NCT04523376

Pilot Study PBSCT with TCRab Depletion for Hemoglobinopathies

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
• Phase: N/A
• Study type: Interventional
• Enrollment: 9 (actual)
• Sponsor: Timothy Olson · Academic / Other
• Sex: All
• Age: 2 Years – 25 Years
• Healthy volunteers: Not accepted

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).

Detailed 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.

Conditions

• Sickle Cell Disease
• Thalassemia Major

Interventions

Type	Name	Description
DEVICE	CliniMACS	Peripheral blood stem cells from closely matched unrelated donors will be processed using the CliniMACS device to remove TCRalpha/beta T cells and B cells, in accordance with the Investigator Brochure and Technical Manual following the laboratory standard operating procedures (SOPs) and using aseptic technique

Timeline

• Start date: 2020-05-14
• Primary completion: 2026-06-01
• Completion: 2027-06-01
• First posted: 2020-08-21
• Last updated: 2025-02-25

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated device study