Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

Sickle Cell Disease Clinical Trial

Official title: Closely Matched Unrelated Donor Peripheral Blood Stem Cell Transplantation With TCRαβ+ T Cell and B Cell Depletion For Patients With Sickle Cell Disease and Thalassemia Major

Status Active, not recruiting

Clinical Trial Summary

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).

Clinical Trial Description

This is a single arm pilot study of peripheral stem cell transplantation (PSCT) with ex vivo TCRαβ+ T cell and CD19+ B cell depletion of URD grafts using the CliniMACS device in patients with SCD and BTM. Apart from CliniMACS-based cell processing, PSCT will be performed according to current standards of care in the Children's Hospital of Philadelphia (CHOP) Cell Therapy and Transplant Section, including the use of a standard chemotherapy conditioning regimen and standard follow-up laboratory assessments. The study will determine efficacy of this strategy in terms of engraftment, rates of acute and chronic Graft versus Host Disease (GvHD), and one-year overall and event-free survival. ;

Related Conditions & MeSH terms

• Anemia, Sickle Cell
• beta-Thalassemia
• Sickle Cell Disease
• Thalassemia
• Thalassemia Major

• NCT number: NCT04523376
• Study type: Interventional
• Source: Children's Hospital of Philadelphia
• Status: Active, not recruiting
• Phase: N/A
• Start date: May 14, 2020
• Completion date: December 1, 2024