HLA-haploidentical stem cell transplantation for aggressive sickle cell disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• University of Illinois at Chicago
  Chicago, Illinois, United States Recruiting

Study contacts

• Principal investigator: Damiano Rondelli, MD — University of Illinois Chicago
• Study coordinator: Damiano Rondelli, MD
• Email: drond@uic.edu
• Phone: 312 413-3547

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Hematopoietic stem cell editing to treat sickle cell disease

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• Virtual reality options to help with VOC pain in adults with SCD

  This study tests whether headset-based virtual reality can help reduce pain for adults with sickle cell disease during vaso-occlusive crises treated in an infusion center.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.