Half-matched donor stem cell transplant in people with sickle cell disease
Tests half-matched donor stem cell transplants in sickle cell disease.
Plain-language title generated from the registry record
Registry title: Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
Key facts
- Where
- Chicago, United StatesSee the site
- Phase
- Phase 2
AI summary: who the study is looking for
From the trial's eligibility criteria. Machine-read from the registry text on this page, so it can be wrong. The registry text below and the trial team decide who can take part.
The trial is looking for people aged 16 to 60 with sickle cell disease who have had serious complications and have a half-matched relative who can donate stem cells.
- Age
- 16 to 60 years
- Sex
- All sexes can join
- Disease stage
- Sickle cell disease with at least one serious complication, such as a stroke or nervous system event lasting longer than 24 hours, frequent painful crises (3 or more per year), repeated priapism (2 or more per year), repeated acute chest syndrome (2 or more events), 2 or more red-cell antibodies during long-term transfusions, serious eye damage, bone death in 2 or more joints, kidney damage or pulmonary hypertension
- Biomarker or test result
- Has a half-matched (HLA-haploidentical) relative as donor. No fully HLA-matched sibling donor. Karnofsky performance status of 60 or higher.
Main reasons for not taking part
- Has a fully HLA-matched sibling donor
- Heart pumping function (left ventricular ejection fraction) below 40%
- Lung diffusion capacity below 50% of predicted
- Estimated kidney filtration (GFR) below 50mL/min/1.73m2
- HIV-positive or pregnant
Read by machine on 12 November 2026.
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 16 and 60 years old?
Source: NCT03121001, who can take part
Where in United States
- University of Illinois at ChicagoChicago, United StatesContact: Damiano Rondelli, MDTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Contact the study team: see the registry record → Contacts and Locations
Print a page for my doctorTrial details
- Who runs it
- University of Illinois Chicago
- Trial code
- NCT03121001
- Record updated
- 8 July 2026
- Original description
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).Read full original descriptionShow less
A Phase II Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
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:; 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 (≥ 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; Age 16-60 years; Karnofsky performance status of 60 or higher (Appendix A); Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40%; Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥ 50% predicted (after adjustment for hemoglobin concentration); Estimated GFR ≥ 50mL/min/1.73m2 as calculated by the modified MDRD equation; ALT ≤ 3x upper limit of normal; HIV-negative; Patient is not pregnant; Patient is able and willing to sign informed consent; Patient does not have a fully HLA-matched sibling donor; 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 protocol; Donor consent will be obtained as per standard protocol of the bone marrow transplant unit.