Clinical trials: Beta Thalassemia-Major
Thalassemia Major
Monitoring of Erythroid Lineage Specific Chimerism Following Allogeneic Hematopoietic Transplantation for Thalassemia Major
The purpose of this study is to collect peripheral blood and bone marrow aspirate samples from thalassemia patients in Tehran, in a collaborative effort to develop an erythroid lineage specific chimerism assay applicable to…
- Trial name
- NCT00480506
- Where
- 2 sites in United States, Iran
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Dana-Farber Cancer Institute
Microcytic Hypochromic Anemia
Red Cell Distribution Width Index Versus Red Cell Distribution Width as Discriminating Guide for Iron Deficiency Anaemia and Beta Thalassemia Trait .
Red Cell Distribution Width Index versus Red Cell Distribution Width as Discriminating Guide for Iron Deficiency Anaemia and Beta Thalassemia Trait .
- Trial name
- NCT03868306
- Testing
- CBC , Iron study and Haemoglobin electrophoresis .
- Volunteers
- 100 people
- Sponsor
- Assiut University
Anesthesia, Pediatric Cancer
the Efficacy and Safety of Sugammadex in Children 0-2 Years Old
Sugammadex is a selective antidote to muscle relaxants rocuronium bromide and vecuronium bromide. Sugammadex is a modified gamma-cyclodextrin, a compound that selectively binds rocuronium bromide and vecuronium bromide.
- Conditions
- Anesthesia, Pediatric Cancer, Pediatric Tumor, Pediatric Hepatoblastoma
- Trial name
- NCT03728543
- Testing
- Sugammadex
- Where
- Russia
- Volunteers
- 62 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
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
B Thalassemia
Effect of Metoprolol on Thalassemia Cardiomyopathy
effect of B blocker was first evaluated in patient with cardiomyopathy not induced by ischemia and idiopathic which as the most common causes of cardiomyopathy.
- Trial name
- NCT01863173
- Testing
- metoprolol
- Where
- Iran
- Volunteers
- 45 people
- Sponsor
- Shiraz University of Medical Sciences
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
Transfusion-dependent Hemachromatosis, Thalassemia Major
Pilot Study for Patients With Poor Response to Deferasirox
This purpose of this study is to understand the differences between people who have a good response to deferasirox (exjade) compared to people who have a poor response to this medication when used for transfusion-dependent iron…
- Conditions
- Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
- Trial name
- NCT00749515
- Testing
- Deferoxamine, Deferasirox, HIDA
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Boston Children's Hospital
Anemia (Iron-Loading), Beta-Thalassemia
Chelation Therapy of Iron Overload With Pyridoxal Isonicotinoyl Hydrazone
To demonstrate the safety and effectiveness of orally-administered pyridoxal isonicotinoyl hydrazone (PIH) for the chronic treatment of iron overload.
- Conditions
- Anemia (Iron-Loading), Beta-Thalassemia, Hematologic Diseases, Hemoglobinopathies
- Trial name
- NCT00000588
- Testing
- Chelation therapy
- Volunteers
- 120 people
- Sponsor
- Case Western Reserve University
β-thalassemia Major
A Phase 2 Safety and Efficacy Study Evaluating CS-101 in Participants With β-Thalassemia Major
The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating patients with β-Thalassemia Major
- Trial name
- NCT07489196
- Testing
- CS-101 injection
- Where
- 3 sites in China
- Volunteers
- 20 people
- Sponsor
- CorrectSequence Therapeutics Co., Ltd
Beta Thalassemia
Evaluation of Heart Status in Patients of Beta Thalassemia Using Echocardiogram
Heart failure from myocardial iron deposition is a severe complication for patients with transfusion-dependent beta thalassemia .
- Trial name
- NCT04929574
- Testing
- Echocardiogram and Hematological Analysis
- Where
- China
- Volunteers
- 1,200 people
- Sponsor
- Nanfang Hospital, Southern Medical University
Erythrocyte Transfusion, Beta-Thalassemia
An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia
This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require…
- Trial name
- BELIEVE (NCT02604433)
- Testing
- Luspatercept
- Where
- 76 sites in United States, Australia and 13 more
- Volunteers
- 336 people
- Sponsor
- Celgene
Beta-Thalassemia
Safety and Efficacy Evaluation of BRL-101 in Subjects With Transfusion-Dependent β-Thalassemia
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in subjects with Transfusion-Dependent β-Thalassemia (TDT).
