NCT03279094

Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

A Pilot Study of Pre-transplant Immunosuppressive Therapy for Haploidentical Transplants in Patients With Sickle Cell Disease

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: City of Hope Medical Center · Academic / Other
• Sex: All
• Age: 1 Year – 30 Years
• Healthy volunteers: Not accepted

Summary

This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.

Detailed description

All patients will receive an haploidentical hematopoietic stem cell transplant with the following conditioning and GvHD prevention: Pre-transplant immunosuppressive therapy: 2 cycles of Fludarabine and Dexamethasone x 5 days each cycle Conditioning regimen: rATG daily x 3 days, Fludarabine daily x 6 days and Busulfan daily x 4 days GVHD prophylaxis: Cyclophosphamide day +3 and +4, Tacrolimus and Mycophenolate mofetil

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
BIOLOGICAL	Hematopoietic stem cell transplantation	Haploidentical stem cell transplantation with pre-transplant immunosuppressive therapy

Timeline

• Start date: 2018-02-02
• Primary completion: 2026-02-24
• Completion: 2026-02-24
• First posted: 2017-09-12
• Last updated: 2025-06-22

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study