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Beta-Thalassemia clinical trials

View clinical trials related to Beta-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: 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: 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.

NCT ID: NCT06239389 Completed - Beta-Thalassemia Clinical Trials

Comparison Of Efficacy And Safety Of Thalidomide Vs Hydroxyurea In Thalassemia Patients: A Single-Centre Pilot Study.

Start date: September 1, 2021
Phase: Phase 2
Study type: Interventional

The goal of this study is to compare the effectiveness and safety of two hemoglobin F inducer. This is single centered interventional pilot study is to compare the efficacy and safety parameters in beta thalassemia patients. As this is a pilot study, the investigator took a small number of patients. The Sample size was calculated by the World health organization sample size calculator. After screening 39 patients and 24 patients were eligible for enrollment in this study. The main objective was to evaluate safety of both drugs in genetic disorder like thalassemia. for safety evaluation, hematological parameters were evaluated that includes total bilirubin , indirect bilirubin, Serum Glutamate Pyruvate Transaminase, serum glutamic-oxaloacetic transaminase, urea, creatinine and lactate dehydrogenase were monitored . Moreover to evaluate the efficacy of drug, hematological parameters that includes hemoglobin, red blood cells , nucleated red blood cells , reticulocytes count, Red blood cells indices ( mean corpuscular hemoglobin, mean corpuscular volume and mean corpuscular hemoglobin concentration) , white blood cells and platelets were done. Another important parameters to evaluate the efficacy of hemoglobin F inducer is transfusion frequency. Test were done at baseline and after completion of study means after 06 months.

NCT ID: NCT06219239 Recruiting - Clinical trials for Transfusion-dependent Beta-Thalassemia

Safety and Efficacy of the Lentiviral Vector in Gene Therapy of Beta-thalassemia Patients

Start date: January 4, 2024
Phase: N/A
Study type: Interventional

This is a non-randomized, open-label, single-dose study. The aim of this study is to evaluate the safety and efficacy of the treatment with lentiviral vector encoding βA-T87Q-globin gene transduced autologous hematopoietic stem cells transfusion in subjects with β-thalassemia major.