Actively Recruiting
Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
Led by University of Illinois at Chicago · Updated on 2026-01-26
50
Participants Needed
1
Research Sites
475 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of sickle cell disease with at least one of these complications: stroke or central nervous system event lasting over 24 hours; frequent vaso-occlusive pain episodes (three or more per year) requiring emergency or hospital care or home bedrest; recurrent priapism (two or more episodes per year) needing emergency visits; acute chest syndrome with two or more lifetime hospitalizations; red-cell alloimmunization with two or more antibodies; bilateral proliferative retinopathy causing major vision loss in one eye; osteonecrosis in two or more joints; sickle cell nephropathy with a GFR below 90 mL/min/1.73m2 or macroalbuminuria; pulmonary hypertension with mean pulmonary artery pressure over 25 mmHg
- Age between 16 and 60 years
- Karnofsky performance status of 60 or higher
- Adequate cardiac function with left ventricular ejection fraction of 40% or higher
- Adequate pulmonary function with diffusion lung capacity for carbon monoxide at least 50% predicted
- Estimated glomerular filtration rate (GFR) of 50 mL/min/1.73m2 or higher
- Alanine aminotransferase (ALT) level at or below three times the upper limit of normal
- HIV-negative status
- Not pregnant
- Able and willing to sign informed consent
- Does not have a fully HLA-matched sibling donor
- Has an HLA-haploidentical relative donor available
You will not qualify if you...
- Presence of a fully HLA-matched sibling donor willing to donate stem cells
- HIV-positive status
- Pregnancy
- Karnofsky performance status below 60
- Left ventricular ejection fraction less than 40%
- Diffusion lung capacity for carbon monoxide less than 50% predicted
- Estimated GFR below 50 mL/min/1.73m2
- Alanine aminotransferase (ALT) above three times the upper limit of normal
- Lack of an HLA-haploidentical relative donor
- Inability or unwillingness to provide informed consent
- Any serious medical conditions that would prevent safe participation in the transplant protocol or study procedures
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 1 location
1
University of Illinois at Chicago
Chicago, Illinois, United States, 60612
Actively Recruiting
Research Team
D
Damiano Rondelli, MD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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