Trial Summary
What is the purpose of this trial?The investigators propose to determine the engraftment and transplant related morbidity and mortality after a non-myeloablative allogeneic hematopoietic stem cell transplant protocol using immune- suppressive agents and low-dose total body irradiation (TBI) without standard chemotherapy in patients with aggressive sickle cell disease who are not candidates for or experienced complications from hydroxyurea therapy.
Fully HLA matched siblings will be used as donors for hematopoietic stem cells to reduce the risk of morbidity and mortality in this cohort of patients.
Eligibility Criteria
This trial is for individuals aged 16-60 with aggressive sickle cell disease who haven't responded well to or can't take hydroxyurea. Candidates must have a fully matched sibling donor, good heart and lung function, no HIV or chronic hepatitis, and be willing to sign consent. They should have had complications like frequent pain episodes, stroke, vision problems due to retinopathy, kidney issues, or severe anemia requiring transfusions.Inclusion Criteria
Patients with sickle cell disease, subtype Hgb SS, SC, or SB disease who are on chronic transfusion therapy for a prior stroke or those patients who were intolerant of hydroxyurea therapy or were being treated with hydroxyurea therapy and were complicated by at least one of the following: Stroke or central nervous system event lasting longer than 24 hours, Frequent vaso-occlusive pain episodes, Recurrent episodes of priapism, Acute chest syndrome with recurrent hospitalizations, Red-cell alloimmunization, Bilateral proliferative retinopathy with major visual impairment in at least one eye, Osteonecrosis of 2 or more joints, Sickle cell nephropathy, Stage I or II sickle lung disease, Symptoms of pulmonary hypertension and mean pulmonary artery pressure > 25mmHg, Age 16-60 years, Karnofsky performance status of 70 or higher, Adequate cardiac function, Adequate pulmonary function, Estimated GFR ≥ 30mL/min, ALT ≤ 3x upper limit of normal, No evidence of chronic active hepatitis or cirrhosis, HIV-negative, Patient is not pregnant, History of compliance with medications and medical care, Patient is able and willing to sign informed consent, Patient has an HLA-identical matched related donor
Participant Groups
The study tests a less harsh stem cell transplant from fully matched siblings using immune-suppressing drugs (Alemtuzumab and Sirolimus) and low-dose radiation instead of standard chemo. The goal is to see if this method helps patients accept the new cells with fewer complications.
1Treatment groups
Experimental Treatment
Group I: Allogeneic Non-Myeloablative Stem Cell TransplantationExperimental Treatment3 Interventions
The transplant regimen will consist of alemtuzumab 1mg/kg divided over five days, 300 cGy TBI, followed by sirolimus dosed for a target serum trough level of 10- 15 ng/mL.
Alemtuzumab is already approved in United States, European Union, European Union for the following indications:
🇺🇸 Approved in United States as Campath for:
- Chronic lymphocytic leukemia
- Multiple sclerosis
🇪🇺 Approved in European Union as Lemtrada for:
- Multiple sclerosis
🇪🇺 Approved in European Union as Campath for:
- Chronic lymphocytic leukemia
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
University of Illinois at ChicagoChicago, IL
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Who Is Running the Clinical Trial?
University of Illinois at ChicagoLead Sponsor