A Phase II Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
University of Illinois at Chicago
Summary
The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v. cyclophosphamide conditioning prior to human leukocyte antigen (HLA)-haploidentical hematopoietic stem cell transplant (HSCT). The primary objective of the study is to determine the engraftment at Day +60 following HLA-haploidentical hematopoietic stem cell transplant protocol using immunosuppressive agents and low-dose total body irradiation (TBI) for conditioning and post-transplant cyclophosphamide in patients with sickle cell disease.
Eligibility
- Age range
- 16–60 years
- Sex
- All
- Healthy volunteers
- No
Patient Eligibility: 1. Patients with sickle cell disease are eligible if they have any of the following complications: 1.1 Stroke or central nervous system event lasting longer than 24 hours 1.2 Frequent vaso-occlusive pain episodes, defined as ≥ 3 per year requiring emergency room, acute care center, hospital admissions, or home bedrest leading to absence from work or school. 1.3 Recurrent episodes of priapism, defined as ≥ 2 per year requiring emergency room visits 1.4 Acute chest syndrome with recurrent hospitalizations, defined as ≥ 2 lifetime events 1.5 Red-cell alloimmunization (≥…
Interventions
- DrugATG
0.5 mg/kg IV on day -9, and 2 mg/kg on days -8 and day -7
- Drugfludarabine
30 mg/m2 IVPB daily for day -6 (6 days before stem cell infusion) through day -2
- Drugcyclophosphamide
14.5 mg/kg IV on days -6 and -5 and 50 mg/kg/d on days +3 and +4
- RadiationTotal body irradiation
3 Gy on day -1
- ProcedureStem cell infusion
Stem cell product infused according to BMT unit policy on day 0.
- DrugSirolimus
loading dose of 15 mg followed by 5 mg per day on day +5
- Drugmycophenolate mofetil
Location
- University of Illinois at ChicagoChicago, Illinois