Clinical trials: B Thalassemia
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)
The primary objective of this study was to compare the effect of mitapivat versus placebo on transfusion burden in participants with α- or β-transfusion-dependent thalassemia.
- Trial name
- ENERGIZE-T (NCT04770779)
- Testing
- Mitapivat
- Where
- 75 sites in United States, Brazil and 17 more
- Volunteers
- 258 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
AL-Hijama in Thalassmia Major
Study of The Therapeutic Benefits of Al-hijamah in Children With Beta Thalassemia Major
1. Evaluate the therapeutic effects, health benefits, and immunological effect of Al-hijamah in children with beta thalassemia major. 2.
- Trial name
- Al-hijamah (NCT02761395)
- Testing
- Al-hijamah, Deferasirox
- Where
- 2 sites in Egypt
- Volunteers
- 60 people
- Sponsor
- Tanta University
Thalassemia, Splenectomy; Status
Zinc Supplementation on Cellular Immunity in Thalassemia Major
Randomized controlled trial was conducted in post-splenectomy patients aged >12 years. Subjects are randomly assigned to two groups (zinc and placebo). 1.5 mg/kg/day (max 50 mg/day) of Zinc is administered.
- Conditions
- Thalassemia, Splenectomy; Status, Immunosuppression
- Trial name
- NCT03117192
- Testing
- Zinc Sulfate, Sucrose Syrup
- Where
- Indonesia
- Volunteers
- 58 people
- Sponsor
- Fakultas Kedokteran Universitas Indonesia
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
Beta-Thalassemia
Gene Therapy of Beta Thalassemia Using a Self-inactivating Lentiviral Vector
This is a Phase I/II clinical trial of gene transfer for treating Beta-thalassemia using a self-inactivating lentiviral vector to functionally correct the defective gene(s).
- Trial name
- NCT03351829
- Testing
- Gene-modified autologous hematopoeitic stem cells
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
B Thalassemia
Assessment of Cognitive Functions, Psychological Symptoms, and Quality of Life Among B Thalassemia Patients and Their Clinical Correlates.
Multiple studies showed the negative impact thalassemia disease and its therapy have on the cognition of these patients. This study aims to assess and compare the cognition, psychological impact and quality of life among patients…
- Trial name
- NCT05105659
- Testing
- Wechsler IQ for Adults, MAS (Memory assessment scale), Health-Related Quality of Life (HRQoL)
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Assiut University
Iron Overload Due to Repeated Red Blood Cell Transfusions, Thalassemia Majors (Beta-Thalassemia Major)
Evaluate the Efficacy and Safety of Irochel for the Management of Iron Overload in Thalassemia Patients in Comparison With Deferoxamine
This research study was an experimental, randomized, controlled clinical trial to evaluate the safety and efficacy of coded Unani formulation Irochel for the management of IOL due to repetitive blood transfusion in comparison…
- Trial name
- NCT07782931
- Testing
- Irochel, Deferoxamine
- Where
- Pakistan
- Volunteers
- 132 people, healthy volunteers welcome
- Sponsor
- Hamdard University
Sickle Cell Disease, Thalassemia
Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure
Background: Some sickle cell disease or beta-thalassemia can be cured with transplant. Researchers want to test a variation of transplant that uses low dose radiation and a combination of immunosuppressive drugs.
- Conditions
- Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
- Trial name
- NCT02105766
- Testing
- Alemtuzumab, Sirolimus, Cyclophosphamide
- Where
- United States
- Volunteers
- 56 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Beta Thalassemia Major
Anthropometric Measurements in Children Having Transfusion-dependent Beta Thalassemia
The aim of this study was to determine the anthropometric measurements in transfusion-dependent β-thalassemia children in Pakistan. The secondary aim was to correlate serum ferritin with the physical growth
- Trial name
- NCT03947632
- Testing
- serum ferritin and hemoglobin test
- Volunteers
- 367 people
- Sponsor
- Aga Khan University
Thalassemia, Beta
Thalidomide and Hydroxyurea Combination in β-Thalassemia Patients
Objectives Primary objective: To determine the efficacy and safety of the combination therapy of Hydroxyurea and thalidomide in beta-thalassemia patients.
- Trial name
- NCT06153784
- Testing
- Hydroxyurea and Thalidomide
- Where
- Pakistan
- Volunteers
- 603 people
- Sponsor
- Children's Hospital Karachi
β-thalassemia Intermedia
Apotransferrin in Patients With β-thalassemia
The aim of the trial is to study the effect of apotransferrin administration in patients suffering from β-thalassemia intermedia in order to restore the erythropoiesis as reflected by enhanced haemoglobin levels or reduced…
- Trial name
- NCT03993613
- Testing
- human apotransferrin
- Where
- Netherlands
- Volunteers
- 10 people
- Sponsor
- Prothya Biosolutions
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
Neutropenia, Sickle Cell Anemia
Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders
OBJECTIVES: I. Determine the efficacy of bone marrow transplantation using matched related donors in patients with nonmalignant hematologic disorders. II.
- Conditions
- Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
- Trial name
- NCT00005893
- Testing
- anti-thymocyte globulin, busulfan, cyclophosphamide
- Where
- United States
- Sponsor
- Fairview University Medical Center
Beta-Thalassemia Major, Graft-versus-host Disease (GVHD)
Efficacy of Basiliximab in the Prevention of Acute Graft-versus-host Disease in Unrelated Allogeneic Hematopoietic Stem Cell Transplantation Therapy for Thalassemia Major
The purpose of this study is to evaluate the basiliximab for prevention of graft-versus-host disease in unrelated allo-genetic hematopoietic stem cell transplantation for thalassemia major.
- Trial name
- NCT02342145
- Testing
- Basiliximab,, Tacrolimus, Methotrexate
- Where
- 3 sites in China
- Volunteers
- 205 people
- Sponsor
- Zhongming Zhang
Beta-thalassemia, Glioma
Plasticizer Exposure and Its Consequences on Health
Plasticizers are chemicals commonly found in many everyday items, from food packaging to medical equipment. Although they are pervasive in our daily lives, researchers still don't have a clear picture of their long-term effects…
- Conditions
- Beta-thalassemia, Glioma, Healthy Controls, Transfusion-dependent Beta-Thalassemia
- Trial name
- PEACH (NCT06512298)
- Where
- Austria
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Medical University of Vienna
Sickle Cell Anemia, Sickle - Beta Thalassemia
Hepcidin Levels in Sickle Cell Disease (SCD)
The investigators propose that patients with HbSβ-thalassemia have lower levels of hepcidin and higher levels of GDF-15 than HbSS patients during the non-crisis, "steady states." In addition, the investigators propose that when…
- Trial name
- NCT02258997
- Volunteers
- 42 people
- Sponsor
- Kenneth Ataga, MD
β-thalassemia, Ineffective Erythropoiesis
Safety Study for Beta Thalassemia Subjects on PTG-300
In this study will investigate long term safety and use of the PTG-300 in Beta Thalassemia patients.
- Trial name
- TRANSCEND (NCT04054921)
- Testing
- PTG-300
- Where
- 16 sites in United States, Greece and 6 more
- Volunteers
- 34 people
- Sponsor
- Protagonist Therapeutics, Inc.
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
Thalassemia, Iron Overload
Amlodipine 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
- AmloThal (NCT01395199)
- Testing
- Amlodipine
- Where
- Brazil
- Volunteers
- 62 people
- Sponsor
- University of Campinas, Brazil