ß-thalassemia Major Clinical Trials

NCT ID: NCT05776173 Recruiting - ß-thalassemia Major Clinical Trials

Safety and Efficacy of Gene Modified Autologous Hematopoietic Stem Cells to Treat Transfusion-dependent β-thalassemia

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

This study will be intented to evaluate the safety, tolerability, and engraftment efficacy after myeloablative preconditioning and transplantation of autologous CD34+ hematopoietic stem cells transduced with a lentiviral vector encoding the human βA-T87Q-globin gene in patients with transfusion-dependent (TDT) β-thalassemia.

NCT ID: NCT04592458 Not yet recruiting - ß-Thalassemia Major Clinical Trials

Safety and Efficacy Evaluation of β-globin Restored Autologous Hematopoietic Stem Cells in β-thalassemia Major Patients

Start date: November 1, 2020
Phase: Phase 1
Study type: Interventional

This is an open label study to evaluate the safety and efficacy of β-globin Restored Autologous Hematopoietic Stem Cells in ß-Thalassemia Major Patients

NCT ID: NCT04205435 Terminated - ß-thalassemia Major Clinical Trials

β-globin Restored Autologous HSC in β-thalassemia Major Patients

Start date: November 1, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

This is a single center, single arm, open-label study to determine the safety and efficacy of β-globin restored autologous hematopoietic stem cells in β- thalassemia major patients with CVS-654 mutation.

NCT ID: NCT01846923 Active, not recruiting - ß-thalassemia Major Clinical Trials

B Memory Cell Response to Vaccination With the 13-valent Pneumococcal Conjugate Vaccine in Asplenic Individuals

Start date: October 2010
Phase: Phase 4
Study type: Interventional

The purpose of this study is to determine whether one dose of the 13-valent pneumococcal conjugate vaccine (PCV13) induces immunological memory in asplenic adults and whether previously administered immunizations with the 23-valent polysaccharide pneumococcal vaccine influence the cellular immune response to PCV13 in this group.

NCT ID: NCT01745120 Completed - ß-thalassemia Major Clinical Trials

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

Start date: August 2013
Phase: Phase 1/Phase 2
Study type: Interventional

This is a non-randomized, open label, multi-site, single-dose, phase 1/2 study in up to 18 participants (including at least 3 adolescents between 12 and 17 years of age, inclusive) with β-thalassemia major. The study will evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin BB305 Drug Product [autologous CD34+ hematopoietic stem cells transduced with LentiGlobin BB305 lentiviral vector encoding the human βA-T87Q-globin gene].

NCT ID: NCT01724138 Withdrawn - ß-thalassemia Major Clinical Trials

An Open Label Study to Evaluate the Pharmacokinetics, Safety, Tolerability and Efficacy of Deferasirox Administered to Chinese Patients With β-thalassemia Major Aged From 2 to Less Than 6 Years Old

MACS2163

Start date: June 2013
Phase: Phase 4
Study type: Interventional

To characterize the PK of deferasirox in pediatric β-thalassemia major patients aged from 2 to less than 6 years old, when administrated with a fixed starting dose of 20 mg/kg/day.

NCT ID: NCT00390858 Completed - Clinical trials for Transfusional Iron Overload

A 4-year Extension Study to Core 1-year Study of Iron Chelation Therapy With Deferasirox in β-thalassemia Major Pediatric Patients With Transfusional Iron Overload.

Start date: September 2003
Phase: Phase 2
Study type: Interventional

In this 4-year extension study the safety, efficacy and and pharmacokinetics of deferasirox in regularly transfused pediatric patients with β-thalassemia major was assessed. Patients who successfully completed the main 1 year trial (NCT00390858) were eligible to continue in this extension trial and receive chelation therapy with deferasirox for up to 4 years.

ß-thalassemia Major clinical trials