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Thalassemia clinical trials

View clinical trials related to Thalassemia.

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NCT ID: NCT06328764 Not yet recruiting - Beta-Thalassemia Clinical Trials

A Clinical Trial Evaluating the Safety and Efficacy of CS-101 in Treating Subjects With β-thalassemia

Start date: March 20, 2024
Phase: Early Phase 1
Study type: Interventional

The goal of this open label, single-arm clinical study is to learn about the safety and efficacy of CS-101 in treating β-thalassemia.

NCT ID: NCT06314529 Recruiting - Thalassemia, Beta Clinical Trials

Long-term Follow-up Study of BHC001 for TDT

Start date: December 14, 2022
Phase:
Study type: Observational

Observe long-term safety risk and long-term efficacy after intravenous infusion of BHC001 in TDT subjects.

NCT ID: NCT06313398 Not yet recruiting - Sickle Cell Disease Clinical Trials

Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

Start date: March 31, 2024
Phase: Early Phase 1
Study type: Interventional

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. SCD and other blood disorders can be managed with drugs or cured with a bone marrow transplant. Researchers want to know how long RBCs survive in people with SCD and other blood disorders before and after treatment compared to those who had a bone marrow transplant. Objective: To learn how long RBCs survive in the body in people with SCD and other blood disorders compared to those whose disease was cured with a bone marrow transplant. Eligibility: People aged 18 years or older with SCD or another inherited blood disorder. People whose SCD or blood disorder was cured with a bone marrow transplant are also needed. Design: Participants will be screened. They will have a physical exam with blood and urine tests. Participants will have about 7 tablespoons of blood drawn. In the lab, this blood will be mixed with a vitamin called biotin. Biotin sticks to the outside of RBCs. This process is called "biotin labeling of RBCs." The next day, the participant s own biotin-labeled RBCs will be returned to their bloodstream. Participants will return regularly to have smaller blood samples (about 2 teaspoons) drawn. These samples will be tested to detect the percentage of cells that have biotin labels. These visits may be every 2 weeks, 4 weeks, or some other interval. Participants will continue this schedule for up to 20 weeks or until biotin can no longer be detected....

NCT ID: NCT06308159 Not yet recruiting - Beta-Thalassemia Clinical Trials

An Open-label Study of a Gene Therapy Product in Beta-Thalassemia

Start date: April 1, 2024
Phase: Phase 1/Phase 2
Study type: Interventional

This is an interventional study to evaluate the safety and efficacy of autologous Hematopoietic Stem Cells (HSCs) transduced with lentiviral vector encoding functional hemoglobin subunit beta (HBB) gene in patients with transfusion-dependent beta-thalassemia.

NCT ID: NCT06302491 Not yet recruiting - ß -Thalassemia Clinical Trials

A Study of Safety and Efficiency of AND017 in Patients With Transfusion Dependent and Non-transfusion Dependent β-thalassemia

Start date: May 2024
Phase: Phase 2
Study type: Interventional

This is a phase II, randomized, double-blinded, placebo-controlled study to treat patients with transfusion-dependent and non-transfusion dependent β -thalassemia with AND017 and optimal supportive care, including blood transfusion and iron removal, based on the clinician's judgment and practice.

NCT ID: NCT06299670 Recruiting - Clinical trials for Transfusion-dependent Thalassemia

Efficacy of Combination of Hdroxyurea and Thalidomide Over Either Hydroxyurea or Thalidomide Alone in the Treatment of Transfusion Dependent Thalassemia in Children: A Quasi-Randomised Clinical Trial

Start date: November 1, 2023
Phase: Phase 4
Study type: Interventional

Transfusion Dependent Thalassemia (TDT) is an emerging global public health concern. Hemopoietic stem cell transplantation (HSCT) is the only curative treatment. But its adoption is limited due to lack of HLA matched donor, experienced centers and high initial cost. So, researches are going on in search of an effective, safe, easily available treatment option. Use of fetal haemoglobin inducing agents shows promising effects in treatment of TDT patients. Thalidomide an immunomodulating and anti-angiogenic drug has been shown to induce γ-globin gene expression and increase the proliferation of erythroid cells. Furthermore Hydroxyurea (HU) is known to increase haemoglobin (Hb) by HbF induction and reduction of inflammation and hypercoagulability. Recent studies with combination of HU and Thalidomide have shown promising results in treatment of Thalassemia patients. However, most of those studies are retrospective or single arm nonrandomized trials & The study population includes both adult and children age group . So the effectiveness of combination therapy of Thalidomide and HU needs to be established through randomized trials. This single centered non blinded quasi randomized clinical trial will be conducted at the Department of Pediatric Hematology and Oncology in Bangabandhu Sheikh Mujib Medical University (BSMMU), Bangladesh for one year of period. Thirty transfusion dependent thalassemia children of 3-18 years old will be included in each group. The objective of this study is to assess the effectiveness of combination of Thalidomide and Hydroxyurea in TDT children. It will play an important role in planning a cost effective and affordable treatment option for TDT children.This study will involve minimum physical risk to the patient. Written informed consent will be taken from parents or study subjects after brief explanation of the purpose and procedure. They will also be informed about the freedom to participate or not to participate at any time. Privacy and confidentiality will be safe guarded. History regarding age, sex, height, weight of these patients will be taken. Thorough physical examinations and laboratory investigations including CBC, Hb electrophoresis, serum Ferritin, serum creatinine, SGPT will be done. Data will be collected in a predesigned questionnaire and will be kept confidential. Statistical analysis will be done using the statistical software SPSS.

NCT ID: NCT06298630 Not yet recruiting - Thalassemia, Beta Clinical Trials

Long-term Follow-up Study of BRL-101 for TDT

Start date: December 8, 2024
Phase:
Study type: Observational

Observe long-term safety risk and long-term efficacy after intravenous infusion of BRL-101 in TDT subjects.

NCT ID: NCT06291961 Not yet recruiting - Clinical trials for Beta-Thalassemia Major

A Safety and Efficacy Study Evaluating CS-101 in Subjects With β-Thalassemia Major

Start date: March 2024
Phase: Phase 1
Study type: Interventional

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 anemia.

NCT ID: NCT06280378 Not yet recruiting - Clinical trials for Transfusion-dependent Beta-Thalassemia

A Phase I/II Clinical Study of the KL003 Cell Injection in β-Thalassemia Major Participants

Start date: February 2024
Phase: Phase 1/Phase 2
Study type: Interventional

This is a non-randomized, open label, single-dose study in up to 41 participants with β-thalassemia major. The goal of this clinical trial is to evaluate the safety and efficacy of KL003 cell injection in subjects with β-thalassemia major.

NCT ID: NCT06271512 Recruiting - Beta-Thalassemia Clinical Trials

A Study of Participants With β-Thalassemia Treated With Betibeglogene Autotemcel

Start date: January 23, 2024
Phase:
Study type: Observational [Patient Registry]

The main aim of this study is to collect real-world longitudinal data on participants with β-thalassemia treated with betibeglogene autotemcel (beti-cel) in the post marketing setting. To assess the long-term safety, including the risk of newly diagnosed malignancies, after treatment with beti-cel and evaluate the long-term effectiveness of treatment with beti-cel.