A Phase II Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

Study point of contact

Damiano Rondelli, MD
312 413-3547
[email protected]


1 United States site


16 to 60 Years


Phase 2

Study type










About the study

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.


Patient Eligibility:

1. Patients with sickle cell disease are eligible if they have any of the following

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 (≥ 2 antibodies) during
long-term transfusion therapy 1.6 Bilateral proliferative retinopathy with major
visual impairment in at least one eye 1.7 Osteonecrosis of 2 or more joints 1.8 Sickle
cell nephropathy, defined by a GFR < 90mL/min/1.73m2 or the presence of macroalbuminuria (urine albumin > 300 mg/g creatinine) 1.9 Pulmonary hypertension,
defined by a mean pulmonary arterypressure >25mmHg

2. Age 16-60 years

3. Karnofsky performance status of 60 or higher (Appendix A)

4. Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40%

5. Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥
50% predicted (after adjustment for hemoglobin concentration)

6. Estimated GFR ≥ 50mL/min/1.73m2 as calculated by the modified MDRD equation

7. ALT ≤ 3x upper limit of normal

8. HIV-negative

9. Patient is not pregnant

10. Patient is able and willing to sign informed consent

11. Patient does not have a fully HLA-matched sibling donor

12. Patient has an HLA-haploidentical relative

Donor Eligibility Relatives (parents, offspring, siblings, aunts/uncles, cousins) will be
tested by molecular typing of HLA class I (A, B, and C) and class II (DRB1) at low
resolution. Only those that are an HLA-haploidentical match (≥ 4/8) will be considered as a
potential donor. NOTE: If during testing, a fully HLA-matched sibling donor is found and is
willing to donate his/her stem cells, the potential subject will not be eligible for this

Donor consent will be obtained as per standard protocol of the bone marrow transplant unit.


  • Chicago, Illinois, United States, University of Illinois at Chicago, 60612 [Recruiting]