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Thalassemia, Beta clinical trials

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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: NCT06041620 Recruiting - Thalassemia Major Clinical Trials

Safety and Efficacy Evaluation of Autologous CRISPR-Cas12b Edited Hematopoietic Stem Cells

Start date: August 31, 2023
Phase: N/A
Study type: Interventional

This is a single-arm, open, single-injection exploratory clinical study with two transfusion-dependent β thalassemia (β-TDT) participants planned to enroll.

NCT ID: NCT05799118 Recruiting - Sickle Cell Disease Clinical Trials

Study of the Role of Genetic Modifiers in Hemoglobinopathies

INHERENT
Start date: October 1, 2022
Phase:
Study type: Observational

This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).

NCT ID: NCT05736419 Recruiting - Sickle Cell Disease Clinical Trials

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

Start date: February 9, 2023
Phase: Phase 2
Study type: Interventional

Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.

NCT ID: NCT03183375 Recruiting - Thalassemia, Beta Clinical Trials

The Efficacy and Safety of HYDROXYUREA in Management of Beta Thalassemia Patients in Karachi Pakistan

Start date: August 21, 2017
Phase: Phase 2
Study type: Interventional

Objectives Primary objectives: - To determine the efficacy of Hydroxyurea in the study participants. - Hypothesis: The study will result in either maintenance or rise in hemoglobin as compared to the control treatment. Secondary objectives: - To determine the compliance of Hydroxyurea in study participants. - To determine the safety of Hydroxyurea in the study participants. Design and Outcomes An open label randomized controlled trial to test the efficacy and safety of Hydroxyurea on beta thalassemia major patients. It is a six months study. Findings of physical examination, vital sign variables, laboratory variables and ultrasound at baseline, during and end of the study will be listed. Schedule of intervention is mentioned in section 6.1. later in the protocol. Interventions and Duration Hydroxyurea will be given to the participants in intervention arm along with the standard treatment if thalassemia (blood transfusion and iron chelation therapy) and the control arm will receive the standard treatment (blood transfusion and iron chelation therapy) only. Each participant will be followed up for 6 months after initiating the intervention. Intervention will be given for 6 months or until the participant withdraws from the study or due to any reason, the investigator stops the intervention. Sample Size and Population This pilot study will be done on 100 patients initially. Stratified randomization will be done on the basis of presence of Xmn polymorphism. And the study population will be assigned to intervention or control arm randomly through a computer software (randomizer.org).