically improved the prognosis of thalassemia major patients.Deferiprone (DFP) has been compared with deferoxamine (DFO), using…
- Trial name
- SEQDFPDFO (NCT00733811)
- Testing
- Deferiprone (DFP) and Deferoxamine (DFO), Deferiprone (DFP)
- Where
- Italy
- Volunteers
- 213 people
- Sponsor
- Azienda Ospedaliera V. Cervello
Beta-Thalassemia
Thrombophilia In Beta Thalassemia
β-thalassemia disease is one of the most common congenital hemolytic anemia commonly found in the malarial belt areas including the Mediterranean, the Middle East, Africa, Southeast Asian countries, and China.
- Trial name
- NCT04219449
- Testing
- PT, Protein C, Platelet aggregation by ADP and arachidonic acid
- Volunteers
- 100 people
- Sponsor
- Assiut University
Liver Iron Overload
Safety and Efficacy of Deferasirox in Patients With Transfusion Dependent Iron Overload - a Non-comparative Extension Study
The purpose of this study is to assess the safety and the effects on liver iron of Deferasirox when given for a long treatment period in patients with transfusion dependent iron overload.
- Trial name
- NCT01033747
- Testing
- Deferasirox
- Where
- 4 sites in Italy
- Volunteers
- 70 people
- Sponsor
- Novartis Pharmaceuticals