Clinical trials: Thalassaemia
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
Thalassemia, Brain Function (Measured by ERP Level)
A Case Control Study to Evaluate the Cognitive and Brain Function of β-thalassemia Patients.
β-thalassemia syndromes are a group of hereditary disorders characterized by a genetic deficiency in the synthesis of beta-globin chains.
- Trial name
- NCT01935661
- Where
- Israel
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- HaEmek Medical Center, Israel
Beta Thalassemia Major
Monitoring of Chimerism After Transplantation in Patients With β Thalassemia Major and the Treatment Strategies for the Reduction of Chimerism
Hematopoietic stem cell transplantation is currently the only way to cure thalassemia, one of its main obstacles is the rejection after transplantation, chimerism continued to decline, which eventually lead to transplant failure.
- Trial name
- NCT03101423
- Testing
- Interleukin-2, Donor Regulatory T-Lymphocytes
- Where
- China
- Volunteers
- 30 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Non Malignant Disorders, Immunodeficiencies
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients < 21…
- Conditions
- Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies
- Trial name
- NCT00744692
- Testing
- Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Duke University
Bone Marrow Failure Syndrome, Thalassemia
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
In this study, the investigators test 2 dose levels of thiotepa (5 mg/kg and 10 mg/kg) added to the backbone of targeted reduced dose IV busulfan, fludarabine and rabbit anti-thymocyte globulin (rATG) to determine the minimum…
- Conditions
- Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia
- Trial name
- NCT03513328
- Testing
- Thiotepa--single daily dose, Thiotepa--escalated dose
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- University of Florida
Thalassemia Major
Study to Evaluate Efficacy and Safety of S303 Treated Red Blood Cells (RBCs)in Subjects With Thalassemia Major Requiring Chronic RBC Transfusion
To evaluate the efficacy and safety of S 303 treated red blood cells (RBCs) in subjects who require chronic transfusion support due to thalassemia major.
- Trial name
- NCT01740531
- Testing
- S-303 Treated Red Blood Cells (RBCs), Conventional, untreated Red Blood Cells
- Where
- 3 sites in Italy, Turkey (Türkiye)
- Volunteers
- 86 people
- Sponsor
- Cerus Corporation
Beta Thalassemia Major Anemia, Iron Overload
Safety and Efficacy of Early Treatment With Deferiprone in Infants and Young Children
This study is looking at the effects of giving early treatment of deferiprone to young children with beta thalassemia who have started receiving regular blood transfusions but have not yet reached the criteria for starting on…
- Trial name
- START (NCT03591575)
- Testing
- Deferiprone oral solution
- Where
- 4 sites in Egypt, Indonesia
- Volunteers
- 64 people
- Sponsor
- Chiesi Canada Corp
Thalassemia
Safety and Efficacy of Hemoglobin F Inducers in Patients With Beta Thalassemia
The aim of this study is to determine the safety and therapeutic effect of HbF inducers (combination therapy: thalidomide and hydroxyurea) on beta thalassemia patients.
- Trial name
- NCT07673302
- Testing
- Thalidomide, Hydroxy Urea
- Where
- Pakistan
- Volunteers
- 240 people
- Sponsor
- Riphah International University
Beta-thalassemia Major, Hemosiderosis
Extension Study of the Efficacy and Safety of Deferasirox Treatment in Beta-thalassemia Patients With Transfusional Hemosiderosis (Study Amended to 2-year Duration)
To allow patients treated with deferasirox in the core study to continue iron chelation therapy for 2 years or until the drug became locally commercially available.
- Conditions
- Beta-thalassemia Major, Hemosiderosis, Iron Overload, Rare Anemia
- Trial name
- ESCALATOR (NCT00171301)
- Testing
- Deferasirox
- Where
- 5 sites in Egypt, Lebanon and 3 more
- Volunteers
- 233 people
- Sponsor
- Novartis Pharmaceuticals
β-thalassemia Major
A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants
This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major.
