HLA-haploidentical stem cell transplantation for aggressive sickle cell disease
A Phase II Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
This study is testing a new type of stem cell transplant to see if it can help people with severe sickle cell disease feel better.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 16 Years to 60 Years |
| Sex | All |
| Sponsor | University of Illinois at Chicago Academic / other |
| Drugs / interventions | cyclophosphamide, fludarabine |
| Locations | 1 site (Chicago, Illinois) |
| Trial ID | NCT03121001 on ClinicalTrials.gov |
What this trial studies
This Phase II clinical trial investigates the use of HLA-haploidentical hematopoietic stem cell transplantation to treat patients with clinically aggressive sickle cell disease. Participants will undergo conditioning with intensity modulated total body irradiation and immunosuppressive agents before receiving stem cell infusions. The primary goal is to assess the engraftment success at Day +60 post-transplant. The study aims to provide a new treatment option for patients suffering from severe complications of sickle cell disease.
Who should consider this trial
Good fit: Ideal candidates are patients with sickle cell disease experiencing severe complications such as recurrent strokes, frequent pain episodes, or pulmonary hypertension.
Not a fit: Patients with mild sickle cell disease or those without significant complications may not benefit from this intervention.
Why it matters
Potential benefit: If successful, this approach could significantly improve the quality of life and health outcomes for patients with severe sickle cell disease.
How similar studies have performed: Other studies have shown promise with similar stem cell transplantation approaches, indicating potential for success in this area.
Eligibility criteria
Show full inclusion / exclusion criteria
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 (≥ 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 protocol. Donor consent will be obtained as per standard protocol of the bone marrow transplant unit.
Where this trial is running
Chicago, Illinois
- University of Illinois at Chicago — Chicago, Illinois, United States (Recruiting)
Study contacts
- Principal investigator: Damiano Rondelli, MD — University of Illinois at Chicago
- Study coordinator: Damiano Rondelli, MD
- Email: drond@uic.edu
- Phone: 312 413-3547
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.