- Trial name
- NCT05577312
- Testing
- BRL-101
- Where
- 7 sites in China
- Volunteers
- 39 people
- Sponsor
- Bioray Laboratories
Thalassemia Major
Evaluation of Nutritional Status in Thalassemia Major Patients in Assiut Children Hospital
Thalassemia is a blood disorder passed down through families in which the body makes an abnormal form of hemoglobin. There are 2 main types of thalassemia; Alpha \& Beta thalassemia.
- Trial name
- NCT03161899
- Where
- Egypt
- Volunteers
- 246 people
- Sponsor
- Assiut University
Transfusional Iron Overload, Thalassemia Major
High-Tc Susceptometer to Monitor Transfusional Iron Overload
The proposed research project will continue the application and development of a new method (biomagnetic susceptometry) that measures magnetic fields to determine how much iron is in the liver.
- Conditions
- Transfusional Iron Overload, Thalassemia Major, Sickle Cell Disease, Myelodysplastic Syndromes
- Trial name
- NCT01241357
- Testing
- Hepatic biomagnetic susceptibility measurement
- Where
- United States
- Volunteers
- 99 people, healthy volunteers welcome
- Sponsor
- Columbia University
Beta Thalassemia Major
Iron Accumulation in Erythrocytes in Patients
Thalassemia refers to a group of blood disorders characterized by a decrease or absence of the synthesis of one or more normal globin chains.
- Trial name
- NCT07486531
- Where
- Turkey (Türkiye)
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Ankara City Hospital Bilkent
Fetal Hemoglobin, Beta-Thalassemia
Long Term Beta Thalassemia Treatment: Findings From The Extension Period
The project, titled "Long Term Beta Thalassemia Treatment: Findings From The Extension Period Of Phase 2 Clinical Trial," aims to compare the efficacy and safety of combination therapy (thalidomide and hydroxyurea) versus…
- Conditions
- Fetal Hemoglobin, Beta-Thalassemia, Hemoglobinopathies
- Trial name
- Thalidomide (NCT06490601)
- Testing
- thalidomide and hydroxyurea, Thalidomide
- Where
- Pakistan
- Volunteers
- 30 people
- Sponsor
- National Institute of Blood and Marrow Transplant (NIBMT), Pakistan
Transfusion Dependent Beta-Thalassaemia
A Safety and Efficacy Study Evaluating ET-01 in Subjects with Transfusion Dependent Β-Thalassaemia
This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia.
- Trial name
- ET-01 (NCT04925206)
- Testing
- ET-01
- Where
- 4 sites in China
- Volunteers
- 8 people
- Sponsor
- EdiGene (GuangZhou) Inc.
Beta Thalassemia Major Anemia
The Potential Hepatoprotective Effect of Metformin in Patients With Beta Thalassemia Major
Beta Thalassemia is a major public health problem in Mediterranean countries.In Egypt, it is considered as the most common chronic hemolytic anemia.one of the major complications in this inherited disorder is iron overload which…
- Trial name
- NCT02984475
- Testing
- Metformin
- Where
- 2 sites in Egypt
- Volunteers
- 60 people
- Sponsor
- Cairo University
Thalassemia
Effect of Antioxidant Cocktail in Beta-thalassemia/Hb E Patients
The purpose of this study is to determine the efficacy of antioxidant cocktails on the alleviation of oxidative stress and iron overload in beta-thalassemia/Hb E patients.
- Trial name
- NCT01597765
- Testing
- curcuminoids, N-acetylcysteine, deferiprone and vitamin E, Curcuminoids and alpha-tocopherol
- Where
- Thailand
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Mahidol University
Thalassemia Major
Amlodipine Use in the Prevention and Treatment of Iron Overload in Patients With Thalassemia Major
This study aims to investigate the use of amlodipine, a drug that blocks the uptake of calcium into cells, in the prevention and treatment of iron overload in patients with thalassemia major.
- Trial name
- AnloThal (NCT01125254)
- Testing
- Amlodipine
- Where
- Brazil
- Volunteers
- 10 people
- Sponsor
- University of Campinas, Brazil