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Beta-Thalassemia clinical trials

View clinical trials related to Beta-Thalassemia.

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NCT ID: NCT06024876 Active, not recruiting - Beta-Thalassemia Clinical Trials

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

Start date: August 26, 2023
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: NCT05752123 Active, not recruiting - Clinical trials for Transfusion Dependent Beta-Thalassaemia

A Study to Evaluate the Safety and Efficacy of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia.

Start date: February 18, 2023
Phase: N/A
Study type: Interventional

This is an open label, single site study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.

NCT ID: NCT04925206 Active, not recruiting - Clinical trials for Transfusion Dependent Beta-Thalassaemia

A Safety and Efficacy Study Evaluating ET-01 in Subjects With Transfusion Dependent β-Thalassaemia

ET-01
Start date: August 17, 2021
Phase: Phase 1
Study type: Interventional

This is a single-arm, open label, multi-center, single-dose phase 1 study in subjects with transfusion dependent β-thalassaemia. The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using ET-01.

NCT ID: NCT04770779 Active, not recruiting - Clinical trials for 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)

ENERGIZE-T
Start date: November 30, 2021
Phase: Phase 3
Study type: Interventional

The primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-dependent alpha- or beta-thalassemia (TDT).

NCT ID: NCT04770753 Active, not recruiting - Clinical trials for Non-Transfusion-dependent Beta-Thalassemia

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

ENERGIZE
Start date: November 8, 2021
Phase: Phase 3
Study type: Interventional

The primary purpose of this study is to compare the effect of mitapivat versus placebo on anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).

NCT ID: NCT04523376 Active, not recruiting - Sickle Cell Disease Clinical Trials

Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

Start date: May 14, 2020
Phase: N/A
Study type: Interventional

This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo t-cell receptor alpha beta+(TCRαβ+) T cell and cluster of differentiation 19+ beta (CD19+ B) cell depletion of unrelated donor (URD) grafts using the CliniMACS device in patients with sickle cell disease (SCD) and beta thalassemia major (BTM).

NCT ID: NCT04390971 Active, not recruiting - Clinical trials for Transfusion Dependent Beta-Thalassaemia

Safety and Efficacy Evaluation of ET-01 Transplantation in Subjects With Transfusion Dependent β-Thalassaemia

Start date: February 10, 2023
Phase: N/A
Study type: Interventional

This is an open label, multi-center study to evaluate the safety and Efficacy of ET-01 Transplantation in subjects with Transfusion Dependent β-Thalassaemia.

NCT ID: NCT03655678 Active, not recruiting - Clinical trials for Hematologic Diseases

A Safety and Efficacy Study Evaluating CTX001 in Subjects With Transfusion-Dependent β-Thalassemia

Start date: September 14, 2018
Phase: Phase 2/Phase 3
Study type: Interventional

This is a single-arm, open-label, multi-site, single-dose Phase 1/2/3 study in subjects with transfusion-dependent β-thalassemia (TDT). The study will evaluate the safety and efficacy of autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) using CTX001.

NCT ID: NCT03275051 Active, not recruiting - Beta Thalassaemia Clinical Trials

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)

Start date: October 4, 2017
Phase: N/A
Study type: Interventional

OTL-300 is a gene therapy drug product consisting of autologous hematopoietic stem/progenitor cluster of differentiation (CD) 34+ cells genetically modified with a lentiviral vector (GLOBE) encoding the human beta globin gene. The TIGET-BTHAL is a phase I/II study evaluating safety and efficacy of OTL-300 in subjects with transfusion dependent beta-thalassemia for two years post gene-therapy. Subjects with rare disease who have undergone gene therapy are followed for efficacy and possible delayed adverse events. Thus, this study is designed to follow patients who have received gene therapy on TIGET-BTHAL for an additional six years (for a total of eight years).

NCT ID: NCT03101423 Active, not recruiting - Clinical trials for Beta Thalassemia Major

Monitoring of Chimerism After Transplantation in Patients With β Thalassemia Major and the Treatment Strategies for the Reduction of Chimerism

Start date: August 1, 2016
Phase: N/A
Study type: Interventional

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. chimerism is a key indicator of the succession of immune response, which is a key indicator for predicting the failure of hematopoietic stem cell transplantation and provides an important basis for early detection of rejection. Transplantation of continuous chimerism can detect early unstable chimeras and rejection.The chimerism rates after transplantation were continuously monitored using fluorescence labeled multiplex PCR amplification of short tandem repeats (STR-PCR) ,and then follow our STR different rates for early interventional therapy to prevent further reduction in chimerism leading to lead to graft failure.