- Trial name
- NCT01745120
- Testing
- LentiGlobin BB305 Drug Product
- Where
- 6 sites in United States, Australia and 1 more
- Volunteers
- 19 people
- Sponsor
- Genetix Biotherapeutics Inc.
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)
β-thalassemia Major
β-globin Restored Autologous HSC in β-thalassemia Major Patients
This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.
- Trial name
- NCT04205435
- Testing
- β-globin restored autologous HSC
- Where
- China
- Volunteers
- 2 people
- Sponsor
- Bioray Laboratories
Thalassemia
Short-term Clinical Study of CN128 in Thalassemia Patients
1. Primary objectives: To study the tolerance and safety of multiple oral administration of CN128 in patients with thalassemia aged 16 years and above.
- Trial name
- NCT03935633
- Testing
- CN128 Tablets
- Where
- China
- Volunteers
- 16 people
- Sponsor
- Hangzhou Zede Pharma-Tech Co., Ltd.
Thalassemia Trait, Thalassemia; Mixed
Thalassemia Screening by Third Generation Sequencing
Test globin gene in participants
- Trial name
- NCT05552690
- Testing
- blood draw
- Where
- Thailand
- Volunteers
- 20 people
- Sponsor
- Chiang Mai University
Sickle Cell Disease, Thalassemia
Use of Mobile Technology for Intensive Training in Medication Management
Purpose: Assess whether intensive training with education and daily remote monitoring with provider involvement has a lasting positive impact on adherence to medication management.
- Trial name
- NCT02133560
- Testing
- Video recording, Medication Administration Log, Education
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Duke University
Thalassemia
Study of Effects of YisuiShengxueGranules on Thalassemia
The primary objectives of this study are to evaluate whether Yisui Shengxue Granules therapy can increase the hemoglobin level in peripheral blood and alleviate the symptoms, and at the same time, evaluate its safety in the…
- Trial name
- thalessemia (NCT01549080)
- Testing
- Yisuishengxue Granule
- Volunteers
- 2 people, healthy volunteers welcome
- Sponsor
- Guang'anmen Hospital of China Academy of Chinese Medical Sciences
Sickle Cell Disease, Thalassemia
Allo-HCT MUD for Non-malignant Red Blood Cell (RBC) Disorders: Sickle Cell, Thal, and DBA: Reduced Intensity Conditioning, Co-tx MSCs
The main purpose of this project is to cure patients with high risk Sickle cell disease and other red cell disorders including thalassemia and diamond-blackfan anemia by bone marrow transplantation.
- Conditions
- Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
- Trial name
- NCT00957931
- Testing
- Bone marrow transplantation, Mesenchymal Stromal Cells
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Stanford University
Beta-Thalassemia
Gene Therapy for Transfusion Dependent Beta-thalassemia
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment of patients affected by…
- Trial name
- TIGET-BTHAL (NCT02453477)
- Testing
- Autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- IRCCS San Raffaele
Vitamin c, Thalassemia Major
Role of Vitamin C to Augment Iron Chelation With DFP or DFX
role of Vit C to Augment iron chelation with DFP or DFX in thalassemic patients.
- Conditions
- Vitamin c, Thalassemia Major, Iron Chelation
- Trial name
- NCT02083575
- Testing
- Vitamin C, Defriprone, deferisarox, deferiprone , deferesarox
- Where
- Egypt
- Volunteers
- 50 people
- Sponsor
- Ain Shams University
Thalassemia
Demographic, Clinical, Laboratory and Genetical Characteristics of Patients With Beta Thalassemia Intermedia
The definition of Thalassemia Intermedia is not generally accepted and the specific clinical and laboratory characteristics varies between patients.
- Trial name
- NCT01443312
- Testing
- Laboratory analysis.
- Where
- Israel
- Volunteers
- 28 people
- Sponsor
- HaEmek Medical Center, Israel