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Thalassemia clinical trials

View clinical trials related to 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: NCT05552690 Active, not recruiting - Thalassemia; Mixed Clinical Trials

Thalassemia Screening by Third Generation Sequencing

Start date: February 1, 2022
Phase: N/A
Study type: Interventional

Test globin gene in participants

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: NCT04691323 Active, not recruiting - Sickle Cell Disease Clinical Trials

The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.

Start date: March 2, 2021
Phase: N/A
Study type: Interventional

The primary objective of this study is to better understand factors contributing to variations in hydroxyurea (HU) adherence behavior in adolescents and young adults (AYA) with sickle cell disease (SCD). To meet this objective, the researchers will conduct a prospective cohort study to determine the longitudinal relationship between HU adherence and health-related quality of life (HRQOL) overtime among AYA with SCD. The long-term goal of this research is to promote medication adherence behavior and improve health outcomes in AYA with SCD.

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: NCT03692052 Active, not recruiting - Thalassemia Clinical Trials

A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia

Start date: March 20, 2019
Phase: Phase 2
Study type: Interventional

Study AG348-C-010 is a multicenter study to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of treatment with AG-348 in adult participants with non-transfusion-dependent thalassemia (NTDT). This study includes a core period (up to 24 weeks) followed by an extension period (up to 10 years) for eligible participants. 20 participants with NTDT were enrolled. The initial dose of AG-348 was 50 milligrams (mg) twice daily (BID) with one potential dose-level increase to 100 mg BID at the Week 6 visit based on the participant's safety and hemoglobin (Hb) concentrations.